Q2 2024 Biomarin Pharmaceutical Inc Earnings Call

August 5, 2024

Operator: Please note that this call is being recorded. All lines have been placed on mute to prevent any background noise. After the speakers’ remarks, there will be a question-and-answer session. If you'd like to ask a question, simply press star, then the number one on your telephone keypad. To withdraw your question, press star one again. I will now turn the call over to Traci McCarty, Head of Investor Relations. You may begin your conference.

Speaker Change: Good afternoon and welcome to the Biomarin Pharmaceuticals second quarter 2024 conference call. Please note that this call is being recorded. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session.

Speaker Change: If you would like to ask a question. During this time simply press Star then the number one on your telephone keypad to withdraw your question Press Star one again.

Operator: Please note that this call is being recorded. All lines have been placed on mute to prevent any background noise.

Alexander Hardy: I will now turn the call back to CEO Alexander Hardy for his closing remarks.

Operator: After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press star, then the number one on your telephone keypad. To withdraw your question, press star one again. I will now turn the call over to Traci McCarty, Head of Investor Relations. You may begin your conference.

Speaker Change: I will now turn the call over to Traci Mccarty head of Investor Relations you May begin your conference.

Speaker Change: If you would like to ask a question during this time, simply press star, then the number one on your telephone keypad. To withdraw your question, press star one again.

Traci McCarty: Thank you, Operator. To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of Biomarin Pharmaceutical Inc., including expectations regarding Biomarin's financial performance, commercial products and potential future products in different areas of therapeutic search and development. Results may differ materially depending on the progress of Biomarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market and developments by competitors and those factors detailed in Biomarin's filings with the Securities and Exchange Commission, such as 10-Q, 10-K and 8-K reports. In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today. These non-GAAP measures should not be considered in isolation from, as substitutes for or superior to financial measures prepared in accordance with U.S. GAAP, and you can find the related reconciliations to U.S. GAAP in the earnings release and earnings presentation, both of which are available in the Investor Relations section of our website.

Brian Mueller: While we plan to communicate our complete capital allocation strategy at Investor Day, we determined that funding this debt maturity with our balance sheet was an important improvement to our capital structure and a valuable use of shareholder capital. In summary, we are pleased with strong execution demonstrated across the organization during the quarter. With our decision on ROCTAVIAN behind us, we have confidence in our long-term revenue and income growth and operating margin trajectory into 2025 and beyond, further evidenced by this year's significant operating margin improvement and increased earnings per share. We are excited to share details of our long-term plans with you at Investor Day next month. To remind you, we plan to provide details of our new corporate strategy, including a framework for driving operational effectiveness, efficiency, and cost management.

Alexander Hardy: Thank you very much, everybody, and thank you for joining our call with all your questions. We're very proud of the quarter and the strong growth both in revenue and profitability that Biomarin achieved this quarter. I'm very excited about the outlook and raising our guidance on both the top and the bottom lines. We're also very excited about the progress we've made so far this year with the priorities we set out in January.

Speaker Change: I will now turn the call over to Traci McCarty, Head of Investor Relations. You may begin your conference.

Traci Mccarty: Thank you, Operator. To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of Biomarin Pharmaceutical Inc., including expectations regarding Biomarin's financial performance, commercial products, and potential future products in different areas of therapeutic research and development. Results may differ materially depending on the progress of Biomarin's product programs, the actions of regulatory authorities, the availability of capital, future actions in the pharmaceutical market, and developments by competitors, and those factors detailed in Biomarin's filings with the Securities and Exchange Commission, such as 10-Q, 10-K, and 8-K reports.

Traci Mccarty: Thank you, Operator. To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of Biomarin Pharmaceutical Inc.

Traci McCarty: Results may differ materially depending on the progress of Biomarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market and developments by competitors and those factors detailed in Biomarin's filings with the Securities and Exchange Commission, such as 10-Q, 10-K and 8-K reports.

Speaker Change: Results may differ materially depending on the progress of Biomarin <unk> product programs actions of regulatory authorities availability of capital future actions in the pharmaceutical market developments by competitors and those factors detailed in Biomarin filings with the Securities and Exchange Commission, such as 10-Q, 10-K and 8-K reports. In addition, we will use non-GAAP financial measures as defined in regulation G. During the call today. These non-GAAP measures should not be considered in isolation from as substitutes for or superior to financial measures prepared in accordance with U S. GAAP and you can find the related reconciliations to U S. GAAP in the earnings release and earnings. <unk> both of which are available in the Investor Relations section of our website.

Speaker Change: Including expectations regarding Biomarin's financial performance, commercial products, and potential future products in different areas of therapeutic research and development.

Speaker Change: Results may differ materially depending on the progress of Biomarin's product programs.

Traci McCarty: In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today. These non-GAAP measures should not be considered in isolation from, as substitutes for or superior to financial measures prepared in accordance with U.S. GAAP, and you can find the related reconciliations to U.S. GAAP in the earnings release and earnings presentation, both of which are available in the Investor Relations section of our website.

Speaker Change: Actions of Regulatory Authorities

Speaker Change: In addition, we will use non-GAAP financial measures as defined in regulation G. During the call today. These non-GAAP measures should not be considered in isolation from as substitutes for or superior to financial measures prepared in accordance with U S. GAAP and you can find the related reconciliations to U S. GAAP in the earnings release and earnings. <unk> both of which are available in the Investor Relations section of our website.

Speaker Change: Availability of capital, future actions in the pharmaceutical market,

Speaker Change: Developments by Competitors, and those factors detailed in Biomarin's filings with

Speaker Change: Securities and Exchange Commission such as 10-Q, 10-K, and 8-K reports.

Traci Mccarty: In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today. However, these non-GAAP measures should not be considered in isolation from, as substitutes for, or superior to financial measures prepared in accordance with U.S. GAAP. And you can find the related reconciliations to U.S. GAAP in the earnings release and earnings presentation, both of which are available in the investor relations section of our website. On the call from Biomarin Management today are Alexander Hardy, President and Chief Executive Officer, Hank Fuchs, President of Worldwide R&D, Brian Mueller, Executive Vice President, Chief Financial Officer, and Kristen Hubbard, Executive Vice President, Chief Commercial Officer. I will now turn the call over to Biomarin's President and CEO, Alexander Hardy.

Brian Mueller: We will also share updates on our pipeline and life cycle management plans, long-term financial targets including revenue and operating margin for the next several years, and our capital allocation strategy. We are pleased with the work that has been done over the last few quarters and believe we have a very compelling growth story to share. Thank you for your continued support, and we will now open the call to your questions. Operator.

Speaker Change: In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today. These non-GAAP measures should not be considered in isolation from

Speaker Change: <unk> both of which are available in the Investor Relations section of our website.

Speaker Change: As substitutes for, or superior to, financial measures prepared in accordance with US GAAP, and you can find related reconciliations to US GAAP in the earnings release and earnings presentation, both of which are available in the investor relations section of our website.

Traci McCarty: On the call from Biomarin management today are Alexander Hardy, President and Chief Executive Officer; Hank Fuchs, President of Worldwide R&D; Brian Mueller, Executive Vice President, Chief Financial Officer; and Cristin Hubbard, Executive Vice President, Chief Commercial Officer. I will now turn the call over to Biomarin's President and CEO, Alexander Hardy.

Operator: Thank you. We will now take your questions. If you have dialed in and would like to ask a question, please press star 1 on your telephone keypad to raise your hand and join the queue. If you would like to withdraw your question, simply press star 1 again. If you are dialed in and listening via loudspeaker on your device, please pick up your handset and ensure that your device is not on mute when asking your question. We kindly ask that you limit yourself to one question and one follow-up to allow time for all participants. Our first question comes from Phil Nado with TD Cowen. Please go ahead.

Speaker Change: On the call from Biomarin Management today are Alexander Hardy, President and Chief Executive Officer, Hank Fuchs, President of Worldwide R&D, Brian Mueller, Executive Vice President, Chief Financial Officer, and Kristen Hubbard, Executive Vice President, Chief Commercial Officer.

Speaker Change: Hardy.

Alexander Hardy: Thank you, Traci, and good afternoon, everyone. Thank you all for joining us today. We're pleased to today share our strong quarterly results and the decision on ROCTAVIAN. This quarter, we've also made significant progress towards finalizing BioMarin's evolved corporate vision and strategy. This work gives us confidence we can amplify our progress and mission to deliver more impact on patients' lives and greater value for shareholders. We look forward to sharing more about our vision and strategy, the sustainable growth, accelerated profitability next month at Investor Day. Moving briefly to specific progress made in the quarter on our four strategic priorities outlined in January. Beginning with our opportunity to accelerate and maximize VOXZOGO, we were pleased with continued strong demand and patient uptake in all geographies, resulting in nearly 900 children beginning VOXZOGO treatment in the first half of this year, the highest in VOXZOGO history. And now the third year of our commercial launch, we have seen rapid global expansion, VOXZOGO across all ages and particularly from families pursuing treatment for their infants and young children to allow for the maximum potential therapeutic benefit.

Alexander Hardy: Reminder of those priorities, one was to accelerate and maximize the Boxergo opportunity, and as you can see from these strong results, Boxergo is growing very, very well in just its first indication, and we're less than 20% penetrated in that, and we have a path forward with five additional indications, and we made progress so far this year on those, both in terms of regulatory feedback and progress in the development program. Secondly, to establish the Roktavian opportunity, and today, with this decision, we have clarity on a path forward, and a position for Roktavian within the portfolio.

Speaker Change: We're pleased to today show our strong quarterly results. And the decision on rotated. This quarter, we also made significant progress towards finalizing biomarin evolved coopervision structured. This what gives us confidence, we can amplify our progress and mission to deliver more impact on patients' lives. Greater value for shareholders. We look forward to sharing more about our vision and strategy to sustainable growth accelerated profitability next month at Investor Day. Moving briefly to specific progress made in the quarter on our four strategic priorities outlined in January. Beginning with our opportunity to accelerate and maximize book Saga. We were pleased with continued strong demand and patient uptake in all geographies, resulting in nearly 900 children beginning books OCA treatment in the first half of this year the highest in <unk> history. And now the third year of our commercial launch we have seen rapid global expansion for OXXO go across all ages, and particularly from families pursuing treatment for their infants and young children to allow for the maximum potential therapeutic benefit.

Speaker Change: I will now turn the call over to Biomarin's President and CEO , Alexander Hardy.

Speaker Change: And the decision on rotated. This quarter, we also made significant progress towards finalizing biomarin evolved coopervision structured. This what gives us confidence, we can amplify our progress and mission to deliver more impact on patients' lives. Greater value for shareholders. We look forward to sharing more about our vision and strategy to sustainable growth accelerated profitability next month at Investor Day. Moving briefly to specific progress made in the quarter on our four strategic priorities outlined in January. Beginning with our opportunity to accelerate and maximize book Saga. We were pleased with continued strong demand and patient uptake in all geographies, resulting in nearly 900 children beginning books OCA treatment in the first half of this year the highest in <unk> history. And now the third year of our commercial launch we have seen rapid global expansion for OXXO go across all ages, and particularly from families pursuing treatment for their infants and young children to allow for the maximum potential therapeutic benefit.

Alexander Hardy: Thank you, Traci, and good afternoon, everyone. Thank you all for joining us today.

Alexander Hardy: We're excited by the signs we're seeing of progress, but we're also confident in our strategy of reducing the expenditure down to $60 million in 2025 and confident in our ability for this product to be profitable in that time frame. Thirdly, as you can see so far this year, we prioritized our R&D efforts. We focus on three programs that we're most excited about. We've made consequential decisions around stopping programs that don't meet the high bar for innovation.

Speaker Change: This quarter, we also made significant progress towards finalizing biomarin evolved coopervision structured. This what gives us confidence, we can amplify our progress and mission to deliver more impact on patients' lives. Greater value for shareholders. We look forward to sharing more about our vision and strategy to sustainable growth accelerated profitability next month at Investor Day. Moving briefly to specific progress made in the quarter on our four strategic priorities outlined in January. Beginning with our opportunity to accelerate and maximize book Saga. We were pleased with continued strong demand and patient uptake in all geographies, resulting in nearly 900 children beginning books OCA treatment in the first half of this year the highest in <unk> history. And now the third year of our commercial launch we have seen rapid global expansion for OXXO go across all ages, and particularly from families pursuing treatment for their infants and young children to allow for the maximum potential therapeutic benefit.

Alexander Hardy: Thank you, Traci, and good afternoon, everyone. Thank you all for joining us today. We're pleased to today share our strong quarterly results and the decision on Roktavion.

Alexander Hardy: We're pleased to today share our strong quarterly results and the decision on Roktavia. This quarter, we've also made significant progress towards finalizing Biomarin's evolved corporate vision and strategy. This work gives us confidence we can amplify our progress and mission to deliver more impact on patients' lives and greater value for Shell. We look forward to sharing more about our vision and strategy for sustainable growth and accelerated profitability next month at Invest Today.

Speaker Change: This what gives us confidence, we can amplify our progress and mission to deliver more impact on patients' lives. Greater value for shareholders. We look forward to sharing more about our vision and strategy to sustainable growth accelerated profitability next month at Investor Day. Moving briefly to specific progress made in the quarter on our four strategic priorities outlined in January. Beginning with our opportunity to accelerate and maximize book Saga. We were pleased with continued strong demand and patient uptake in all geographies, resulting in nearly 900 children beginning books OCA treatment in the first half of this year the highest in <unk> history. And now the third year of our commercial launch we have seen rapid global expansion for OXXO go across all ages, and particularly from families pursuing treatment for their infants and young children to allow for the maximum potential therapeutic benefit.

Phil Nadeau: Good afternoon. Thanks for taking our question on the updated ROCTAVIAN strategy. Can you talk a bit more about the changes? Aside from eliminating a bunch of territories, are you changing the strategy or investment in the regions where you're going to stay, US, Germany, and Italy?

Alexander Hardy: This quarter, we've also made significant progress towards finalizing Biomarin's evolved corporate vision and strategy.

Speaker Change: Greater value for shareholders. We look forward to sharing more about our vision and strategy to sustainable growth accelerated profitability next month at Investor Day. Moving briefly to specific progress made in the quarter on our four strategic priorities outlined in January. Beginning with our opportunity to accelerate and maximize book Saga. We were pleased with continued strong demand and patient uptake in all geographies, resulting in nearly 900 children beginning books OCA treatment in the first half of this year the highest in <unk> history. And now the third year of our commercial launch we have seen rapid global expansion for OXXO go across all ages, and particularly from families pursuing treatment for their infants and young children to allow for the maximum potential therapeutic benefit.

Speaker Change: We look forward to sharing more about our vision and strategy to sustainable growth accelerated profitability next month at Investor Day. Moving briefly to specific progress made in the quarter on our four strategic priorities outlined in January. Beginning with our opportunity to accelerate and maximize book Saga. We were pleased with continued strong demand and patient uptake in all geographies, resulting in nearly 900 children beginning books OCA treatment in the first half of this year the highest in <unk> history. And now the third year of our commercial launch we have seen rapid global expansion for OXXO go across all ages, and particularly from families pursuing treatment for their infants and young children to allow for the maximum potential therapeutic benefit.

Alexander Hardy: This work gives us confidence we can amplify our progress and mission to deliver more impact on patients' lives and greater value for shareholders.

Alexander Hardy: Moving briefly to specific progress made in the quarter on our four strategic priorities outlined in January. Beginning with our opportunity to accelerate and maximize VOXZOGO, we were pleased with continued strong demand and patient uptake in all geographies, resulting in nearly 900 children beginning VOXZOGO treatment in the first half of this year, the highest in VOXZOGO history. And now the third year of our commercial launch, we have seen rapid global expansion, VOXZOGO across all ages and particularly from families pursuing treatment for their infants and young children to allow for the maximum potential therapeutic benefit.

Speaker Change: Moving briefly to specific progress made in the quarter on our four strategic priorities outlined in January. Beginning with our opportunity to accelerate and maximize book Saga. We were pleased with continued strong demand and patient uptake in all geographies, resulting in nearly 900 children beginning books OCA treatment in the first half of this year the highest in <unk> history. And now the third year of our commercial launch we have seen rapid global expansion for OXXO go across all ages, and particularly from families pursuing treatment for their infants and young children to allow for the maximum potential therapeutic benefit.

Alexander Hardy: Thank you all for joining us today. We're really excited and proud of the progress we've made on increasing profitability faster than revenue. You can see that with our results this quarter and our increasing guidance. So we're looking forward to seeing you all at INVEST today to share our outlook for the future, Biomarin. It's very, very exciting. Thank you very much for joining us and I wish you a good rest of your day.

Alexander Hardy: We look forward to sharing more about our vision and strategy for sustainable growth and accelerated profitability next month at Invest Today.

Alexander Hardy: Yeah. Thanks very much for the question. So we're very pleased with the clarity that we're able to provide for ROCTAVIAN. If you remember, we said we would, by Investor Day, communicate the timing, the criteria for making a decision. We're pleased to be able to share this at Q2. So the approach that we're taking allows us to focus geographically on those three geographies: United States, Germany, and Italy, and also to focus our efforts within those geographies on what we have seen and we've learned to be the most important thing for the success of ROCTAVIAN, which is the patient pull-through activities mainly at the site level. So that's where we're going to focus geographically, but also in terms of types of activity within those geographies. We're also focusing our activities from an R&D and development perspective.

Speaker Change: Beginning with our opportunity to accelerate and maximize book Saga. We were pleased with continued strong demand and patient uptake in all geographies, resulting in nearly 900 children beginning books OCA treatment in the first half of this year the highest in <unk> history. And now the third year of our commercial launch we have seen rapid global expansion for OXXO go across all ages, and particularly from families pursuing treatment for their infants and young children to allow for the maximum potential therapeutic benefit.

Alexander Hardy: Moving briefly to specific progress made in the quarter on our four strategic priorities outlined in January, beginning with our opportunity to accelerate and maximize VoxOgo, we were pleased with continued strong demand and patient uptake in all geographies, resulting in nearly 900 children beginning VoxOgo treatment in the first half of this year, the highest in VoxOgo's history.

Operator: This concludes today's conference. You may now disconnect.

Alexander Hardy: Moving briefly to specific progress made in the quarter on our four strategic priorities outlined in January .

Operator: Please wait; the conference will begin shortly.

Alexander Hardy: Beginning with our opportunity to accelerate and maximize VoxOgo, we were pleased with continued strong demand and patient uptake in all geographies.

Speaker Change: And now the third year of our commercial launch we have seen rapid global expansion for OXXO go across all ages, and particularly from families pursuing treatment for their infants and young children to allow for the maximum potential therapeutic benefit.

Alexander Hardy: Resulting in nearly 900 children beginning VoxOgo treatment in the first half of this year, the highest in VoxOgo's history.

Alexander Hardy: In now the third year of our commercial launch, we have seen rapid global expansion, Voxogo across all ages, and particularly from families pursuing treatment for their infants and young children to allow for the maximum potential therapeutic benefits. Outside of the U.S., we've seen significant penetration in key strategic markets, but still with room to grow in those markets. In the United States, our largest potential market opportunity, we saw a strong uptake of children of all ages, but note that new prescriptions for children under the age of five accounted for the majority.

Alexander Hardy: Outside of the U.S., we've seen significant penetration in key strategic markets, but still with room to grow in those markets. In the United States, our largest potential market opportunity, we saw a strong uptake, children of all ages and note that new prescriptions for children under the age of five accounted for the majority. Going forward, we intend to leverage the momentum we're seeing in the United States and expand penetration rates across all other geographies. Even with this tremendous progress, we are still at the beginning of realizing the full opportunity with VOXZOGO in achondroplasia, and we intend to build on our expanding leadership to rapidly advance across multiple new indications. In the quarter, we made significant progress advancing the development programs in hypochondroplasia, idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency and Hank will provide an update momentarily.

Speaker Change: Still with room to grow in those markets. In the United States, our largest potential market opportunity. We saw strong uptake children of all ages, but note that new prescriptions for children under the age of five accounted for the majority. Going forward, we intend to leverage the momentum we're seeing in the United States. Expand penetration rates across all other geographies. Even with this tremendous progress we are still at the beginning of realizing the full opportunity with folks saga in achondroplasia. And we intend to build on our expanding leadership to rapidly advance across multiple new indications. In the quarter, we made significant progress advancing the development programs and hypochondria pleasure idiopathic short stature. Syndrome, China syndrome, and <unk> deficiency. To provide an update momentarily. To provide an update momentarily.

Speaker Change: In the United States, our largest potential market opportunity. We saw strong uptake children of all ages, but note that new prescriptions for children under the age of five accounted for the majority. Going forward, we intend to leverage the momentum we're seeing in the United States. Expand penetration rates across all other geographies. Even with this tremendous progress we are still at the beginning of realizing the full opportunity with folks saga in achondroplasia. And we intend to build on our expanding leadership to rapidly advance across multiple new indications. In the quarter, we made significant progress advancing the development programs and hypochondria pleasure idiopathic short stature. Syndrome, China syndrome, and <unk> deficiency. To provide an update momentarily. To provide an update momentarily.

Alexander Hardy: Outside of the US, we've seen significant penetration in key strategic markets, but still with room to grow in those markets.

Speaker Change: Going forward, we intend to leverage the momentum we're seeing in the United States. Expand penetration rates across all other geographies. Even with this tremendous progress we are still at the beginning of realizing the full opportunity with folks saga in achondroplasia. And we intend to build on our expanding leadership to rapidly advance across multiple new indications. In the quarter, we made significant progress advancing the development programs and hypochondria pleasure idiopathic short stature. Syndrome, China syndrome, and <unk> deficiency. To provide an update momentarily. To provide an update momentarily.

Alexander Hardy: We are stopping our life cycle development activities just to focus on the current main indication. We'll continue to generate long-term data. We're also reducing our manufacturing expenses associated with ROCTAVIAN. All of this allows us to operate in an envelope of $60 million in 2025 and underpins our confidence that we're going to be able to achieve profitability for ROCTAVIAN in that time with this focused strategy.

Alexander Hardy: In the United States, our largest potential market opportunity, we saw strong uptake, children of all ages. But note that new prescriptions for children under the age of five accounted for the majority.

Alexander Hardy: Going forward, we intend to leverage the momentum we're seeing in the United States and expand penetration rates across all other geographies. Even with this tremendous progress, we are still at the beginning of realizing the full opportunity with VOXZOGO in achondroplasia, and we intend to build on our expanding leadership to rapidly advance across multiple new indications. In the quarter, we made significant progress advancing the development programs in hypochondroplasia, idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency and Hank will provide an update momentarily.

Speaker Change: Expand penetration rates across all other geographies. Even with this tremendous progress we are still at the beginning of realizing the full opportunity with folks saga in achondroplasia. And we intend to build on our expanding leadership to rapidly advance across multiple new indications. In the quarter, we made significant progress advancing the development programs and hypochondria pleasure idiopathic short stature. Syndrome, China syndrome, and <unk> deficiency. To provide an update momentarily. To provide an update momentarily.

Speaker Change: Even with this tremendous progress we are still at the beginning of realizing the full opportunity with folks saga in achondroplasia. And we intend to build on our expanding leadership to rapidly advance across multiple new indications. In the quarter, we made significant progress advancing the development programs and hypochondria pleasure idiopathic short stature. Syndrome, China syndrome, and <unk> deficiency. To provide an update momentarily. To provide an update momentarily.

Alexander Hardy: Going forward, we intend to leverage the momentum we're seeing in the United States and expand penetration rates across all other geographies.

Speaker Change: And we intend to build on our expanding leadership to rapidly advance across multiple new indications. In the quarter, we made significant progress advancing the development programs and hypochondria pleasure idiopathic short stature. Syndrome, China syndrome, and <unk> deficiency. To provide an update momentarily. To provide an update momentarily.

Alexander Hardy: Even with this tremendous progress, we are still at the beginning of realizing the full opportunity with VoxOgo in achondroplasia. And we intend to build on our expanding leadership to rapidly advance across multiple new indications.

Speaker Change: In the quarter, we made significant progress advancing the development programs and hypochondria pleasure idiopathic short stature. Syndrome, China syndrome, and <unk> deficiency. To provide an update momentarily. To provide an update momentarily.

Alexander Hardy: In the quarter, we made significant progress advancing the development programs in hypochondroplasia, idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency and Hank will provide an update momentarily.

Alexander Hardy: And we intend to build on our expanding leadership to rapidly advance across multiple new indications. In the quarter, we made significant progress advancing the development programs in hyperchondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, and shock deficiency, and Hank will provide an update momentarily. The growing body of evidence supporting the safety profile, the proven mechanism of action, and the expanding data across many attributes beyond height give us a high degree of confidence in our sustainable leadership strategy across multiple growth-related conditions.

Phil Nadeau: When will we hear the next update on the strategy? It sounds like this is a framework, an initial strategy, but you're going to continue to identify the investment and success of ROCTAVIAN. When do you think you'd be in a position to give a further confirmation of the strategy or revision of the strategy if necessary?

Speaker Change: Syndrome, China syndrome, and <unk> deficiency. To provide an update momentarily. To provide an update momentarily.

Speaker Change: To provide an update momentarily. To provide an update momentarily.

Speaker Change: To provide an update momentarily.

Hank Fuchs: In the quarter, we made significant progress advancing the development programs in hyperchondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, and shocks deficiency, and Hank will provide an update momentarily.

Alexander Hardy: The growing body of evidence supporting the safety profile, the proven mechanism of action and the expanding data across many attributes beyond height give us a high degree of confidence in our sustainable leadership strategy across multiple growth-related conditions. The second priority to establish the ROCTAVIAN opportunity was addressed today in a separate press release. The result of our evaluation over the last couple of quarters supported our decision to enable ROCTAVIAN ability to contribute to long-term revenue growth while balancing resource allocation consistent with BioMarin's profitability. This decision on ROCTAVIAN was based on four conclusions: One, our belief in the therapeutic profile of ROCTAVIAN and its role in hemophilia A.; second, our understanding that a launch like this takes time; third, signs of progress in the United States, Germany and Italy; and lastly, a revised expense profile that gives us confidence ROCTAVIAN will contribute to profitability.

Alexander Hardy: Now, this is a decision going forward for ROCTAVIAN. We will continue to update you on our progress. As we've said before, the success metric we're all focused on is patients infused and treated. And so we'll update you on our progress on those metrics. But this is our strategy. We've reached a decision. We have confidence that ROCTAVIAN can be a contributor to BioMarin's profitability going forward.

Hank Fuchs: The growing body of evidence supporting the safety profile, the proven mechanism of action and the expanding data across many attributes beyond height give us a high degree of confidence in our sustainable leadership strategy across multiple growth-related conditions.

The second priority to establish the Octavian opportunity was addressed today in a separate press release. The results of our evaluation over the last couple of quarters supported our decision to enable Octavian <unk> ability to contribute to long term revenue growth, while balancing resource allocation consistent with biomarin profitability. This decision on rocked Hagen was based on full conclusions one our belief in the therapeutic profile of Octavian and its role in hemophilia a. Understanding that a launch like this takes time. Good signs of progress in the United States, Germany, and Italy. Lastly, our revised expense profile that gives us confidence repaving will contribute to profitability.

Speaker Change: The results of our evaluation over the last couple of quarters supported our decision to enable Octavian <unk> ability to contribute to long term revenue growth, while balancing resource allocation consistent with biomarin profitability. This decision on rocked Hagen was based on full conclusions one our belief in the therapeutic profile of Octavian and its role in hemophilia a. Understanding that a launch like this takes time. Good signs of progress in the United States, Germany, and Italy. Lastly, our revised expense profile that gives us confidence repaving will contribute to profitability.

Alexander Hardy: The result of our evaluation over the last couple of quarters supported our decision to enable ROCTAVIAN ability to contribute to long-term revenue growth while balancing resource allocation consistent with BioMarin's profitability. This decision on ROCTAVIAN was based on four conclusions: One, our belief in the therapeutic profile of ROCTAVIAN and its role in hemophilia A.; second, our understanding that a launch like this takes time; third, signs of progress in the United States, Germany and Italy; and lastly, a revised expense profile that gives us confidence ROCTAVIAN will contribute to profitability.

Alexander Hardy: The second priority, to establish the Roktavian opportunity, was addressed today in a separate press release. The result of our evaluation over the last couple of quarters supported our decision to enable Roktavian's ability to contribute to long-term revenue growth while balancing resource allocation consistent with Biomarin's profitability. This decision on Roctavian was based on four conclusions. One, our belief in the therapeutic profile of Roctavian and its role in Haemophilia A.

Hank Fuchs: The second priority, to establish the Roktavian opportunity, was addressed today in a separate press release.

Hank Fuchs: The result of our evaluation over the last couple of quarters supported our decision to enable Roktavian's ability to contribute to long-term revenue growth while balancing resource allocation consistent with Biomarin's profitability.

Phil Nadeau: That's very helpful. Thanks for taking our questions.

Speaker Change: This decision on rocked Hagen was based on full conclusions one our belief in the therapeutic profile of Octavian and its role in hemophilia a. Understanding that a launch like this takes time. Good signs of progress in the United States, Germany, and Italy. Lastly, our revised expense profile that gives us confidence repaving will contribute to profitability.

Operator: Our next question comes from Salveen Richter with Goldman Sachs. Please go ahead.

Salveen Richter: Good afternoon. Thank you for taking my questions. I just want to dig in further into ROCTAVIAN. With regard to the break-even situation by year-end 2025, could you just speak to the metrics that are giving you confidence at this time point? Is it the demand aspect? Is it 340B dynamics improving in the US? And just maybe help us understand where all these moving parts are coming from to give you that conclusion.

Speaker Change: Understanding that a launch like this takes time. Good signs of progress in the United States, Germany, and Italy. Lastly, our revised expense profile that gives us confidence repaving will contribute to profitability.

Speaker Change: Good signs of progress in the United States, Germany, and Italy. Lastly, our revised expense profile that gives us confidence repaving will contribute to profitability.

Hank Fuchs: This decision on Roctavian was based on four conclusions. One, our belief in the therapeutic profile of Roctavian and its role in Haemophilia A. Second, our understanding that a launch like this takes time.

Speaker Change: Lastly, our revised expense profile that gives us confidence repaving will contribute to profitability.

Alexander Hardy: Second, our understanding that a launch like this takes time. 3rd Signs of Progress in the United States, Germany, and Italy. And lastly, a revised expense profile that gives us confidence that Octavian will contribute to profitability. By focusing commercial research and manufacturing efforts, we expect to reduce the annual direct Octavian expenses to approximately $60 million. We will begin to operationalize these reductions in Roktaven expenses by the end of this year, so the new lowered expenses are in effect beginning for the full year 2025.

Alexander Hardy: By focusing commercial, research and manufacturing efforts, we expect to reduce the annual direct ROCTAVIAN expenses to approximately $60 million. We will begin to operationalize these reductions in ROCTAVIAN expenses by the end of this year. So, the new lowered expenses are in effect beginning for the full year 2025. As a result of these changes, the Company is committed to ROCTAVIAN being profitable by the end of 2025. In addition to changes in resource allocation to allow ROCTAVIAN to realize its potential, as well as to contribute to Biomarin's profitability, we will reorganize the global ROCTAVIAN support team to be a dedicated unit that will focus exclusively on ROCTAVIAN success and enable the rest of the enterprise to focus only on the rest of our portfolio. We believe that this direct accountability will enable ROCTAVIAN's maximum potential of this revised strategy. Christian will elaborate on the incremental progress we are seeing in the three prioritized markets where we have made progress in securing reimbursement and providing access to patients.

Speaker Change: Marshall Research and manufacturing efforts, we expect to reduce the annual direct rotating expenses to approximately $60 million. We will begin to operationalize these reductions in <unk> expenses by the end of this year, so the new lowered expenses. In effect beginning for the full year 2025. As a result of these changes the company has committed to Octavian being profitable by the end of 2025. In addition to changes in resource allocation to allow octavian to realize its potential as well as to contribute to biomarin profitability, we will reorganize the global rotate and support team to be a dedicated unit that will focus exclusively on Octavian success and enable the rest of the enterprise to focus. Only on the rest of our portfolio. We believe that this direct accountability will enable pagans maximum potential. This revised strategy. Christian will elaborate on the incremental progress we are seeing in the three prioritized markets, where we have made progress in securing reimbursement and providing access to patients.

Hank Fuchs: 3rd Signs of Progress in the United States

Hank Fuchs: Germany and Italy and lastly a revised expense profile that gives us confidence Octavian will contribute to profitability

Cristin Hubbard: Yeah. Thank you so much for the question. This is Cristin Hubbard. I think that really the progress that we're seeing, and I'll be specific in the three geographies, is that in the United States, we really are encouraged with the three patients that were treated in the quarter. And really what this is around is ensuring that the sites are operationally ready to infuse product. And we've seen a fourfold increase in this. Our commercial team is very focused here, and we've seen a fourfold increase in this just since January. But then also, as Alexander alluded to, the patient pull-through. And it's really about building comfort around working through the single case agreements that we know will be true to ensure that patients are being treated.

Speaker Change: We will begin to operationalize these reductions in <unk> expenses by the end of this year, so the new lowered expenses. In effect beginning for the full year 2025. As a result of these changes the company has committed to Octavian being profitable by the end of 2025. In addition to changes in resource allocation to allow octavian to realize its potential as well as to contribute to biomarin profitability, we will reorganize the global rotate and support team to be a dedicated unit that will focus exclusively on Octavian success and enable the rest of the enterprise to focus. Only on the rest of our portfolio. We believe that this direct accountability will enable pagans maximum potential. This revised strategy. Christian will elaborate on the incremental progress we are seeing in the three prioritized markets, where we have made progress in securing reimbursement and providing access to patients.

Hank Fuchs: By focusing commercial research and manufacturing efforts, we expect to reduce the annual direct Roktavian expenses to approximately $60 million.

Speaker Change: In effect beginning for the full year 2025. As a result of these changes the company has committed to Octavian being profitable by the end of 2025. In addition to changes in resource allocation to allow octavian to realize its potential as well as to contribute to biomarin profitability, we will reorganize the global rotate and support team to be a dedicated unit that will focus exclusively on Octavian success and enable the rest of the enterprise to focus. Only on the rest of our portfolio. We believe that this direct accountability will enable pagans maximum potential. This revised strategy. Christian will elaborate on the incremental progress we are seeing in the three prioritized markets, where we have made progress in securing reimbursement and providing access to patients.

Speaker Change: As a result of these changes the company has committed to Octavian being profitable by the end of 2025. In addition to changes in resource allocation to allow octavian to realize its potential as well as to contribute to biomarin profitability, we will reorganize the global rotate and support team to be a dedicated unit that will focus exclusively on Octavian success and enable the rest of the enterprise to focus. Only on the rest of our portfolio. We believe that this direct accountability will enable pagans maximum potential. This revised strategy. Christian will elaborate on the incremental progress we are seeing in the three prioritized markets, where we have made progress in securing reimbursement and providing access to patients.

Hank Fuchs: We will begin to operationalize these reductions in Roktaven expenses by the end of this year, so the new lowered expenses are in effect beginning for the full year 2025.

Alexander Hardy: In addition to changes in resource allocation to allow ROCTAVIAN to realize its potential, as well as to contribute to Biomarin's profitability, we will reorganize the global ROCTAVIAN support team to be a dedicated unit that will focus exclusively on ROCTAVIAN success and enable the rest of the enterprise to focus only on the rest of our portfolio. We believe that this direct accountability will enable ROCTAVIAN's maximum potential of this revised strategy. Christian will elaborate on the incremental progress we are seeing in the three prioritized markets where we have made progress in securing reimbursement and providing access to patients.

Speaker Change: In addition to changes in resource allocation to allow octavian to realize its potential as well as to contribute to biomarin profitability, we will reorganize the global rotate and support team to be a dedicated unit that will focus exclusively on Octavian success and enable the rest of the enterprise to focus. Only on the rest of our portfolio. We believe that this direct accountability will enable pagans maximum potential. This revised strategy. Christian will elaborate on the incremental progress we are seeing in the three prioritized markets, where we have made progress in securing reimbursement and providing access to patients.

Alexander Hardy: As a result of these changes, the company is committed to Roktavion being profitable by the end of 2025. In addition to changes in resource allocation to allow Roktavion to realize its potential, as well as to contribute to Biomarin's profitability, we will reorganise the global Roktavion support team to be a dedicated unit that will focus exclusively on Roktavion's success and enable the rest of the enterprise to focus only on the rest of our portfolio. We believe that this direct accountability will enable Octavian to maximize the potential of this revised strategy.

Hank Fuchs: As a result of these changes, the company is committed to Roktavion being profitable by the end of 2025.

Hank Fuchs: In addition to changes in resource allocation to allow Roktavion to realize its potential, as well as to contribute to Biomarin's profitability, we will reorganize the global Roktavion support team to be a dedicated unit that will focus exclusively on Roktavion's success.

Cristin Hubbard: So with that type of experience, we recognize that that will create more progress moving forward and a comfort with doing so. We're seeing a lot of that in the US today. In Italy, in particular, we are really pleased with the rapid progress that we've made in ROCTAVIAN since only receiving pricing and reimbursement since January. So we've treated a couple of patients there, and we're seeing large amounts of strong advocacy from KOLs as well as experience in the sites. Then lastly, with Germany, as I'd said a little bit earlier, we have reached a negotiated agreement with the sub-insurer. We are happy to report that we now have approximately 25% of patient lives covered in Germany, and we will continue to push forward with progress with the sub-insurers there. We know there's demand.

Speaker Change: Only on the rest of our portfolio. We believe that this direct accountability will enable pagans maximum potential. This revised strategy. Christian will elaborate on the incremental progress we are seeing in the three prioritized markets, where we have made progress in securing reimbursement and providing access to patients.

Speaker Change: We believe that this direct accountability will enable pagans maximum potential. This revised strategy. Christian will elaborate on the incremental progress we are seeing in the three prioritized markets, where we have made progress in securing reimbursement and providing access to patients.

Speaker Change: This revised strategy. Christian will elaborate on the incremental progress we are seeing in the three prioritized markets, where we have made progress in securing reimbursement and providing access to patients.

Speaker Change: Christian will elaborate on the incremental progress we are seeing in the three prioritized markets, where we have made progress in securing reimbursement and providing access to patients.

Hank Fuchs: and enable the rest of the enterprise to focus only on the rest of our portfolio.

Hank Fuchs: We believe that this direct accountability will enable Octavian's maximum potential of this revised strategy. Gritsen will elaborate on the incremental progress we are seeing in the three prioritized markets, where we have made progress in securing reimbursement and providing access to patients.

Alexander Hardy: Kristen will elaborate on the incremental progress we are seeing in the three prioritized markets where we have made progress in securing reimbursement and providing access to patients. The third priority is our focus on the most promising R&D assets. We made continued progress on BMN-351, BMN-349, and BMN-333, the three prioritized programs announced last quarter. All five new indications with FOXOGO have also advanced and developed, and we look forward to providing an update on all these programs, as well as our internal and external innovation strategy at Investor Day, including how it fits into our capital allocation strategy.

Alexander Hardy: The third priority is our focus on the most promising R&D assets. We made continued progress on BMN 351, BMN 349 and BMN 333, the three prioritized programs announced last quarter. All five new indications with VOXZOGO also advanced and developed. And we look forward to providing an update on all these programs as well as our internal external innovation strategy at Investor Day, including how it fits into our capital allocation strategy. Lastly, our fourth priority to increase profitability faster than originally planned. Today, we announced 20% growth in total revenues and 78% growth in non-GAAP earnings per share, proof of our ability to drive growth, realize cost efficiencies and accelerate profitability. Strength across the business resulted in today's increase in all full year guidance items. Today's results are just the beginning of the profitability expansion we are planning over the next couple of years and into the longer term.

Christian: Men 333, the three prioritized programs announced last quarter. Five new indications with Fox AGA also advanced in development and we look forward to providing an update on all of these programs as well as our internal and external innovation strategy at Investor day, including how it fits into our capital allocation strategy. Lastly, our fourth priority to increase profitability faster than originally planned. Today, we announced 20% growth in total revenues and 78% growth in non-GAAP earnings per share. Our ability to drive growth realized cost efficiencies and accelerate profitability. Strength across the business resulted in today's increase in all full year guidance items. Today's results are just the beginning. Of the profitability expansion, we are planning over the next couple of years and into the longer term.

Gritsen: The third priority is our focus on the most promising R&D assets. We made continued progress on BMN-351, BMN-349 and BMN-333, the three prioritized programs announced last quarter.

Christian: Five new indications with Fox AGA also advanced in development and we look forward to providing an update on all of these programs as well as our internal and external innovation strategy at Investor day, including how it fits into our capital allocation strategy. Lastly, our fourth priority to increase profitability faster than originally planned. Today, we announced 20% growth in total revenues and 78% growth in non-GAAP earnings per share. Our ability to drive growth realized cost efficiencies and accelerate profitability. Strength across the business resulted in today's increase in all full year guidance items. Today's results are just the beginning. Of the profitability expansion, we are planning over the next couple of years and into the longer term.

Cristin Hubbard: We do have KOL advocacy in Germany, and we want to continue on that front. These are the types of metrics that we're looking at. I'll pass it to Brian for more.

Brian Mueller: Yeah. Hi, Selviine. This is Brian. Just to add to Cristin's comments, in addition to those signs of progress and our continued execution across those three markets with this focused strategy, that plus this revised $60 million cost envelope is what gives us confidence that we can manage the asset to be profitable next year.

Speaker Change: All five new indications with VOXERGO also advanced and developed. And we look forward to providing an update on all these programs, as well as our internal, external innovation strategy at Investor Day, including how it fits into our capital allocation strategy.

Christian: Lastly, our fourth priority to increase profitability faster than originally planned. Today, we announced 20% growth in total revenues and 78% growth in non-GAAP earnings per share. Our ability to drive growth realized cost efficiencies and accelerate profitability. Strength across the business resulted in today's increase in all full year guidance items. Today's results are just the beginning. Of the profitability expansion, we are planning over the next couple of years and into the longer term.

Alexander Hardy: Today, we announced 20% growth in total revenues and 78% growth in non-GAAP earnings per share, proof of our ability to drive growth, realize cost efficiencies and accelerate profitability. Strength across the business resulted in today's increase in all full year guidance items. Today's results are just the beginning of the profitability expansion we are planning over the next couple of years and into the longer term.

Christian: Today, we announced 20% growth in total revenues and 78% growth in non-GAAP earnings per share. Our ability to drive growth realized cost efficiencies and accelerate profitability. Strength across the business resulted in today's increase in all full year guidance items. Today's results are just the beginning. Of the profitability expansion, we are planning over the next couple of years and into the longer term.

Alexander Hardy: Lastly, our fourth priority, to increase profitability faster than originally planned. Today, we announce 20% growth in total revenues and 78% growth in non-GAAP earnings per share. Proof of our ability to drive growth, realize cost efficiencies, and accelerate profitability. Strength across the business resulted in today's increase in all four-year guidance sites.

Speaker Change: Lastly, our fourth priority, to increase profitability faster than originally planned.

Christian: Our ability to drive growth realized cost efficiencies and accelerate profitability. Strength across the business resulted in today's increase in all full year guidance items. Today's results are just the beginning. Of the profitability expansion, we are planning over the next couple of years and into the longer term.

Operator: Our next question comes from Ellie Murrell with UBS. Please go ahead.

Speaker Change: Today, we announce 20% growth in total revenues and 78% growth in non-GAAP earnings per share. Proof of our ability to drive growth, realize cost efficiencies, and accelerate profitability.

Christian: Strength across the business resulted in today's increase in all full year guidance items. Today's results are just the beginning. Of the profitability expansion, we are planning over the next couple of years and into the longer term.

Ellie Merle: Hey, guys. Thanks for taking the question. Just in terms of 333, how are you thinking about the development of 333 relative to VOXZOGO across different indications? Are there any indications where you would move forward with 333 instead of VOXZOGO? And then just a second question on idiopathic short stature. Given the much larger prevalence, what are your plans to potentially focus on a subset of patients with ISS within that 600,000 prevalence that you've cited? Thanks.

Christian: Today's results are just the beginning. Of the profitability expansion, we are planning over the next couple of years and into the longer term.

Christian: Of the profitability expansion, we are planning over the next couple of years and into the longer term.

Speaker Change: Strength across the business resulted in today's increase in all four-year guidance items.

Alexander Hardy: Also, at the end of July, we were very pleased to have received approval for BRINEURA that expands access to children under the age of three years with CLN2 disease. This expansion now enables treatment for children of all ages with CLNL2 disease regardless of whether they're symptomatic or pre-symptomatic. In this rapidly progressing neurodegenerative disease beginning BRINEURA treatment as early as possible has the potential to alter the natural course of the disease. I want to extend my thanks to the families in our program and Biomarin's R&D team who work diligently to gain this expanded approval on their behalf. In summary, we have made significant progress across the enterprise to reshape operations to enable greater efficiencies and focus on what we do best, create innovative and high-impact medicines for patients. We look forward to seeing you next month to share more specifics on our outlook and strategy. Thank you for your attention. And I will now turn the call over to Cristin to provide an overview on commercial highlights in the quarter.

Alexander Hardy: Today's results are just the beginning of the profitability expansion we are planning over the next couple of years and into the longer term. Also, at the end of July, we were very pleased to have received approval for Benira that expands access to children under the age of three years with CLN2 disease. This expansion now enables treatment for children of all ages with CLNL2 disease, regardless of whether they're symptomatic or pre-symptomatic. In this rapidly progressing neurodegenerative disease, beginning bronera treatment as early as possible has the potential to alter the natural course of the disease.

Speaker Change: Today's results are just the beginning.

Speaker Change: of the profitability expansion we are planning over the next couple of years and into the longer term.

Speaker Change: Also at the end of July , we were very pleased to have received approval for Benira that expands access to children under the age of three years with CLN2 disease.

Hank Fuchs: So hi, Ellie. This is Hank. As far as development plans for 333, our initial thinking is, first of all, to get it into the clinic, which will start shortly, and thereafter to proceed with the most expedited path to approval, leveraging potential for superior efficacy, superior convenience, or both. As regards the program of idiopathic short stature, the opportunity there is in patients with the greatest unmet need, potentially more specifically patients who have severe statural deficiency or patients who have been unsatisfied with available current therapy. So I hope that addresses your questions.

Speaker Change: Of whether they're symptomatic or pre symptomatic. In this rapidly progressing neurodegenerative disease, beginning bernero treatment as early as possible has the potential to alter the natural course of the disease I wanted to extend my thanks the families in our program and Biomarin is R&D team, who worked diligently to gain this expanded approval on their behalf. In summary, we have made significant progress across the enterprise to reshape operations to enable greater efficiencies and focus on what we do best create innovative and high impact medicines for patients. We look forward to seeing you next month. Have more specifics on our outlook and strategy. Thank. Thank you for your attention and I will now turn the call over to Christian to provide an overview on commercial highlights in the quarter.

Speaker Change: In this rapidly progressing neurodegenerative disease, beginning bernero treatment as early as possible has the potential to alter the natural course of the disease I wanted to extend my thanks the families in our program and Biomarin is R&D team, who worked diligently to gain this expanded approval on their behalf. In summary, we have made significant progress across the enterprise to reshape operations to enable greater efficiencies and focus on what we do best create innovative and high impact medicines for patients. We look forward to seeing you next month. Have more specifics on our outlook and strategy. Thank. Thank you for your attention and I will now turn the call over to Christian to provide an overview on commercial highlights in the quarter.

Speaker Change: This expansion now enables treatment for children of all ages with CLNL2 disease, regardless of whether they're symptomatic or pre-symptomatic.

Alexander Hardy: I want to extend my thanks to the families in our program and Biomarin's R&D team who work diligently to gain this expanded approval on their behalf. In summary, we have made significant progress across the enterprise to reshape operations to enable greater efficiencies and focus on what we do best, create innovative and high-impact medicines for patients. We look forward to seeing you next month to share more specifics on our outlook and strategy. Thank you for your attention. And I will now turn the call over to Cristin to provide an overview on commercial highlights in the quarter.

Speaker Change: In this rapidly progressing neurodegenerative disease, beginning bronera treatment as early as possible has the potential to alter the natural course of the disease.

Speaker Change: In summary, we have made significant progress across the enterprise to reshape operations to enable greater efficiencies and focus on what we do best create innovative and high impact medicines for patients. We look forward to seeing you next month. Have more specifics on our outlook and strategy. Thank. Thank you for your attention and I will now turn the call over to Christian to provide an overview on commercial highlights in the quarter.

Alexander Hardy: I want to extend my thanks to the families in our program and Biomarin's R&D team who worked diligently to gain this expanded approval on their behalf. In summary, we have made significant progress across the enterprise to reshape operations, to enable greater efficiencies, and focus on what we do best, creating innovative and high-impact medicines for patients. We look forward to seeing you next month to share more specifics on our outlook and strategy. Thank you for your attention, and I will now turn the call over to Kristen to provide an overview of commercial highlights from the course.

Speaker Change: I want to extend my thanks to the families in our program and Biomarin's R&D team who worked diligently to gain this expanded approval on their behalf.

Speaker Change: We look forward to seeing you next month. Have more specifics on our outlook and strategy. Thank. Thank you for your attention and I will now turn the call over to Christian to provide an overview on commercial highlights in the quarter.

Speaker Change: Have more specifics on our outlook and strategy. Thank. Thank you for your attention and I will now turn the call over to Christian to provide an overview on commercial highlights in the quarter.

Speaker Change: Thank you for your attention and I will now turn the call over to Christian to provide an overview on commercial highlights in the quarter.

Ellie Merle: Great. Thanks.

Speaker Change: We look forward to seeing you next month to share more specifics on our outlook and strategy. Thank you for your attention, and I will now turn the call over to Kristen to provide an overview on commercial highlights in the quarter.

Cristin Hubbard: Thank you, Alexander. I'm pleased to join you on my first quarterly results call since joining Biomarin in May. Now I've been impressed by the breadth of commercial expertise and patient focus at Biomarin, and I'm excited about what lies ahead as we drive continued patient impact through the expansion of our innovative medicines. Turning to quarterly highlights. Strength across our brands drove 20% revenue growth year-over-year, including notable contributions from VOXZOGO, NAGLAZYME, PALYNZIQ, BRINEURA, VIMIZIM and ROCTAVIAN. Moving to specific accelerators in the quarter. VOXZOGO revenues were up 62% to $184 million compared to the second quarter of last year. By the end of the second quarter, 3,500 children were receiving VOXZOGO treatment. As we told you previously, we expect it to reach ample supply capacity by midyear, and we did. We've navigated through the supply constraint of the last few quarters, enabling high market penetration rates in several strategic markets in the quarter, including strong momentum in the United States, our largest potential market. Our successful supply chain efforts allowed customers to normalize stock events in Q2, and we expect this to meet demand going forward.

Operator: Our next question comes from Corey.

Alexander Hardy: Ellie, we'll share more information on the development plans for VOXZOGO at Investor Day. We'll be sharing the specifics around the design of our studies and also the TAMs we're expecting to be able to achieve with those development programs.

Christian: Been impressed by the breadth of commercial expertise and patient focus it Bob Moran and I'm excited about what lies ahead as we drive continued patient impact due to the expansion of our innovative medicines. Turning to quarterly highlights the strength of concert brands drove 20% revenue growth year over year, including notable contributions from Fox Hill, though <unk> LNG from Euro Vimizim and rotating in. Moving to specific accelerators in the quarter October revenues were up 62% to $184 million compared to the second quarter of last year.

Kristen Hubbard: Thank you, Alexander. I'm pleased to join you on my first quarterly results call since joining Biomarin in May. Now I've been impressed by the breadth of commercial expertise and patient focus at Biomarin, and I'm excited about what lies ahead as we drive continued patient impact through the expansion of our innovative medicine. Turning to quarterly highlights, Strength Across Our Brands drove 20% revenue growth year over year, including notable contributions from Soxobo, Naglazyme, Alanzik, Bruneira, Femizim, and Rakuten. Moving to specific accelerators in the quarter, FOGSOGA revenues were up 62% to $184 million compared to the second quarter of last year.

Kristen Hubbard: Thank you, Alexander. I'm pleased to join you on my first quarterly results call since joining Biomarin in May. Now, I've been impressed by the breadth of commercial expertise and patient focus at Biomarin, and I'm excited about what lies ahead as we drive continued patient impact through the expansion of our innovative medicines.

Christian: Turning to quarterly highlights the strength of concert brands drove 20% revenue growth year over year, including notable contributions from Fox Hill, though <unk> LNG from Euro Vimizim and rotating in. Moving to specific accelerators in the quarter October revenues were up 62% to $184 million compared to the second quarter of last year.

Cristin Hubbard: Turning to quarterly highlights. Strength across our brands drove 20% revenue growth year-over-year, including notable contributions from VOXZOGO, NAGLAZYME, PALYNZIQ, BRINEURA, VIMIZIM and ROCTAVIAN. Moving to specific accelerators in the quarter. VOXZOGO revenues were up 62% to $184 million compared to the second quarter of last year. By the end of the second quarter, 3,500 children were receiving VOXZOGO treatment. As we told you previously, we expect it to reach ample supply capacity by midyear, and we did. We've navigated through the supply constraint of the last few quarters, enabling high market penetration rates in several strategic markets in the quarter, including strong momentum in the United States, our largest potential market. Our successful supply chain efforts allowed customers to normalize stock events in Q2, and we expect this to meet demand going forward.

Operator: Our next question comes from Corey Kazimov with Evercore. Please go ahead.

Christian: Moving to specific accelerators in the quarter October revenues were up 62% to $184 million compared to the second quarter of last year.

Kristen Hubbard: Turning to quarterly highlights, strength across our brands drove 20% revenue growth year-over-year, including notable contributions from Foxogo, Naglazyme, Alanzik, Brenura, Vemizem, and Roktavion.

Brian Mueller: Great. Thanks for taking the question. I have a two-part question on VOXZOGO. First, kind of big picture. So assuming you're successful in adding additional indications here, what are your current expectations out of Washington on the potential impact of the IRA when it comes to multi-indication products for orphan diseases? And then the second one is we're thinking about the hypochondroplasia opportunity. Curious how this indication relates to, or how it's similar or different to achondroplasia in terms of the level of market awareness and maybe the motivation on the part of the patients to get treatment as we think about kind of as you proceed through your phase 3 trial. Thanks a lot.

Christian: By the end of the second quarter 3500 children were receiving box to have that treatment. As we told you previously we expect it to reach ample supply of capacity by midyear and we get we've navigated through the supply constraint over the last few quarters, enabling high market penetration rates in several strategic markets in the quarter, including strong momentum in the <unk>. States are largest potential market or. Our successful supply chain efforts allow customers to normalized stock events in Q2, and we expect this to meet demand going forward.

Cristin Hubbard: As we told you previously, we expect it to reach ample supply capacity by midyear, and we did. We've navigated through the supply constraint of the last few quarters, enabling high market penetration rates in several strategic markets in the quarter, including strong momentum in the United States, our largest potential market. Our successful supply chain efforts allowed customers to normalize stock events in Q2, and we expect this to meet demand going forward.

Kristen Hubbard: Moving to specific accelerators in the quarter, FOGSOGA revenues were up 62% to $184 million compared to the second quarter of last year.

Kristen Hubbard: By the end of the second quarter, 3,500 children were receiving Voxogo treatment. As we told you previously, we expected to reach adequate supply capacity by mid-year, and we did. We've navigated through the supply constraints of the last few quarters, enabling high market penetration rates in several strategic markets this quarter, including strong momentum in the United States, our largest potential market. Our successful supply chain efforts allowed customers to normalize stock events in Q2, and we expect this to meet demand going forward.

Kristen Hubbard: By the end of the second quarter, 3,500 children were receiving Boxogo treatment.

Christian: States are largest potential market or. Our successful supply chain efforts allow customers to normalized stock events in Q2, and we expect this to meet demand going forward.

Kristen Hubbard: As we told you previously, we expected to reach ample supply capacity by mid-year, and we did. We have navigated through the supply constraint of the last few quarters, enabling high market penetration rates in several strategic markets in the quarter, including strong momentum in the United States, our largest potential market.

Christian: Our successful supply chain efforts allow customers to normalized stock events in Q2, and we expect this to meet demand going forward.

Cristin Hubbard: Now in the U.S., confidence in VOXZOGO's safety and efficacy from treating physicians and families is solidifying our leadership in achondroplasia and is underscored by our more than 25 years of experience treating children with skeletal dysplasias. We intend to build on strong demand in Q2 to drive continued market expansion in the United States over the coming quarters. Our efforts to expand our base of prescribers for families seeking treatment with VOXZOGO, are rapidly gaining momentum. Now outside the U.S., we've achieved significant penetration rates in all strategic markets and expect to continue to grow in those markets, albeit at a more measured pace. VOXZOGO is the only approved product for achondroplasia and has an extremely strong track record of safety and efficacy with more than 6,000 patient years of evidence to support it.

Kristen Hubbard: Our successful supply chain efforts allowed customers to normalize stock events in Q2, and we expect this to meet demand going forward.

Alexander Hardy: Hey, Corey. Thanks very much for your questions. I'll handle the first one and then hand over to Cristin for the second one. So the first one with regard to the potential impact of IRA. We actually think there'll be minimal impact of IRA on VOXZOGO's life cycle development plans, while the IRA overall, from a legislation standpoint, is a very significant factor for the industry in terms of impacting innovation in many important areas. We don't think it's going to have a significant impact on VOXZOGO, and that's because of the payer mix that we have for VOXZOGO with these indications where we see minimal exposure to Medicare. But I'll hand over now to Hank for the second question.

Kristen Hubbard: Now in the U.S., confidence in Voxogo's safety and efficacy from treating physicians and families is solidifying our leadership in achondroplasia and is underscored by our more than 25 years of experience treating children with skeletal dysplasia. We intend to build on strong demand in Q2 to drive continued market expansion in the U.S. over the coming quarter. Our efforts to expand our base of prescribers for families seeking treatment with OXODO are rapidly gaining momentum.

Christian: We intend to build on strong demand in Q2 to drive continued market expansion in the United States over the coming quarters. Our efforts to expand our base of prescribers for families seeking treatment with Fox out are rapidly gaining momentum. Now outside the U S. We've achieved significant penetration rates in all strategic markets and expect to continue to grow in those markets, albeit at a more measured pace. Are those the only approved product for achondroplasia and has an extremely strong track record of safety and efficacy with more than 6000 patient years of evidence to support it as.

Kristen Hubbard: Now in the U.S., confidence in Voxobo's safety and efficacy from treating physicians and families is solidifying our leadership in achondroplasia and is underscored by our more than 25 years of experience treating children with skeletal dysplasia.

Christian: Our efforts to expand our base of prescribers for families seeking treatment with Fox out are rapidly gaining momentum. Now outside the U S. We've achieved significant penetration rates in all strategic markets and expect to continue to grow in those markets, albeit at a more measured pace. Are those the only approved product for achondroplasia and has an extremely strong track record of safety and efficacy with more than 6000 patient years of evidence to support it as.

Cristin Hubbard: Now outside the U.S., we've achieved significant penetration rates in all strategic markets and expect to continue to grow in those markets, albeit at a more measured pace. VOXZOGO is the only approved product for achondroplasia and has an extremely strong track record of safety and efficacy with more than 6,000 patient years of evidence to support it.

Christian: Now outside the U S. We've achieved significant penetration rates in all strategic markets and expect to continue to grow in those markets, albeit at a more measured pace. Are those the only approved product for achondroplasia and has an extremely strong track record of safety and efficacy with more than 6000 patient years of evidence to support it as.

Kristen Hubbard: We intend to build on strong demand in Q2 to drive continued market expansion in the United States over the coming quarters.

Kristen Hubbard: Our efforts to expand our base of prescribers for families seeking treatment with Oxodo are rapidly gaining momentum.

Speaker Change: Are those the only approved product for achondroplasia and has an extremely strong track record of safety and efficacy with more than 6000 patient years of evidence to support it as.

Kristen Hubbard: Now, outside the US, we've achieved significant penetration rates in all strategic markets and expect to continue to grow in those markets, albeit at a more measured pace. Voxobo is the only approved product for achondroplasia and has an extremely strong track record of safety and efficacy with more than 6,000 patient years of evidence to support it.

Kristen Hubbard: Now, outside the U.S., we've achieved significant penetration rates in all strategic markets and expect to continue to grow in those markets, albeit at a more measured pace.

Cristin Hubbard: This experience facilitated direct entry into our pivotal study in hypochondroplasia our second indication and will set us up for continued expansion into four additional statural indications, none of which have any competitors on the horizon. In my experience, having the opportunity to build the standard of care with a brand like VOXZOGO does not happen that frequently. It's a tremendous opportunity to have an impact on the lives of thousands of children across a multitude of growth-related conditions and I look forward to helping build and sustain our leadership. Turning now to market dynamics in the quarter across our enzyme therapies. We were pleased to see combined quarter-over-quarter growth of 15% compared to Q2 last year. 47% growth of NAGLAZYME year-over-year was driven by the timing of a few large orders and 18% growth in PALYNZIQ was driven by new patient starts in the quarter, primarily in the U.S.

Kristen Hubbard: Voxobo is the only approved product for achondroplasia and has an extremely strong track record of safety and efficacy with more than 6,000 patient years of evidence to support it.

Brian Mueller: Right. And your second question, Corey, just remind me real fast. Yeah. And the differences or similarities or differences between achondroplasia and hypochondroplasia in terms of level of market awareness of these patients and desire to seek treatment? Yeah. Hypochondroplasia doesn't occur as early in life for diagnostic purposes. So it's underdiagnosed, and we're undertaking a lot of effort to increase its diagnosis. Having said that, we're really quite pleased with the rate of progress of the FEDER study. And I'll give you more of an update about that. But as with a lot of genetic conditions, once a therapy is in place, then there's more diagnostic effort undertaken. Once there's more diagnostic effort undertaken, there's more ambition to treat. So during the development program, we're hoping to try to begin the process of increasing awareness and ultimately uptake in the hypochondroplasia population. Perfect. That's helpful, Hank. Thank you.

Kristen Hubbard: This experience facilitated direct entry into our pivotal study in hypochondroplasia, our second indication, and will set us up for continued expansion into four additional spectral indications, none of which have any competitors on the horizon. In my experience, having the opportunity to build the standard of care with a brand like Voxobo does not happen that frequently. It's a tremendous opportunity to have an impact on the lives of thousands of children across a multitude of growth-related conditions, and I look forward to helping build and sustain our leadership.

Speaker Change: And my experience, having the opportunity to build the standard of care with a brand like Fox, although it does not happen that frequently it's a tremendous opportunity to have an impact on the lives of thousands of children across a multitude of growth related conditions, and I look forward to helping build and sustain our leadership. Turning now to market dynamics in the quarter across our enzyme therapies. We were pleased to see combined quarter over quarter growth of 15% compared to Q2 last year. 47% growth in <unk> year over year was driven by the timing of a few large orders and 18% growth in policy, Inc. Was driven by new patient starts in the quarter, primarily in the U S.

Speaker Change: In my experience, having the opportunity to build the standard of care with a brand like Voxobo does not happen that frequently. It's a tremendous opportunity to have an impact on the lives of thousands of children across a multitude of growth-related conditions, and I look forward to helping build and sustain our leadership.

Speaker Change: Turning now to market dynamics in the quarter across our enzyme therapies. We were pleased to see combined quarter over quarter growth of 15% compared to Q2 last year. 47% growth in <unk> year over year was driven by the timing of a few large orders and 18% growth in policy, Inc. Was driven by new patient starts in the quarter, primarily in the U S.

Cristin Hubbard: Turning now to market dynamics in the quarter across our enzyme therapies. We were pleased to see combined quarter-over-quarter growth of 15% compared to Q2 last year. 47% growth of NAGLAZYME year-over-year was driven by the timing of a few large orders and 18% growth in PALYNZIQ was driven by new patient starts in the quarter, primarily in the U.S.

Speaker Change: 47% growth in <unk> year over year was driven by the timing of a few large orders and 18% growth in policy, Inc. Was driven by new patient starts in the quarter, primarily in the U S.

Kristen Hubbard: Turning now to market dynamics in the quarter across our enzyme therapies, we were pleased to see combined quarter-over-quarter growth of 15% compared to Q2 last year. However, 47% growth of Naglozyme year over year was driven by the timing of a few large orders.

Speaker Change: Turning now to market dynamics in the quarter across our enzyme therapies, we were pleased to see combined quarter-over-quarter growth of 15% compared to Q2 last year.

Cristin Hubbard: Now turning to our ROCTAVIAN results. We were encouraged that patients were treated in both the U.S. and Italy, driving approximately $7 million of revenue in the quarter. Alexander has outlined our updated strategy to reduce the resourcing of ROCTAVIAN and the focus on three existing commercial markets, where the drug is approved and reimbursed, which are the United States, Germany and Italy. To expand on this rationale, the decision was brd on signs of progress in those markets, including the successful treatment with ROCTAVIAN at multiple U.S. hemophilia treatment centers. We believe as more and more centers gain patient treatment experience in the U.S., patient flow will become more straightforward. In Germany, we're also making progress. At our last update, we shared that subinsurers had yet to green light reimbursement for ROCTAVIAN treatment despite there being an improved government price. And I'm pleased to share that we have finalized an agreement with one subinsurer, opening access for some launching treatment with ROCTAVIAN, and we continue to make good progress with a number of other subinsurers.

Speaker Change: 47% growth of naglozyme year-over-year was driven by the timing of a few large orders and 18% growth in palizinc was driven by new patient starts in the quarter primarily in the US

Speaker Change: Alexandra has outlined our updated strategy to reduce the resourcing of Octavian and a focus on three existing commercial markets, where the drug is approved and reimbursed which are the United States, Germany and Italy. To expand on this rationale the decision was based on signs of progress on those markets, including the successful treatment with Octavian at multiple U S hemophilia treatment centers. We believe as more and more centers gain patient treatment experience in the U S patient flow will become more straightforward and.

Kristen Hubbard: And 18% growth in palizink was driven by new patient starts in the quarter, primarily in the U.S. Now turning to our Octavian results, we were encouraged that patients were treated in both the U.S. and Italy, driving approximately $7 million of revenue in the quarter. Alexander has outlined our updated strategy to reduce the resourcing of Roctavian and focus on three existing commercial markets where the drug is approved and reimbursed, which are the United States, Germany, and Italy.

Speaker Change: Now turning to our Octavian results, we were encouraged that patients were treated in both the U.S. and Italy, driving approximately $7 million of revenue in the quarter.

Cristin Hubbard: To expand on this rationale, the decision was brd on signs of progress in those markets, including the successful treatment with ROCTAVIAN at multiple U.S. hemophilia treatment centers. We believe as more and more centers gain patient treatment experience in the U.S., patient flow will become more straightforward. In Germany, we're also making progress. At our last update, we shared that subinsurers had yet to green light reimbursement for ROCTAVIAN treatment despite there being an improved government price. And I'm pleased to share that we have finalized an agreement with one subinsurer, opening access for some launching treatment with ROCTAVIAN, and we continue to make good progress with a number of other subinsurers.

Speaker Change: To expand on this rationale the decision was based on signs of progress on those markets, including the successful treatment with Octavian at multiple U S hemophilia treatment centers. We believe as more and more centers gain patient treatment experience in the U S patient flow will become more straightforward and.

Speaker Change: Alexander has outlined our updated strategy to reduce the resourcing of Roktavien and to focus on three existing commercial markets where the drug is approved and reimbursed, which are the United States, Germany, and Italy.

Operator: Our next question comes from Jessica Fai with J.P. Morgan. Please go ahead.

Speaker Change: We believe as more and more centers gain patient treatment experience in the U S patient flow will become more straightforward and.

Kristen Hubbard: To expand on this rationale, the decision was based on signs of progress in those markets, including successful treatment with Roctavian at multiple U.S. hemophilia treatment centers. We believe as more and more centers gain patient treatment experience in the U.S., patient flow will become more straightforward, and Germany is also making progress. At our last update, we shared that sub-insurers had yet to green-light reimbursement for Roktavian treatment despite there being an approved government price.

Ellie Merle: Hey, guys. Good afternoon. Thanks for taking my question. How are you thinking about the evolution of the competitive landscape in achondroplasia when we think about TransCon CNP and infigratinib potentially on the horizon? And maybe as a follow-up, would you ever consider evaluating VOXZOGO in combination with growth hormone in achondroplasia? Thank you.

Alexander Hardy: To expand on this rationale, the decision was based on signs of progress in those markets, including the successful treatment with Roctavian at multiple U.S. hemophilia treatment centers.

Speaker Change: In Germany, we're also making progress at our last update we shared that southern sharers have yet to green light reimbursement from Octavian treatment. Despite there being an approved government price and. And I'm pleased to share that we have finalized an agreement with one seven share opening access for some wanting came out with Octavian and we continue to make good progress with a number of other southern chairs.

Cristin Hubbard: At our last update, we shared that subinsurers had yet to green light reimbursement for ROCTAVIAN treatment despite there being an improved government price. And I'm pleased to share that we have finalized an agreement with one subinsurer, opening access for some launching treatment with ROCTAVIAN, and we continue to make good progress with a number of other subinsurers.

Alexander Hardy: We believe as more and more centers gain patient treatment experience in the U.S., patient flow will become more straightforward.

Speaker Change: And I'm pleased to share that we have finalized an agreement with one seven share opening access for some wanting came out with Octavian and we continue to make good progress with a number of other southern chairs.

Alexander Hardy: In Germany we're also making progress. At our last update we shared that sub-insurers had yet to green light reimbursement for Roktavian treatment, despite there being an approved government price.

Kristen Hubbard: And I'm pleased to share that we have finalized an agreement with one sub-insurer, opening access for some wanting treatment with Rectable, and we continue to make good progress with a number of other sub-insurers. Now in Italy, we're seeing encouraging signs of patient demand, and two patients received treatment there in the second quarter, advancing access and experience. As we shared in our press release today, we are prioritizing these three commercial markets with a focus on facilitating patient access at the site level.

Cristin Hubbard: Now in Italy, we're seeing encouraging signs of patient demand and two patients received treatment there in the second quarter, advancing access and experience. As we shared in our press release today, we are prioritizing these three commercial markets with a focus on facilitating patient access at the site level. With this encouraging progress and the strategic focus on viable commercial markets, enabling a significant reduction in operating expenses related to ROCTAVIAN, we believe there's a path forward to treat more patients with severe hemophilia A, while also contributing to the Company's profitability. We believe that adjusting ROCTAVIAN expenses to approximately $60 million, beginning in 2025, is the right level to be able to make progress in our three prioritized markets. So, in conclusion, strong commercial results in the quarter and expectations for the remainder of 2024 led to today's increase of the full year guidance. I'm excited by the prospects ahead and the opportunity to drive profound patient impact with our innovative therapies. So, thank you for your attention, and I'll now turn the call over to Hank to provide an R&D update. Hank?

Alexander Hardy: Thanks very much for your question, Jessica. So overall, we feel very confident about our competitive profile in achondroplasia and in the subsequent indications. As you know, CNP pathway is amenable to all the five different indications that we're now pursuing after achondroplasia. And even with achondroplasia, we think with the movement in achondroplasia to treat the youngest infants, the 0 to 5 population, which is a very, very important part of the growth, but also very important in terms of optimal patient outcomes. The earlier you start treatment, the greatest possible impact on the child's health outcomes as well as growth. So we think that's a really compelling position in achondroplasia. The lead we have there, the safety profile, the durable efficacy, we feel very, very good about achondroplasia. And then obviously in the subsequent five indications where we think we're going to be very competitive.

Alexander Hardy: And I'm pleased to share that we have finalized an agreement with one sub-insurer, opening access for some wanting treatment with Ructavian. And we continue to make good progress with a number of other sub-insurers.

Speaker Change: As we shared in our press release today, we are prioritizing these three commercial markets with a focus on facilitating patient access at the site level with this encouraging progress on our strategic focus on viable commercial markets, enabling a significant reduction in operating expenses related about TVN. We believe are the path forward to treat more patients with severe hemophilia a. While also contributing to the company's profitability. We believe that adjusting Octavian expenses to approximately $60 million beginning in 2025 is the right level to be able to make progress in our three prioritized markets. So in conclusion strong commercial results in the quarter and expectations for the remainder of 2024 led to today's increase in our full year guidance. Provided by the prospects ahead, and the opportunity to drive profound patient impact with our innovative therapies. So thank you for your attention and I'll now turn the call over to Hank to provide an R&D update.

Alexander Hardy: Now in Italy, we're seeing encouraging signs of patient demand, and two patients received treatment there in the second quarter, advancing access and experience.

Cristin Hubbard: With this encouraging progress and the strategic focus on viable commercial markets, enabling a significant reduction in operating expenses related to ROCTAVIAN, we believe there's a path forward to treat more patients with severe hemophilia A, while also contributing to the company's profitability. We believe that adjusting ROCTAVIAN expenses to approximately $60 million, beginning in 2025, is the right level to be able to make progress in our three prioritized markets.

Alexander Hardy: As we shared in our press release today, we are prioritizing these three commercial markets with a focus on facilitating patient access at the site level.

Kristen Hubbard: With this encouraging progress and the strategic focus on viable commercial markets, enabling a significant reduction in operating expenses related to Octavian, we believe there is a path forward to treat more patients with severe hemophilia A while also contributing to the company's profitability. We believe that adjusting Roktavian expenses to approximately $60 million beginning in 2025 is the right level to be able to make progress in our three prioritized markets. So, in conclusion, strong commercial results in the quarter and expectations for the remainder of 2024 led to today's increase in the full-year guide.

Speaker Change: While also contributing to the company's profitability. We believe that adjusting Octavian expenses to approximately $60 million beginning in 2025 is the right level to be able to make progress in our three prioritized markets. So in conclusion strong commercial results in the quarter and expectations for the remainder of 2024 led to today's increase in our full year guidance. Provided by the prospects ahead, and the opportunity to drive profound patient impact with our innovative therapies. So thank you for your attention and I'll now turn the call over to Hank to provide an R&D update.

Alexander Hardy: With this encouraging progress and the strategic focus on viable commercial markets, enabling a significant reduction in operating expenses related to Roktavion, we believe there's a path forward to treat more patients with severe hemophilia A while also contributing to the company's profitability.

Speaker Change: We believe that adjusting Octavian expenses to approximately $60 million beginning in 2025 is the right level to be able to make progress in our three prioritized markets. So in conclusion strong commercial results in the quarter and expectations for the remainder of 2024 led to today's increase in our full year guidance. Provided by the prospects ahead, and the opportunity to drive profound patient impact with our innovative therapies. So thank you for your attention and I'll now turn the call over to Hank to provide an R&D update.

Cristin Hubbard: So, in conclusion, strong commercial results in the quarter and expectations for the remainder of 2024 led to today's increase of the full year guidance. I'm excited by the prospects ahead and the opportunity to drive profound patient impact with our innovative therapies. So, thank you for your attention, and I'll now turn the call over to Hank to provide an R&D update. Hank?

Speaker Change: So in conclusion strong commercial results in the quarter and expectations for the remainder of 2024 led to today's increase in our full year guidance. Provided by the prospects ahead, and the opportunity to drive profound patient impact with our innovative therapies. So thank you for your attention and I'll now turn the call over to Hank to provide an R&D update.

Alexander Hardy: We believe that adjusting our Octavian expenses to approximately $60 million beginning in 2025 is the right level to be able to make progress in our three prioritized markets.

Speaker Change: Provided by the prospects ahead, and the opportunity to drive profound patient impact with our innovative therapies. So thank you for your attention and I'll now turn the call over to Hank to provide an R&D update.

Alexander Hardy: So in conclusion, strong commercial results in the quarter and expectations for the remainder of 2024 led to today's increase of a full year guidance. I'm excited by the prospects ahead and the opportunity to drive profound patient impact with our innovative therapies.

Kristen Hubbard: I'm excited by the prospects ahead and the opportunity to drive profound patient impact with our innovative barriers. So, thank you for your attention, and I'll now turn the call over to Hank to provide an R&D update.

Hank Fuchs: Thank you, Cristin, and good afternoon, everyone. The R&D team has also been working closely with our colleagues on corporate strategy and operational efficiencies, aligning a more focused approach to R&D has resulted in great efficiencies across our VOXZOGO development programs in new indications as well as the pipeline assets we prioritized for advancement during the first quarter. On our leadership expansion plans across multiple new growth-related conditions, we were very pleased to gain alignment with global health authorities on development plans for multiple new indications, including idiopathic short stature, Turner Syndrome, Noonan Syndrome and SHOX deficiency with enrollment expected to begin in clinical trials supporting these development programs in the second half of this year. Our pivotal registrational study in hypochondroplasia is also progressing well. We are targeting approval for hypochondroplasia in 2027, subject to enrollment rate and data results. Like Alexander and Cristin, I'm excited about VOXZOGO and the potential it has to treat thousands of children with a number of growth conditions as more evidence is generated.

Hank: R&D team has also been working closely with our colleagues on corporate strategy and operational efficiencies aligning a more focused approach to R&D has resulted in great efficiencies across our box. So those development programs in new indications as well as the pipeline assets, we prioritized for advancements during the first quarter. On our leadership expansion plans across multiple new growth related conditions, we were very pleased to gain alignment with global health authorities on development plans for multiple new indications, including idiopathic short stature Turner syndrome, newness around Rome, and Chuck's deficiency with enrollment expected to begin in clinical trials. Supporting these development programs in the second half of this year, our pivotal Registrational study in Hypochondria pleasure is also progressing well we are targeting approval for hyper contemplated in 2027 subject to enrollment rate and data results. Like Alexander and Kristen I am excited about <unk> and the potential it has to treat thousands of children with a number of growth conditions as more evidence is generated.

Alexander Hardy: With regard to your second question, I will hand it over to Hank to answer the possibility of a combination approach.

Alexander Hardy: So thank you for your attention and I'll now turn the call over to Hank to provide an R&D update. Hank?

Hank Fuchs: Thank you, Kristen, and good afternoon, everyone. The R&D team has also been working closely with our colleagues on corporate strategy and operational efficiency. Aligning a more focused approach to R&D has resulted in great efficiencies across our Voxelgo development programs in new indications, as well as the pipeline assets we prioritized for advancement during the first quarter. On our leadership expansion plans across multiple new growth-related conditions, we were very pleased to gain alignment with global health authorities on development plans for multiple new indications, including idiopathic short stature, Turner syndrome, Noonan syndrome Our pivotal registrational study in hypochondroplasia is also progressing well.

Hank Fuchs: Thank you, Kristen, and good afternoon, everyone. The R&D team has also been working closely with our colleagues on corporate strategy and operational efficiencies.

Brian Mueller: Yeah. Thanks, Alexander and Jessica. VOXZOGO restores growth to about 90% of the average stature population. And if we have a strategy to even further optimize efficacy, it would be more focused on 333 by virtue of offering the dual well, the three advantages would be potentially for superior efficacy, potentially for superior convenience. And as part of that superior convenience, the ability to use one drug, not two drugs. So we think the CNP franchise ultimately can do it all.

Hank: On our leadership expansion plans across multiple new growth related conditions, we were very pleased to gain alignment with global health authorities on development plans for multiple new indications, including idiopathic short stature Turner syndrome, newness around Rome, and Chuck's deficiency with enrollment expected to begin in clinical trials. Supporting these development programs in the second half of this year, our pivotal Registrational study in Hypochondria pleasure is also progressing well we are targeting approval for hyper contemplated in 2027 subject to enrollment rate and data results. Like Alexander and Kristen I am excited about <unk> and the potential it has to treat thousands of children with a number of growth conditions as more evidence is generated.

Hank Fuchs: On our leadership expansion plans across multiple new growth-related conditions, we were very pleased to gain alignment with global health authorities on development plans for multiple new indications, including idiopathic short stature, Turner Syndrome, Noonan Syndrome and SHOX deficiency with enrollment expected to begin in clinical trials supporting these development programs in the second half of this year. Our pivotal registrational study in hypochondroplasia is also progressing well. We are targeting approval for hypochondroplasia in 2027, subject to enrollment rate and data results. Like Alexander and Cristin, I'm excited about VOXZOGO and the potential it has to treat thousands of children with a number of growth conditions as more evidence is generated.

Hank Fuchs: Aligning a more focused approach to R&D has resulted in great efficiencies across our Voxelgo development programs in new indications, as well as the pipeline assets we prioritized for advancement during the first quarter.

Hank: Supporting these development programs in the second half of this year, our pivotal Registrational study in Hypochondria pleasure is also progressing well we are targeting approval for hyper contemplated in 2027 subject to enrollment rate and data results. Like Alexander and Kristen I am excited about <unk> and the potential it has to treat thousands of children with a number of growth conditions as more evidence is generated.

Hank Fuchs: Our pivotal registrational study in hypochondroplasia is also progressing well. We are targeting approval for hypochondroplasia in 2027, subject to enrollment rate and data results. Like Alexander and Cristin, I'm excited about VOXZOGO and the potential it has to treat thousands of children with a number of growth conditions as more evidence is generated.

Hank Fuchs: including idiopathic short stature, Turner syndrome, Noonan syndrome, and Shocks deficiency with enrollment expected to begin in clinical trials supporting these development programs in the second half of this year.

Speaker Change: Like Alexander and Kristen I am excited about <unk> and the potential it has to treat thousands of children with a number of growth conditions as more evidence is generated.

Hank Fuchs: Our pivotal registrational study in hypochondroplasia is also progressing well. We are targeting approval for hypochondroplasia in 2027, subject to enrollment rate and data results.

Ellie Merle: Thank you.

Operator: Our next question comes from Paul Matisse with Stifel. Please go ahead.

Hank Fuchs: During the quarter, at the Pediatric Endocrine Society, we were pleased to see new data from Dr. Andrew Dauber’s study in idiopathic short stature and Noonan Syndrome. For the eight children who completed 12 months of treatment, mean AGV increased from a baseline of 3.7 centimeter per year to 8.5 centimeters per year and a mean height standard deviation change from minus 3.6 standard deviations to minus 2.9 standard deviations. We are pleased to see continued evidence of CNP as a master regulator of bone growth in these new conditions where growth hormone has not been effective providing durable growth. Also presented at the quarter--in this quarter at the International Conference on Children's Bone Health, we are pleased to see new data from an investigator-led analysis of the Phase II 111-205 study, which demonstrated that children who received VOXZOGO had significant increases in bone length and metacarpal cortical area after approximately five years of therapy.

Hank Fuchs: We are targeting approval for hypochondroplasia in 2027, subject to enrollment and data results. Like Alexander and Kristen, I'm excited about Voxego and the potential it has to treat thousands of children with a number of growth conditions as more evidence is generated. During the quarter at the Pediatric Endocrine Society, we were pleased to see new data from Dr. Andrew Dauber's study on idiopathic short stature and Noonan syndrome. For the eight children who completed 12 months of treatment, mean AGV increased from a baseline of 3.7 centimeters per year to 8.5 centimeters per year, and a mean height standard deviation changed from minus 3.6 standard deviations to minus We are pleased to see continued evidence of CNP as a master regulator of bone growth in these new conditions where growth hormone has not been effective in providing durable growth.

Brian Mueller: Great. Thanks so much for taking my question. As we think about the next leg of growth for VOXZOGO, I was curious, in the untreated population, what percent are treated by a specialist today? And I say this because anecdotally, we've been hearing from some of the key opinion leaders that the incremental patient adds are coming from primary care referrals and that the next leg of growth here might be driven by diagnosis. So maybe you can give some context there and just speak to from a marketing effort perspective, what's the kind of incremental effort for the next leg of the next 500 patients versus the last 500? Thanks so much.

Speaker Change: Like Alexander and Kristen, I'm excited about Voxego and the potential it has to treat thousands of children with a number of growth conditions as more evidence is generated.

Speaker Change: During the quarter at the Pediatric Endocrine Society, we were pleased to see new data from Dr. Andrew Dauber's study in idiopathic short stature and Noonan syndrome.

Speaker Change: And I mean height standard deviation change from minus three six standard deviations to minus two nine standard deviation. We were pleased to see continued evidence of CMP is a master regulator of bone growth and these new conditions, where growth hormone has not been effective providing durable growth. Also presented at the quarter and this quarter at the International Conference on children's Bone Health. We were pleased to see new data from an investigator led analysis of the phase III 111, 205 study, which demonstrated that children, who received <unk> had significant increases inbound links and metacarpal critical air. After approximately five years of therapy.

Speaker Change: For the 8 children who completed 12 months of treatment, mean AGV increased from a baseline of 3.7 cm per year to 8.5 cm per year and a mean height standard deviation change from minus 3.6 standard deviations to minus 2.9 standard deviations.

Hank Fuchs: We are pleased to see continued evidence of CNP as a master regulator of bone growth in these new conditions where growth hormone has not been effective providing durable growth. Also presented at the quarter--in this quarter at the International Conference on Children's Bone Health, we are pleased to see new data from an investigator-led analysis of the Phase II 111-205 study, which demonstrated that children who received VOXZOGO had significant increases in bone length and metacarpal cortical area after approximately five years of therapy.

Speaker Change: We were pleased to see continued evidence of CMP is a master regulator of bone growth and these new conditions, where growth hormone has not been effective providing durable growth. Also presented at the quarter and this quarter at the International Conference on children's Bone Health. We were pleased to see new data from an investigator led analysis of the phase III 111, 205 study, which demonstrated that children, who received <unk> had significant increases inbound links and metacarpal critical air. After approximately five years of therapy.

Cristin Hubbard: Yeah. Thanks so much for the question, Paul. I will say I don't know the exact numbers of the actual treaters per se that you're referring to, but I will say where our efforts are focused. So in the United States, for instance, where we believe there to be a large growth opportunity before us, this is really an area where we do see more decentralized care. And so what I mean by that is that you certainly have it treated by some geneticists in some offices and, in fact, some pediatric endocrinologists as well. But what we're finding is a lot of these patients are also sitting with general pediatricians. And so what we're really focused on is ensuring that we have awareness around VOXZOGO in that general ped population and making sure that they know where a local treater, namely in your pediatric endocrinology offices, may be.

Speaker Change: Also presented at the quarter and this quarter at the International Conference on children's Bone Health. We were pleased to see new data from an investigator led analysis of the phase III 111, 205 study, which demonstrated that children, who received <unk> had significant increases inbound links and metacarpal critical air. After approximately five years of therapy.

Speaker Change: We are pleased to see continued evidence of CNP as a master regulator of bone growth in these new conditions where growth hormone has not been effective providing durable growth.

Hank Fuchs: Also presented this quarter at the International Conference on Children's Bone Health, we were pleased to see new data from an investigator-led analysis of the Phase 2, 111205 study, which demonstrated that children who received Voxogo had significant increases in bone length and metacarpal cortical area after approximately five years of therapy. This is important because it is the first clinical study to demonstrate that VoxOvo enabled bones to remain strong as they lengthened, an important consideration for the health and safety of children being treated in their formative years for growth-related conditions.

Speaker Change: Also presented in this quarter at the International Conference on Children's Bone Health

Speaker Change: After approximately five years of therapy. This is important because it is the first clinical study to demonstrate that <unk> enabled bones to remain strong as they lengthened and important consideration for the health and safety of children being treated in their firm formative years for gross related conditions.

After approximately five years of therapy.

Speaker Change: We were pleased to see new data from an investigator-led analysis of the Phase 2, 111-205 study, which demonstrated that children who received Voxogo had significant increases in bone length and metacarpal cortical area after approximately five years of therapy.

Hank Fuchs: This is important because it is the first clinical study to demonstrate that VOXZOGO-enabled bones to remain strong as they lengthened, an important consideration for the health and safety of children being treated in their formative years for growth-related conditions. Also at ICCBH, data observed in the Phase III 111-301 and 111-302 studies demonstrated that VOXZOGO has the potential to improve health-related quality of life among children with achondroplasia, particularly in aspects associated with physical functioning and outcome of significant importance for children and their families. Phase II and Phase III data also showed consistent positive effects on linear growth and improvement in proportionality in children of all ages with growth potential with follow-up conducted up to four years after the initiation of VOXZOGO treatment. We are pleased with the consistent safety and efficacy data being generated that illustrate VOXZOGO's positive impact on bone growth, proportionality and quality of life. We are moving rapidly to advance the development programs with VOXZOGO in these five new indications and are excited to bring a demonstrated safe and effective treatment option to children with growth-related conditions.

Speaker Change: This is important because it is the first clinical study to demonstrate that Vox Ogo enabled bones to remain strong as they lengthened. An important consideration for the health and safety of children being treated in their formative years for growth-related conditions.

Hank Fuchs: Also at ICCBH, data observed in the Phase III 111-301 and 111-302 studies demonstrated that VOXZOGO has the potential to improve health-related quality of life among children with achondroplasia, particularly in aspects associated with physical functioning and outcome of significant importance for children and their families. Phase II and Phase III data also showed consistent positive effects on linear growth and improvement in proportionality in children of all ages with growth potential with follow-up conducted up to four years after the initiation of VOXZOGO treatment.

Speaker Change: Also at ICC BH data observed in the Phase III 111, 301, and 111 302 studies demonstrated that <unk> has the potential to improve health related quality of life among children with achondroplasia, particularly in aspects associated with physical functioning and outcome of significant importance for children and their fans. Please. <unk> II and phase III data also showed consistent positive effects on linear growth and improvement in proportionality and children of all ages with growth potential with follow up conducted up to four years after the initiation of <unk> treatment. We are pleased with the consistent safety and efficacy data being generated that illustrate box. So those positive impact on bone growth proportionality and quality of life. We are moving rapidly to advance the development programs with <unk> in these five new indications and are excited to bring a demonstrated safe and effective treatment option to children with growth related conditions.

Cristin Hubbard: So we're really trying to build those connection points between where we know those patients might be to those who are going to be treated. Now, in ex-US, we do see much more of a centralized care system there where they're treated in centers that specialize in this area, and so in skeletal dysplasias. So, of course, we've seen faster uptake in those areas where you know where those patients are and how to get them. And importantly, while we've seen really good penetration in those countries, there's still room to grow, and that's where we're focused as well. So I'd say that our focus areas are really going to be around that referral pattern in the US, continuing our growth in those kind of highly penetrated strategic markets, and importantly, opening up in new markets where we might have lower penetration or we've not yet opened up the markets.

Hank Fuchs: Also at ICCBH, data observed in the Phase 3, 111.301, and 111.302 studies demonstrated that VOX-EGO has the potential to improve health-related quality of life among children with achondroplasia, particularly in aspects associated with physical functioning, an outcome of significant importance for children and their families. Phase II and Phase III data also showed consistent positive effects on linear growth and improvement in proportionality in children of all ages with growth potential, with follow-up conducted up to four years after the initiation of oxygotriazine.

Speaker Change: Also at ICCBH, data observed in the Phase 3, 111.301 and 111.302 studies demonstrated that Voxelgo has the potential to improve health-related quality of life among children with achondroplasia.

Speaker Change: Please. <unk> II and phase III data also showed consistent positive effects on linear growth and improvement in proportionality and children of all ages with growth potential with follow up conducted up to four years after the initiation of <unk> treatment. We are pleased with the consistent safety and efficacy data being generated that illustrate box. So those positive impact on bone growth proportionality and quality of life. We are moving rapidly to advance the development programs with <unk> in these five new indications and are excited to bring a demonstrated safe and effective treatment option to children with growth related conditions.

Speaker Change: <unk> II and phase III data also showed consistent positive effects on linear growth and improvement in proportionality and children of all ages with growth potential with follow up conducted up to four years after the initiation of <unk> treatment. We are pleased with the consistent safety and efficacy data being generated that illustrate box. So those positive impact on bone growth proportionality and quality of life. We are moving rapidly to advance the development programs with <unk> in these five new indications and are excited to bring a demonstrated safe and effective treatment option to children with growth related conditions.

Speaker Change: and others, particularly in aspects associated with physical functioning, an outcome of significant importance for children and their families.

Hank Fuchs: We are pleased with the consistent safety and efficacy data being generated that illustrate VOXZOGO's positive impact on bone growth, proportionality and quality of life. We are moving rapidly to advance the development programs with VOXZOGO in these five new indications and are excited to bring a demonstrated safe and effective treatment option to children with growth-related conditions.

Speaker Change: Phase II and Phase III data also showed consistent positive effects on linear growth and improvement in proportionality in children of all ages with growth potential, with follow-up conducted up to four years after the initiation of oxogo treatment.

Speaker Change: We are pleased with the consistent safety and efficacy data being generated that illustrate box. So those positive impact on bone growth proportionality and quality of life. We are moving rapidly to advance the development programs with <unk> in these five new indications and are excited to bring a demonstrated safe and effective treatment option to children with growth related conditions.

Cristin Hubbard: So focused in all of those areas.

Speaker Change: We are moving rapidly to advance the development programs with <unk> in these five new indications and are excited to bring a demonstrated safe and effective treatment option to children with growth related conditions.

Hank Fuchs: We are pleased with the consistent safety and efficacy data being generated that illustrate Voxego's positive impact on bone growth, proportionality, and quality of life. We are moving rapidly to advance the development programs with Voxego in these five new indications and are excited to bring a demonstrated safe and effective treatment option to children with growth-related conditions. On the three programs that we've chosen to accelerate that were announced last quarter, but BMN-351 for the treatment of Duchenne muscular dystrophy, BMN-349, a potential first oral therapeutic for the treatment of alpha-1 antitrypsin deficiency, and liver disease, and BMN-333, a long-acting formulation of CMP.

Speaker Change: We are pleased with the consistent safety and efficacy data being generated that illustrate Voxego's positive impact on bone growth, proportionality, and quality of life.

Operator: Our next question comes from Joseph Schwartz with Lerank Partners. Please go ahead.

Brian Mueller: Thank you. I'll ask about a couple of your priority pipeline programs. First, on BMN333, I was hoping you could give us some insight into the long-acting technology that's used there and also help us envision the expected time to market for BMN333. And then what are you hoping to see for BMN351 at the target dose or doses since it's moving behind several other next-gen exon skippers? It would be helpful to hear what you hope to see efficacy-wise at different dose levels. Thank you. Hi, Joe. As far as the specifics about 333 in terms of design, we really tried to leverage a lot of safety and information that's already available on linking chemistry. I'll get into that in a little bit more detail at Investor Day.

Hank Fuchs: On the three programs that we've chosen to accelerate that were announced last quarter, BMN 351 for the treatment of Duchenne muscular dystrophy; BMN 349, a potential first oral therapeutic for the treatment of alpha-1 antitrypsin deficiency, liver disease; and BMN 333, our long-acting formulation of CMP, all programs continue to advance, and I'll share a more detailed update of our progress at Investor Day. Finally, for BMN 293, our gene therapy for hypertrophic cardiomyopathy, we will discontinue development based on the commercial landscape as well as the time and resources anticipated to bring it through development and to market. We are now consistently applying a focused approach to the evaluation of our development programs to ensure that we are investing in assets that have the potential to have the greatest possible impact for patients. Thank you for your attention. I'll now turn the call over to Brian for our financial update. Brian?

Speaker Change: We are moving rapidly to advance the development programs with Voxego and these five new indications and are excited to bring a demonstrated safe and effective treatment option to children with growth-related conditions.

Speaker Change: On the three programs that we've chosen to accelerate that were announced last quarter,

Speaker Change: BMN 351 for the treatment of Duchenne muscular dystrophy.

Speaker Change: <unk> continue to advance and I will share a more detailed update of our progress at Investor day. Finally for <unk> 293, our gene therapy for hypertrophic cardiomyopathy, we will discontinue development based on the commercial landscape as well as the time and resources anticipated to bring it through development and to market. We are now consistently applying our focused approach to the evaluation of our development programs to ensure that we are investing in assets that have the potential to have the greatest possible impact for patients. Thank. Thank you for your attention I'll now turn the call over to Brian for our financial update Brian.

Speaker Change: BMN 349, a potential first oral therapeutic for the treatment of alpha 1 antitrypsin deficiency, liver disease,

Hank Fuchs: Finally, for BMN 293, our gene therapy for hypertrophic cardiomyopathy, we will discontinue development based on the commercial landscape as well as the time and resources anticipated to bring it through development and to market. We are now consistently applying a focused approach to the evaluation of our development programs to ensure that we are investing in assets that have the potential to have the greatest possible impact for patients. Thank you for your attention. I'll now turn the call over to Brian for our financial update. Brian?

Speaker Change: Finally for <unk> 293, our gene therapy for hypertrophic cardiomyopathy, we will discontinue development based on the commercial landscape as well as the time and resources anticipated to bring it through development and to market. We are now consistently applying our focused approach to the evaluation of our development programs to ensure that we are investing in assets that have the potential to have the greatest possible impact for patients. Thank. Thank you for your attention I'll now turn the call over to Brian for our financial update Brian.

Speaker Change: and BMN 333, a long-acting formulation of C&P. All programs continue to advance, and I'll share a more detailed update of our progress at Investor Day.

Hank Fuchs: All programs continue to advance, and I'll share a more detailed update of our progress at Investor Day. Finally, for BMN 293, our gene therapy for hypertrophic cardiomyopathy, we will discontinue development based on the commercial landscape, as well as the time and resources anticipated to bring it through development and to market. We are now consistently applying a focused approach to the evaluation of our development programs to ensure that we are investing in assets that have the potential to have the greatest possible impact for patients.

Speaker Change: We are now consistently applying our focused approach to the evaluation of our development programs to ensure that we are investing in assets that have the potential to have the greatest possible impact for patients. Thank. Thank you for your attention I'll now turn the call over to Brian for our financial update Brian.

Speaker Change: Finally, for BMN 293, our gene therapy for hypertrophic cardiomyopathy, we will discontinue development based on the commercial landscape, as well as the time and resources anticipated to bring it through development and to market.

Speaker Change: Thank you for your attention I'll now turn the call over to Brian for our financial update Brian.

Speaker Change: We are now consistently applying a focused approach to the evaluation of our development programs to ensure that we are investing in assets that have the potential to have the greatest possible impact for patients.

Brian Mueller: Obviously, one of the other critical aspects of the program is to ensure sustained exposure without a lot of early high exposure or dumping, as sometimes is referred to in drug delivery circles. So I'll get into a little bit more details, but really a big orientation around safety and consistency of exposure. On BMN 351, acknowledging that there are a lot of competitors, it doesn't seem like anybody is really able to get to high levels of dystrophin either by conjugating morpholino with CAGs or with transferrin receptor antibodies. So what our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre-mRNA processing. The advantage of that approach has been twofold.

Brian Mueller: Thank you, Hank. Please refer to today's press release summarizing our financial results for full details on the second quarter of 2024, including reconciliations of GAAP to non-GAAP financial measures. All second quarter 2024 results will be available in our upcoming Form 10-Q, which we expect to file later today. In the second quarter of 2024, Biomarin generated record quarterly total revenue of $712 million, representing 20% year-over-year growth and 25% on a constant currency basis, driven by continued strong demand for VOXZOGO. The enzyme therapies comprised of VIMIZIM, NAGLAZYME, ALDURAZYME, BRINEURA and PALYNZIQ contributed $482 million of net product revenues during the second quarter. Looking more closely at Q2 net product revenue, VOXZOGO revenues of $184 million represents 62% year-over-year growth. We are pleased to announce as of early June, we can now satisfy all anticipated customer demand for VOXZOGO, which was a key driver of VOXZOGO's strong Q2 performance.

Brian Mueller: Thank you for your attention. I'll now turn the call over to Brian for our financial update.

Speaker Change: Thank you for your attention. I'll now turn the call over to Brian for our financial update. Brian.

Brian Mueller: Please refer to today's press release, summarizing our financial results, for full details on the second quarter of 2021, including reconciliations of GAAP to non-GAAP financial measures. All second quarter 2024 results will be available in our upcoming Form 10-Q, which we expect to file later today.

Brian: Second quarter of 2024 results will be available in our upcoming Form 10-Q, which we expect to file later today. In the second quarter of 2024. Marin generated record quarterly total revenue of $712 million, representing 20% year over year growth and 25% on a constant currency basis, driven by continued strong demand for <unk>. The enzyme therapies comprised of Vimizim nacco zoning Aldurazyme <unk> and <unk> contributed $482 million of net product revenues during the second quarter. Looking more closely at Q2 net product revenue <unk> revenues of $184 million represents 62% year over year growth. We are pleased to announce as of early June we can now satisfy all anticipated customer demand for box yoga. Key driver of box Youll go strong Q2 performance the.

Brian Mueller: Thank you, Hank.

Brian Mueller: Please refer to today's press release summarizing our financial results for full details on the second quarter of 2024.

Brian: In the second quarter of 2024. Marin generated record quarterly total revenue of $712 million, representing 20% year over year growth and 25% on a constant currency basis, driven by continued strong demand for <unk>. The enzyme therapies comprised of Vimizim nacco zoning Aldurazyme <unk> and <unk> contributed $482 million of net product revenues during the second quarter. Looking more closely at Q2 net product revenue <unk> revenues of $184 million represents 62% year over year growth. We are pleased to announce as of early June we can now satisfy all anticipated customer demand for box yoga. Key driver of box Youll go strong Q2 performance the.

Brian: Marin generated record quarterly total revenue of $712 million, representing 20% year over year growth and 25% on a constant currency basis, driven by continued strong demand for <unk>. The enzyme therapies comprised of Vimizim nacco zoning Aldurazyme <unk> and <unk> contributed $482 million of net product revenues during the second quarter. Looking more closely at Q2 net product revenue <unk> revenues of $184 million represents 62% year over year growth. We are pleased to announce as of early June we can now satisfy all anticipated customer demand for box yoga. Key driver of box Youll go strong Q2 performance the.

Brian Mueller: including reconciliations of GAAP to non-GAAP financial measures. All second quarter 2024 results will be available in our upcoming Form 10-Q , which we expect to file later today.

Brian Mueller: In the second quarter of 2024, Biomarin generated record quarterly total revenue of $712 million, representing 20% year-over-year growth and 25% on a constant currency basis. Driven by continued strong demand for Voxelga, the enzyme therapies, comprised of vimazine, naglazine, aldurazine, brinura, and palanzic, contributed $482 million of net product revenues during the second quarter. Looking more closely at Q2 We are pleased to announce that, as of early June, we can now satisfy all anticipated customer demand for VoxOvernight, a key driver of Voxelgo's strong Q2 performance. The robust and focused efforts of our technical operations organization drove the availability of incremental global VoxOgo supply a couple of months earlier than planned. Well, our 2024 Vox D'Ogo plan.

Brian: The enzyme therapies comprised of Vimizim nacco zoning Aldurazyme <unk> and <unk> contributed $482 million of net product revenues during the second quarter. Looking more closely at Q2 net product revenue <unk> revenues of $184 million represents 62% year over year growth. We are pleased to announce as of early June we can now satisfy all anticipated customer demand for box yoga. Key driver of box Youll go strong Q2 performance the.

Brian Mueller: Looking more closely at Q2 net product revenue, VOXZOGO revenues of $184 million represents 62% year-over-year growth. We are pleased to announce as of early June, we can now satisfy all anticipated customer demand for VOXZOGO, which was a key driver of VOXZOGO's strong Q2 performance.

Brian: Looking more closely at Q2 net product revenue <unk> revenues of $184 million represents 62% year over year growth. We are pleased to announce as of early June we can now satisfy all anticipated customer demand for box yoga. Key driver of box Youll go strong Q2 performance the.

Brian Mueller: The enzyme therapies, comprised of vimazine, naglozyme, aldurazine, brinura, and palanzic, contributed $482 million of net product revenues during the second quarter.

Brian Mueller: One, in preclinical models, we can get dystrophin expression up to 40% not just in skeletal muscle, but also in high levels in diaphragm, high levels in heart. And the second is that we can do that at levels where we have confidence about the safety profile, given our experience with DMD as a population. I'll give more detailed updates on status of the program and expectations for when you're going to see data at Investor Day, but we're very encouraged by the progress that we're making with 351. Great. I'll stay tuned.

Brian Mueller: Looking more closely at Q2 Net Product Revenue, VoxOgo revenues of $184 million represent 62% year-over-year growth. We are pleased to announce as of early June , we can now satisfy all anticipated customer demand for VoxOgo.

Key driver of box Youll go strong Q2 performance the.

Brian Mueller: The robust and focused efforts of our technical operations organization drove the availability of incremental global VOXZOGO supply to occur a couple of months earlier than planned, while our 2024 VOXZOGO plans always assumed that the supply constraint would be relieved by midyear, the incremental supply, earlier than anticipated, enabled certain markets to start new patients earlier and to begin to normalize customer stock levels that were running low during the supply constraint. We believe that Q2 VOXZOGO revenues benefited from this dynamic with approximately $20 million of incremental revenue. As we plan to add hundreds of new VOXZOGO commercial patients in the second half of the year, some of these order timing dynamics could result in patient growth rates exceeding revenue growth rates in the second half of 2024. Having said this, setting aside quarter-to-quarter dynamics, we expect patient growth and revenue growth from normalized and broadly track over longer time frames.

Brian Mueller: which is a key driver of VoxOgo's strong Q2 performance.

Speaker Change: While our 2024 box yoga plans. Always assumed that the supply constraint would be relieved by mid year, the incremental supply earlier than anticipated enabled certain markets to start new patients earlier and to begin to normalize customer stock levels that we're running low during the supply constraint. We believe the Q2 <unk> revenues benefited from this dynamic with approximately $20 million of incremental revenue. As we plan to add hundreds of new box logo commercial patients in the second half of the year. Of these order timing dynamics could result in patient growth rates exceeding revenue growth rates in the second half of 2024. Having said this setting aside quarter to quarter dynamics, we expect patient growth and revenue growth and normalized and broadly track over longer timeframes.

Always assumed that the supply constraint would be relieved by mid year, the incremental supply earlier than anticipated enabled certain markets to start new patients earlier and to begin to normalize customer stock levels that we're running low during the supply constraint. We believe the Q2 <unk> revenues benefited from this dynamic with approximately $20 million of incremental revenue. As we plan to add hundreds of new box logo commercial patients in the second half of the year. Of these order timing dynamics could result in patient growth rates exceeding revenue growth rates in the second half of 2024. Having said this setting aside quarter to quarter dynamics, we expect patient growth and revenue growth and normalized and broadly track over longer timeframes.

Brian Mueller: The robust and focused efforts of our technical operations organization drove the availability of incremental global VoxOgo supply to occur a couple of months earlier than planned.

Brian Mueller: Assuming that the supply constraint would be relieved by mid-year, the incremental supply earlier than anticipated enabled certain markets to start new patients earlier and to begin to normalize customer stock levels that were running low during the supply constraint. We believe that Q2 VoxOgo revenues benefited from this dynamic with approximately $20 million of incremental. We plan to add hundreds of new VoxOvo commercial patients in the second half of the year.

Brian Mueller: Well, our 2024 Vox D'Ogo plans.

Brian Mueller: Always assumed that the supply constraint would be relieved by mid-year, the incremental supply, earlier than anticipated, enabled certain markets to start new patients earlier and to begin to normalize customer stock levels that were running low during the supply constraint.

Operator: Our next question comes from Akash Tewari with Jefferies. Please go ahead.

Speaker Change: We believe the Q2 <unk> revenues benefited from this dynamic with approximately $20 million of incremental revenue. As we plan to add hundreds of new box logo commercial patients in the second half of the year. Of these order timing dynamics could result in patient growth rates exceeding revenue growth rates in the second half of 2024. Having said this setting aside quarter to quarter dynamics, we expect patient growth and revenue growth and normalized and broadly track over longer timeframes.

Ellie Merle: Hi. This is Phoebe on for Akash. Also on BMN 351 for DMD, how should we think about the translation from animal to human models when it comes to dystrophin expression? In mice models, you showed normal dystrophin levels as high as 98%. I guess how well does that data translate to humans percent normal dystrophin? And also for BARA success, what would you need in order to move forward? Thank you.

Brian Mueller: As we plan to add hundreds of new VOXZOGO commercial patients in the second half of the year, some of these order timing dynamics could result in patient growth rates exceeding revenue growth rates in the second half of 2024. Having said this, setting aside quarter-to-quarter dynamics, we expect patient growth and revenue growth from normalized and broadly track over longer time frames.

Speaker Change: As we plan to add hundreds of new box logo commercial patients in the second half of the year. Of these order timing dynamics could result in patient growth rates exceeding revenue growth rates in the second half of 2024. Having said this setting aside quarter to quarter dynamics, we expect patient growth and revenue growth and normalized and broadly track over longer timeframes.

Speaker Change: Of these order timing dynamics could result in patient growth rates exceeding revenue growth rates in the second half of 2024. Having said this setting aside quarter to quarter dynamics, we expect patient growth and revenue growth and normalized and broadly track over longer timeframes.

Brian Mueller: We believe that Q2 VoxOgo revenues benefited from this dynamic with approximately $20 million of incremental revenue.

Speaker Change: Having said this setting aside quarter to quarter dynamics, we expect patient growth and revenue growth and normalized and broadly track over longer timeframes.

Brian Mueller: We plan to add hundreds of new VoxOvo commercial patients in the second half of the year. Some of these order timing dynamics could result in patient growth rates exceeding revenue growth rates in the second half of 2024.

Brian Mueller: Some of these order timing dynamics could result in patient growth rates exceeding revenue growth rates in the second half of 2020. Having said this, setting aside quarter-to-quarter dynamics, we expect patient growth and revenue growth to normalize and broadly track over longer periods. Our stable and growing portfolio of enzyme therapies continued to drive strong performance with 15% year-over-year growth in the quarter, albeit benefited from approximately $20 million of large government orders for select markets that were previously expected in Q3.

Brian Mueller: Yeah. Well, I think one of the challenges of translation is that there hasn't been a lot in humans that's really moved the needle in terms of dystrophin expression. So a little bit of that question is TBD. What we talked about at last year's R&D Day was based on our animal model work. If we can achieve the tissue concentrations of 351 that we achieve for DMD as a person, we should be in a very much higher level of dystrophin expression. And partly, we base that on a relatively unique animal model, which carries the human equivalent of the gene that is to be skipped. And so we have a very fulsome assessment in vivo of skipping potential. And we've got a lot of safety data both from rodents, non-human primates, and humans on our class of compounds.

Brian Mueller: Our stable and growing portfolio of enzyme therapies continued to drive strong performance with 15% year-over-year growth in the quarter, albeit benefited from approximately $20 million of large government orders for select markets that were previously expected in Q3. While Q2 revenues were positively impacted by the timing of those large orders, we continue to observe solid commercial patient growth in these brands that will drive sustainable revenue growth over time. For the full year 2024, the strong performance of VOXZOGO and the enzyme therapies are allowing us to raise full year 2024 revenue guidance to between $2.75 billion and $2.825 billion, representing approximately 15% year-over-year growth at the midpoint. From an operating expense standpoint, the R&D expenses in the second quarter were $184 million, up $7 million year-over-year, primarily due to the support of the VOXZOGO indication expansion development, as we work to accelerate the full VOXZOGO opportunity.

Brian Mueller: Having said this, setting aside corridor-to-corridor dynamics, we expect patient growth and revenue growth to normalize and broadly track over longer time frames.

Speaker Change: While Q2 revenues were positively impacted by the timing of those large orders we continue to observe solid commercial patient growth in these brands that will drive sustainable revenue growth over time. Full year 2020 for the strong performance of box Cisco and the enzyme therapies are allowing us to raise full year 2020 for revenue guidance to between $2 $75 billion. And 282 5 billion. Representing approximately 15% year over year growth at the midpoint. From an operating expense standpoint, GAAP R&D expenses in the second quarter were $184 million up $7 million year over year, primarily due to the support of the <unk> indication expansion development as we work to accelerate the full but still grow opportunity.

Brian Mueller: While Q2 revenues were positively impacted by the timing of those large orders, we continue to observe solid commercial patient growth in these brands that will drive sustainable revenue growth over time. For the full year 2024, the strong performance of VoxOgo and the enzyme therapies are allowing us to raise full year 2024 revenue guidance to between $2.75 billion and $2.825 billion, representing approximately 15% year-over-year growth at the, From an operating expense standpoint, the FR&D expenses in the second quarter were $184 million, up $7 million year over year, primarily due to the support of the VOTSOGO Indication Expansion Development as we work to accelerate the full VOTSOGO opportunity.

Speaker Change: Full year 2020 for the strong performance of box Cisco and the enzyme therapies are allowing us to raise full year 2020 for revenue guidance to between $2 $75 billion. And 282 5 billion. Representing approximately 15% year over year growth at the midpoint. From an operating expense standpoint, GAAP R&D expenses in the second quarter were $184 million up $7 million year over year, primarily due to the support of the <unk> indication expansion development as we work to accelerate the full but still grow opportunity.

Brian Mueller: For the full year 2024, the strong performance of VOXZOGO and the enzyme therapies are allowing us to raise full year 2024 revenue guidance to between $2.75 billion and $2.825 billion, representing approximately 15% year-over-year growth at the midpoint. From an operating expense standpoint, the R&D expenses in the second quarter were $184 million, up $7 million year-over-year, primarily due to the support of the VOXZOGO indication expansion development, as we work to accelerate the full VOXZOGO opportunity.

And 282 5 billion. Representing approximately 15% year over year growth at the midpoint. From an operating expense standpoint, GAAP R&D expenses in the second quarter were $184 million up $7 million year over year, primarily due to the support of the <unk> indication expansion development as we work to accelerate the full but still grow opportunity.

Brian Mueller: For the full year 2024, the strong performance of VoxOvo and the enzyme therapies are allowing us to raise full year 2024 revenue guidance to between $2.75 billion

Speaker Change: Representing approximately 15% year over year growth at the midpoint. From an operating expense standpoint, GAAP R&D expenses in the second quarter were $184 million up $7 million year over year, primarily due to the support of the <unk> indication expansion development as we work to accelerate the full but still grow opportunity.

Brian Mueller: From an operating expense standpoint, the R&D expenses in the second quarter were $184 million, up $7 million year-over-year, primarily due to the support of the VOXZOGO indication expansion development, as we work to accelerate the full VOXZOGO opportunity.

Speaker Change: From an operating expense standpoint, GAAP R&D expenses in the second quarter were $184 million up $7 million year over year, primarily due to the support of the <unk> indication expansion development as we work to accelerate the full but still grow opportunity.

Brian Mueller: and $2.825 billion, representing approximately 15% year-over-year growth at the midpoint.

Brian Mueller: And so we're optimistic based on that that we'll be able to see much more meaningful levels of dystrophin expression. And of course, it's really the near full-length dystrophin that motorizes the muscle, if you will, that will enable much greater function of the muscle. And so far, that hasn't been achieved. And that's what we're looking to achieve with BMN 351.

Brian Mueller: From an operating expense standpoint, the FR&D expenses in the second quarter were $184 million, up $7 million year-over-year, primarily due to the support of the VOXOGO Indication Expansion Development as we work to accelerate the full VOXOGO opportunity.

Brian Mueller: SG&A expenses in the second quarter were $263 million, up $57 million year-over-year, primarily driven mostly by $39 million of restructuring expenses incurred during Q2 as well as continued support of the global VOXZOGO market expansion. Restructuring expenses mostly included severance and wind-down costs associated with the discontinued development programs announced last quarter. Biomarin is executing very well in 2024, earning a non-GAAP operating margin of 31% for the quarter, which we believe is a high watermark for 2024. For example, normalizing Q2 operating margin for just the approximately $40 million of order timing, non-GAAP CAP operating margin in Q2 would have been approximately 28%. Q2 GAAP diluted earnings per share was $0.55, an increase of 90% over Q2 last year. And Q2 non-GAAP diluted earnings per share was $0.96, representing growth of 78% compared to Q2 2023. Our bottom-line results for Q2 benefited from the strong revenue execution driven by underlying patient demand and the order timing just discussed as well as Q2 operating expenses that are reflective of our disciplined resource allocation. While we will discuss details of our cost transformation program at Investor Day, we were able to realize some cost savings in Q2 earlier than anticipated, including lower R&D related to the discontinued programs announced in April.

Speaker Change: $39 million of restructuring expenses incurred during Q2 as well as continued support of the global box Sogo market expansion. Restructuring expenses, mostly included severance and wind down costs associated with the discontinued development programs announced last quarter. Biomarin is executing very well in 2020 for earning a non-GAAP operating margin of 31% for the quarter, which we believe is a high watermark for 2024 core. For example, normalizing Q2 operating margin for just the approximately $40 million of order timing. non-GAAP operating margin in Q2 would have been approximately 28%. Q2, GAAP diluted earnings per share was 55. An increase of 90% over Q2 last year and Q2 non-GAAP diluted earnings per share was <unk> 96, representing growth of 78% compared to Q2 2023. Our bottom line results for Q2 benefited from the strong revenue execution, driven by underlying patient demand and the order timing just discussed as well as Q2 operating expenses that are reflective of our disciplined resource allocation. While we will discuss details of our cost transformation program at Investor Day, we were able to realize some cost savings in Q2 earlier than anticipated, including lower R&D related to the discontinued programs announced in April.

Brian Mueller: The FSG&A expenses in the second quarter were $263 million, up $57 million year-over-year, primarily driven by $39 million of restructuring expenses incurred during Q2, as well as continued support of the global VoxOgo market expansion. These expenses mostly included severance and wind-down costs associated with the discontinued development programs announced last week.

Brian Mueller: The FSG&A expenses in the second quarter were $263 million, up $57 million year over year, primarily driven mostly

Operator: Our next question comes from Chris Raymond with Piper Sandler. Please go ahead.

Speaker Change: Restructuring expenses, mostly included severance and wind down costs associated with the discontinued development programs announced last quarter. Biomarin is executing very well in 2020 for earning a non-GAAP operating margin of 31% for the quarter, which we believe is a high watermark for 2024 core. For example, normalizing Q2 operating margin for just the approximately $40 million of order timing. non-GAAP operating margin in Q2 would have been approximately 28%. Q2, GAAP diluted earnings per share was 55. An increase of 90% over Q2 last year and Q2 non-GAAP diluted earnings per share was <unk> 96, representing growth of 78% compared to Q2 2023. Our bottom line results for Q2 benefited from the strong revenue execution, driven by underlying patient demand and the order timing just discussed as well as Q2 operating expenses that are reflective of our disciplined resource allocation. While we will discuss details of our cost transformation program at Investor Day, we were able to realize some cost savings in Q2 earlier than anticipated, including lower R&D related to the discontinued programs announced in April.

Hank Fuchs: Yep. Thanks. Just maybe two questions on VOXZOGO and hypochondroplasia. Just Hank, just listening to your comments on how hypochondroplasia is diagnosed maybe later in life, just noticing that the pivotal study includes patients aged 3 to 18, should we expect maybe that you won't have to go back for earlier age in the label once approved, or is the diagnostic journey maybe different among hypo and achondroplasia? And then also maybe another sort of pipeline question on 349, just noting that you had a competitor discontinue their oral AAT corrector program. I'm just wondering if you could call attention any points of differentiation between 349 and that molecule and just walk us through what gives you confidence here comparatively. Thank you.

Brian Mueller: $39 million of restructuring expenses incurred during Q2, as well as continued support of the global VoxOgo market expansion.

Brian Mueller: Biomarin is executing very well in 2024, earning a non-GAAP operating margin of 31% for the quarter, which we believe is a high watermark for 2024. For example, normalizing Q2 operating margin for just the approximately $40 million of order timing, non-GAAP CAP operating margin in Q2 would have been approximately 28%. Q2 GAAP diluted earnings per share was $0.55, an increase of 90% over Q2 last year. And Q2 non-GAAP diluted earnings per share was $0.96, representing growth of 78% compared to Q2 2023.

Speaker Change: Biomarin is executing very well in 2020 for earning a non-GAAP operating margin of 31% for the quarter, which we believe is a high watermark for 2024 core. For example, normalizing Q2 operating margin for just the approximately $40 million of order timing. non-GAAP operating margin in Q2 would have been approximately 28%. Q2, GAAP diluted earnings per share was 55. An increase of 90% over Q2 last year and Q2 non-GAAP diluted earnings per share was <unk> 96, representing growth of 78% compared to Q2 2023. Our bottom line results for Q2 benefited from the strong revenue execution, driven by underlying patient demand and the order timing just discussed as well as Q2 operating expenses that are reflective of our disciplined resource allocation. While we will discuss details of our cost transformation program at Investor Day, we were able to realize some cost savings in Q2 earlier than anticipated, including lower R&D related to the discontinued programs announced in April.

Brian Mueller: Restructuring expenses mostly included severance and wind-down costs associated with the discontinued development programs announced last quarter.

Brian Mueller: Biomarin is executing very well in 2024, earning a non-gap operating margin of 31% for the quarter, which we believe is a high watermark for 2024. For example, normalizing Q2 operating margin for just the approximately $40 million of order timing, non-GAAP operating margin in Q2 would have been approximately 28%. Q2 Gap Diluted Earnings Per Share was $0.55, an increase of 90% over Q2 last

Speaker Change: For example, normalizing Q2 operating margin for just the approximately $40 million of order timing. non-GAAP operating margin in Q2 would have been approximately 28%. Q2, GAAP diluted earnings per share was 55. An increase of 90% over Q2 last year and Q2 non-GAAP diluted earnings per share was <unk> 96, representing growth of 78% compared to Q2 2023. Our bottom line results for Q2 benefited from the strong revenue execution, driven by underlying patient demand and the order timing just discussed as well as Q2 operating expenses that are reflective of our disciplined resource allocation. While we will discuss details of our cost transformation program at Investor Day, we were able to realize some cost savings in Q2 earlier than anticipated, including lower R&D related to the discontinued programs announced in April.

Speaker Change: Biomarin is executing very well in 2024, earning a non-gap operating margin of 31% for the quarter, which we believe is a high watermark for 2024. For example, normalizing Q2 operating margin for just the approximately $40 million of order timing

Speaker Change: non-GAAP operating margin in Q2 would have been approximately 28%. Q2, GAAP diluted earnings per share was 55. An increase of 90% over Q2 last year and Q2 non-GAAP diluted earnings per share was <unk> 96, representing growth of 78% compared to Q2 2023. Our bottom line results for Q2 benefited from the strong revenue execution, driven by underlying patient demand and the order timing just discussed as well as Q2 operating expenses that are reflective of our disciplined resource allocation. While we will discuss details of our cost transformation program at Investor Day, we were able to realize some cost savings in Q2 earlier than anticipated, including lower R&D related to the discontinued programs announced in April.

Speaker Change: Q2, GAAP diluted earnings per share was 55. An increase of 90% over Q2 last year and Q2 non-GAAP diluted earnings per share was <unk> 96, representing growth of 78% compared to Q2 2023. Our bottom line results for Q2 benefited from the strong revenue execution, driven by underlying patient demand and the order timing just discussed as well as Q2 operating expenses that are reflective of our disciplined resource allocation. While we will discuss details of our cost transformation program at Investor Day, we were able to realize some cost savings in Q2 earlier than anticipated, including lower R&D related to the discontinued programs announced in April.

Speaker Change: An increase of 90% over Q2 last year and Q2 non-GAAP diluted earnings per share was <unk> 96, representing growth of 78% compared to Q2 2023. Our bottom line results for Q2 benefited from the strong revenue execution, driven by underlying patient demand and the order timing just discussed as well as Q2 operating expenses that are reflective of our disciplined resource allocation. While we will discuss details of our cost transformation program at Investor Day, we were able to realize some cost savings in Q2 earlier than anticipated, including lower R&D related to the discontinued programs announced in April.

Speaker Change: non-GAAP operating margin in Q2 would have been approximately 28%.

Brian Mueller: Our bottom-line results for Q2 benefited from the strong revenue execution driven by underlying patient demand and the order timing just discussed as well as Q2 operating expenses that are reflective of our disciplined resource allocation. While we will discuss details of our cost transformation program at Investor Day, we were able to realize some cost savings in Q2 earlier than anticipated, including lower R&D related to the discontinued programs announced in April.

Speaker Change: Q2 GAAP Diluted Earnings Per Share was $0.55, an increase of 90% over Q2 last year, and Q2 non-GAAP Diluted Earnings Per Share was $0.96, representing growth of 78% compared to Q2 2023.

Speaker Change: Our bottom line results for Q2 benefited from the strong revenue execution, driven by underlying patient demand and the order timing just discussed as well as Q2 operating expenses that are reflective of our disciplined resource allocation. While we will discuss details of our cost transformation program at Investor Day, we were able to realize some cost savings in Q2 earlier than anticipated, including lower R&D related to the discontinued programs announced in April.

Brian Mueller: Q2 non-GAAP diluted earnings per share was $0.96, representing growth of 78% compared to Q2 2021. Our bottom line results for Q2 benefited from the strong revenue execution driven by underlying patient demand and the order timing just discussed, as well as Q2 operating expenses that are reflective of our disciplined resource allocation. While we will discuss details of our cost transformation program at Investor Day, we were able to realize some cost savings in Q2 earlier than anticipated, including lower R&D related to the discontinued programs announced. Our strong first half execution drove our increased profitability guidance for 2024. We are increasing our 2024 non-GAAP operating margin guidance to 26. Transcribed by https://otter.ai. At the midpoint, this would represent 7% expansion versus 2023.

Brian Mueller: Sure thing, Chris. The challenge oftentimes in genetic diseases is the diagnostic odyssey and the delay. We hope that during our development program, we can overcome some of those barriers and start to teach our colleagues that early diagnosis is really the path to improved overall outcomes in genetic conditions. As to regulatory requirements for the under-3 population, that's a TBD. The one thing I'd say about that is we've got a lot of wind at our back with health authorities around the world who recognize the safety and the efficacy of VOXZOGO in children under three years of age. It's essentially approved from infancy around the world in achondroplasia. That's a very good platform to develop even further confidence of VOXZOGO in very young children.

Speaker Change: Our bottom line results for Q2 benefited from the strong revenue execution driven by underlying patient demand and the order timing just discussed, as well as Q2 operating expenses that are reflective of our disciplined resource allocation.

Speaker Change: While we will discuss details of our cost transformation program at Investor Day, we were able to realize some cost savings in Q2 earlier than anticipated, including lower R&D related to the discontinued programs announced in April.

Brian Mueller: Our strong first half execution drove our increased profitability guidance for 2024. We are increasing 2024 non-GAAP operating margin guidance to 26% to 27%, which, at the midpoint, would represent 7% expansion versus 2023. We also raised non-GAAP diluted earnings per share guidance to between $3.10 and $3.25 consistent with our core goal to grow profitability faster than revenues. Important to note is that we observe our 2024 profitability is weighted to the first half of the year as a result of the strong quarter. We are expecting revenue growth in the second half of the year as well as overall profitability as indicated in our Q2 guidance. Q2 benefited from the previously mentioned order timing. And with respect to operating expenses, we are expecting our usual trend of operating expenses being weighted to the second half of the year. Considering some of the 2024 first and second half dynamics, we continue to point to our full year guidance and the annual results as the best measure of our performance and overall growth given the quarter-to-quarter timing differences that occur in our business.

Speaker Change: Sent to 27%, which at the midpoint would represent 7% expansion versus 2023. We also raised non-GAAP diluted earnings per share guidance to between $3 10, and $3 25 <unk>. Consistent with our core goal to grow profitability faster than revenues. Important to note is that we observe our 2020 for profitability is weighted to the first half of the year as a result of the strong quarter. We are expecting revenue growth in the second half of the year as well as overall profitability as indicated in our Q2 guidance Q2 benefited from the previously mentioned order timing and with respect to operating expenses, we are expecting our usual trend of operating expenses being weighted to the second half of the year. Considering some of the 2020 for first and second half dynamics, we continue to point to our full year guidance and the annual results as the best measure of our performance and overall growth given the quarter to quarter timing differences that occur in our business.

Speaker Change: Our strong first-half execution drove our increased profitability guidance for 2024. We are increasing 2024 non-GAAP operating margin guidance to 26%

Speaker Change: We also raised non-GAAP diluted earnings per share guidance to between $3 10, and $3 25 <unk>. Consistent with our core goal to grow profitability faster than revenues. Important to note is that we observe our 2020 for profitability is weighted to the first half of the year as a result of the strong quarter. We are expecting revenue growth in the second half of the year as well as overall profitability as indicated in our Q2 guidance Q2 benefited from the previously mentioned order timing and with respect to operating expenses, we are expecting our usual trend of operating expenses being weighted to the second half of the year. Considering some of the 2020 for first and second half dynamics, we continue to point to our full year guidance and the annual results as the best measure of our performance and overall growth given the quarter to quarter timing differences that occur in our business.

Brian Mueller: Important to note is that we observe our 2024 profitability is weighted to the first half of the year as a result of the strong quarter. We are expecting revenue growth in the second half of the year as well as overall profitability as indicated in our Q2 guidance. Q2 benefited from the previously mentioned order timing.

Speaker Change: percent to 27 percent which at the midpoint would represent seven percent expansion versus 2023.

Speaker Change: Consistent with our core goal to grow profitability faster than revenues. Important to note is that we observe our 2020 for profitability is weighted to the first half of the year as a result of the strong quarter. We are expecting revenue growth in the second half of the year as well as overall profitability as indicated in our Q2 guidance Q2 benefited from the previously mentioned order timing and with respect to operating expenses, we are expecting our usual trend of operating expenses being weighted to the second half of the year. Considering some of the 2020 for first and second half dynamics, we continue to point to our full year guidance and the annual results as the best measure of our performance and overall growth given the quarter to quarter timing differences that occur in our business.

Brian Mueller: We also raise our non-GAAP diluted earnings per share guidance to between $3.10 and $3.25, consistent with our core goal to grow profitability faster than. Important to note is that our 2024 profitability is weighted to the first half of the year as a result of this strong quarter. We are expecting revenue growth in the second half of the year as well as overall profitability as indicated in our Q2 guidance. Q2 benefited from the previously mentioned order timing.

Speaker Change: Important to note is that we observe our 2020 for profitability is weighted to the first half of the year as a result of the strong quarter. We are expecting revenue growth in the second half of the year as well as overall profitability as indicated in our Q2 guidance Q2 benefited from the previously mentioned order timing and with respect to operating expenses, we are expecting our usual trend of operating expenses being weighted to the second half of the year. Considering some of the 2020 for first and second half dynamics, we continue to point to our full year guidance and the annual results as the best measure of our performance and overall growth given the quarter to quarter timing differences that occur in our business.

Speaker Change: We also raised non-GAAP diluted earnings per share guidance to between $3.10 and $3.25.

Speaker Change: We are expecting revenue growth in the second half of the year as well as overall profitability as indicated in our Q2 guidance Q2 benefited from the previously mentioned order timing and with respect to operating expenses, we are expecting our usual trend of operating expenses being weighted to the second half of the year. Considering some of the 2020 for first and second half dynamics, we continue to point to our full year guidance and the annual results as the best measure of our performance and overall growth given the quarter to quarter timing differences that occur in our business.

Speaker Change: Consistent with our core goal to grow profitability faster than revenues.

Brian Mueller: So stay tuned for how the diagnostic odyssey is going to unfold, how the clinical program will unfold, and how the regulatory proceedings might unfold. As Alexander said earlier, we just got BRINEURA approved for the very young population. So I think the agencies are now quite familiar with BioMarin and the strategy of obtaining earlier and earlier approvals. And the rise in confidence in the safety of VOXZOGO and efficacy can only help us. As regards to 349 and Vertex's most recently announced decision, I would just contrast two very different approaches that have been undertaken. Vertex, I think, was kind of shooting for the stars to do two very difficult things simultaneously. One was to restore antitrypsin activity, and the other was to prevent polymerization, which causes the liver disease.

Speaker Change: Important to note is that we observe our 2024 profitability is weighted to the first half of the year as a result of this strong quarter.

Brian Mueller: And with respect to operating expenses, we are expecting our usual trend of operating expenses being weighted to the second half of the year. Considering some of the 2024 first and second half dynamics, we continue to point to our full year guidance and the annual results as the best measure of our performance and overall growth given the quarter-to-quarter timing differences that occur in our business.

Speaker Change: We are expecting revenue growth in the second half of the year, as well as overall profitability, as indicated in our Q2 guidance. Q2 benefited from the previously mentioned order timing. And with respect to operating expenses, we are expecting our usual trend of operating expenses being weighted to the second half of the year.

Speaker Change: Considering some of the 2020 for first and second half dynamics, we continue to point to our full year guidance and the annual results as the best measure of our performance and overall growth given the quarter to quarter timing differences that occur in our business.

Brian Mueller: And with respect to operating expenses, we are expecting our usual trend of operating expenses being weighted to the second half. Considering some of the 2024 first and second half dynamics, we continue to point to our full-year guidance and the annual results as the best measure of our performance and overall growth, given the quarter-to-quarter timing differences that occur in our business. Lastly, as a reminder, our $495 million of convertible notes matured on August 1st.

Speaker Change: Considering some of the 2024 first and second half dynamics, we continue to point to our full-year guidance and annual results as the best measure of our performance and overall growth, given the quarter-to-quarter timing differences that occur in our business.

Brian Mueller: Lastly, as a reminder, our $495 million of convertible notes matured on August 1. As we discussed on our Q1 earnings call, for the settlement of these notes, we wanted to focus on a share neutral outcome and leverage the strength of our cash flow. As such, we repaid the notes in cash and have retired the underlying 4 million shares associated with the notes as an anti-dilutive measure that provides for increased earnings per share going forward. While we plan to communicate our complete capital allocation strategy at Investor Day, we determined that funding this debt maturity with our balance sheet was an important improvement to our capital structure and a valuable use of shareholder capital. In summary, we are pleased with strong execution demonstrated across the organization during the quarter. With our decision on ROCTAVIAN behind us, we have confidence in our long-term revenue and income growth and operating margin trajectory into 2025 and beyond further evidenced by this year's significant operating margin improvement and increased earnings per share.

Speaker Change: As we discussed on our Q1 earnings call for the settlement of these notes we wanted to focus on the share neutral outcome and leverage the strength of our cash flows. As such we repay the notes in cash and have retired the underlying 4 million shares associated with the nodes as an anti dilutive measure that provides for increased earnings per share going forward. We plan to communicate our complete capital allocation strategy at Investor Day, we determined that funding this debt maturity with our balance sheet was an important improvement to our capital structure and a valuable use of shareholder capital. In summary, we are pleased with strong execution demonstrated across the organization during the quarter with our decision on rocked avian behind US we have confidence in our long term revenue and income growth and operating margin trajectory into 2025 and beyond further evidenced by this year's significant operating margin improvement and increased earnings per <unk>.

Speaker Change: Lastly, as a reminder, our $495 million of convertible notes matured on August 1st.

Speaker Change: As such we repay the notes in cash and have retired the underlying 4 million shares associated with the nodes as an anti dilutive measure that provides for increased earnings per share going forward. We plan to communicate our complete capital allocation strategy at Investor Day, we determined that funding this debt maturity with our balance sheet was an important improvement to our capital structure and a valuable use of shareholder capital. In summary, we are pleased with strong execution demonstrated across the organization during the quarter with our decision on rocked avian behind US we have confidence in our long term revenue and income growth and operating margin trajectory into 2025 and beyond further evidenced by this year's significant operating margin improvement and increased earnings per <unk>.

Brian Mueller: As we discussed on our Q1 earnings call, for the settlement of these notes, we wanted to focus on a share-neutral outcome and leverage the strength of our cash flow. As such, we repaid the notes in cash and have retired the underlying $4 million shares associated with the notes as an anti-dilutive measure that provides for increased earnings per share going forward.

Speaker Change: As we discussed on our Q1 earnings call, for the settlement of these notes, we wanted to focus on a share-neutral outcome and leverage the strength of our cash flows.

Speaker Change: We plan to communicate our complete capital allocation strategy at Investor Day, we determined that funding this debt maturity with our balance sheet was an important improvement to our capital structure and a valuable use of shareholder capital. In summary, we are pleased with strong execution demonstrated across the organization during the quarter with our decision on rocked avian behind US we have confidence in our long term revenue and income growth and operating margin trajectory into 2025 and beyond further evidenced by this year's significant operating margin improvement and increased earnings per <unk>.

Brian Mueller: While we plan to communicate our complete capital allocation strategy at Investor Day, we determined that funding this debt maturity with our balance sheet was an important improvement to our capital structure and a valuable use of shareholder capital. In summary, we are pleased with strong execution demonstrated across the organization during the quarter. With our decision on ROCTAVIAN behind us, we have confidence in our long-term revenue and income growth and operating margin trajectory into 2025 and beyond further evidenced by this year's significant operating margin improvement and increased earnings per share.

Speaker Change: As such, we repaid the notes in cash and have retired the underlying $4 million shares associated with the notes as an anti-dilutive measure that provides for increased earnings per share going forward.

Brian Mueller: And they've had now a couple of bites of that apple and have not so far succeeded. And maybe they'll gain further traction preclinically. Don't know. What we've done is a much more single-minded focus, which is to stop one of the two big, big problems. And actually, it's the currently untreated problem in alpha-1 antitrypsin. As you know, there's replacement therapy for the lung disease, the loss of function mutation. The problem that we're addressing is a different molecular problem, which is the gain of function mutation, which causes the alpha-1 mutant proteins to polymerize in the liver. And that is currently unaddressed medically. Doing so has the advantage of and what we've shown preclinically is that we can solubilize these polymers, increase their excretion from the liver, and then restore liver health as a consequence.

Speaker Change: While we plan to communicate our complete capital allocation strategy at Investor Day, we determined that funding this debt maturity with our balance sheet was an important improvement to our capital structure and a valuable use of shareholder capital.

Speaker Change: In summary, we are pleased with strong execution demonstrated across the organization during the quarter with our decision on rocked avian behind US we have confidence in our long term revenue and income growth and operating margin trajectory into 2025 and beyond further evidenced by this year's significant operating margin improvement and increased earnings per <unk>.

Brian Mueller: In summary, we are pleased with strong execution demonstrated across the organization during the quarter. With our decision on ROCTAVIAN behind us, we have confidence in our long-term revenue and income growth and operating margin trajectory into 2025 and beyond further evidenced by this year's significant operating margin improvement and increased earnings per share.

Speaker Change: In summary, we are pleased with strong execution demonstrated across the organization during the quarter.

Speaker Change: With our decision on Roktavion behind us, we have confidence in our long-term revenue and income growth and operating margin trajectory into 2025 and beyond.

Brian Mueller: We are excited to share details of our long-term plans with you at Investor Day next month. To remind you, we plan to provide details of our new corporate strategy, including a framework for driving operational effectiveness and efficiency and cost management, we will also share updates on our pipeline and life cycle management plans long-term financial targets, including revenue and operating margins for the next several years and our capital allocation strategy. We are pleased with the work that has been done over the last few quarters and believe we have a very compelling growth story to share. Thank you for your continued support, and we will now open the call to your questions. Operator?

We are excited to share details of our long term plans with you at Investor Day next month to remind you we plan to provide details of our new corporate strategy, including a framework for driving operational effectiveness and efficiency and cost management. We will also share updates on our pipeline and my cycle management plans long term financial targets, including revenue and operating margin for the next several years and our capital allocation strategy. We are pleased with the work that has been done over the last few quarters and believe we have a very compelling growth story to share. Thank you for your continued support and we will now open the call to your questions operator.

Brian Mueller: This is further evidenced by this year's significant operating margin improvement and increased earnings per share. We are excited to share details of our long-term plans with you at Investor Day next month. To remind you, we plan to provide details of our new corporate strategy, including a framework for driving operational effectiveness and efficiency and cost management. We will also share updates on our pipeline and lifecycle management plans, long-term financial targets, including revenue and operating margin for the next several years, and our capital allocation strategy.

Speaker Change: Further evidenced by this year's significant operating margin improvement and increased earnings per share.

Speaker Change: We are excited to share details of our long-term plans with you at Investor Day next month. To remind you, we plan to provide details of our new corporate strategy, including a framework for driving operational effectiveness and efficiency and cost management.

Speaker Change: We will also share updates on our pipeline and my cycle management plans long term financial targets, including revenue and operating margin for the next several years and our capital allocation strategy. We are pleased with the work that has been done over the last few quarters and believe we have a very compelling growth story to share. Thank you for your continued support and we will now open the call to your questions operator.

Brian Mueller: The related result of that is that in patients who have only one gene copy of the mutant protein, but we can reduce the Z polymerization and leave the M protein unaffected and therefore preserve its anti-tryptic activity. The result of all of that in the competitive landscape is having an oral and therefore better titratable product and also a product that's potentially much more broadly deliverable. So we're very excited about the 349 program. And again, I'll update you further on progress that we're making at Investor Day. Thank you.

Brian Mueller: We are pleased with the work that has been done over the last few quarters and believe we have a very compelling growth story to share. Thank you for your continued support, and we will now open the call to your questions. Operator?

Speaker Change: We are pleased with the work that has been done over the last few quarters and believe we have a very compelling growth story to share. Thank you for your continued support and we will now open the call to your questions operator.

Speaker Change: We will also share updates on our pipeline and lifecycle management plans, long-term financial targets including revenue and operating margin for the next several years, and our capital allocation strategy.

Speaker Change: Thank you for your continued support and we will now open the call to your questions operator.

Operator: Thank you. We will now take your questions. If you have dialed in and would like to ask a question, please press star one on your telephone keypad to raise your hand and joined the queue. If you would like to withdraw your question, simply press star one again. If you are dialed in and listening via loud speaker on your device, please pickup your handset and ensure that your device is not on mute when asking your question. We kindly ask that you limit yourself to one question and one follow up to allow time for all participants. Our first question comes from Phil Nadeau with TD Cowen. Please go ahead.

Speaker Change: We are pleased with the work that has been done over the last few quarters and believe we have a very compelling growth story to share.

Speaker Change: Thank you for your continued support and we will now open the call to your questions.

Operator: Thank you. We will now take your questions. If you have dialed in and would like to ask a question, please press star 1 on your telephone keypad to raise your hand and join the queue. If you would like to withdraw your question, simply press star 1 again. If you are dialed in and listening via the loudspeaker on your device, please pick up your handset and ensure that your device is not on mute when asking your question.

Speaker Change: Thank you. We will now take your questions. If you have dialed in and would like to ask a question, please press star 1 on your telephone keypad to raise your hand and join the queue. If you would like to withdraw your question, simply press star 1 again.

Speaker Change: If you are dialed in and listening via loud speaker on your device. Please pickup your handset and ensure that your device is not on mute when asking your question. We kindly ask that you limit yourself to one question and one follow up to allow time for all participants. Our first question comes from Phil Nadeau with TD Cowen. Please go ahead.

Operator: Our next question comes from Gina Wang with Barclays. Please go ahead.

Speaker Change: We kindly ask that you limit yourself to one question and one follow up to allow time for all participants. Our first question comes from Phil Nadeau with TD Cowen. Please go ahead.

Gena Wang: Thank you. I will have a few questions regarding ROCTAVIAN. Given your comments on 2025, is it fair to assume that your assumption is about 30, 40 patients that will be treated in 2025 in order to make a break even? And then I have one question regarding Germany. That 75% of the insurance, what's the reason they didn't cover now, and when do you expect the cover will be in place? In the US, the 3 patients that treated, were these from 3 different sites? How many sites now past contracting phase and in the process of a payer discussion?

Speaker Change: If you are dialed in and listening via loudspeaker on your device, please pick up your handset and ensure that your device is not on mute when asking your question.

Speaker Change: Our first question comes from Phil Nadeau with TD Cowen. Please go ahead.

Operator: We kindly ask that you limit yourself to one question and one follow-up to allow time for all participants. Our first question comes from Phil Nadeau with TD Cowan. Please go ahead. Jeff Nguyen.

Phil Nadeau: Good afternoon and thanks for taking our question. On the updated ROCTAVIAN strategy. Can you talk a bit more about the changes, aside from eliminating a bunch of territories, are you changing the strategy or investment in the regions in where you're going to stay, U.S., Germany and Italy?

Speaker Change: We kindly ask that you limit yourself to one question and one follow-up to allow time for all participants.

Speaker Change: Aside from eliminating a bunch of territories are you changing the strategy or investment in the regions. Where are you going to stay U S, Germany and Italy.

Speaker Change: Our first question comes from Phil Nadeau with TD Cowan. Please go ahead.

Jeff Nguyen: Good afternoon, thanks for taking our question on the updated Roktavian strategy. Can you talk a bit more about the changes? Aside from eliminating a bunch of territories, are you changing the strategy or investment in the regions where you're going to stay? U.S., Germany, and Italy?

Phil Nadeau: Good afternoon, thanks for taking our question on the updated Roktavian strategy. Can you talk a bit more about the changes, aside from eliminating a bunch of territories, are you changing the strategy or investment?

Speaker Change: Where are you going to stay U S, Germany and Italy.

Alexander Hardy: Thanks very much for the question. We're very pleased with the clarity that we're able to provide for ROCTAVIAN. If you remember, we said we would, by Investor Day, communicate the timing criteria for making a decision. We're pleased to be able to share this at the second quarter. So, the approach that we're taking allows us to focus geographically on those three geographies: United States, Germany and Italy also to focus our efforts within those geographies on what we have seen and we've learned to be the most important thing for the success of ROCTAVIAN, which is the patient pull-through activities mainly at the site level. So that's where we're going to focus geographically, but also in terms of types of activity within those geographies. We're also focusing our activities from an R&D and development perspective. We are stopping our life cycle development activities. Just to focus on the current main indication. We'll continue to generate long-term data, and we're also reducing our manufacturing expenses associated with ROCTAVIAN. All of this allows us to operate in an envelope of $60 million in 2025 and underpins our confidence that we're going to be able to achieve profitability for ROCTAVIAN in that time with this focused strategy.

Speaker Change: Are we are very pleased with the clarity that we're able to provide for brookdale. If you remember we said we would. By Investor day communicate the timing the criteria for making a decision we're pleased to be able to share. This at the second quarter. So. The approach that. We're taking allows us to focus geographically or those three geographies. Those states, Germany, and Italy, and also to focus our efforts within those geographies. What we have seen and we've looked to be the most important thing for the success of rotate in which is the patient pull through activities, mainly at the site level. So that's why we're going to focus geographically, but also in terms of types of activity within those geographies. We're also focusing our activities from a. R&D and development perspective. Stopping <unk>. Lifecycle development activities just to focus on the current vein indication will continue to generate long term data and we're also reducing our manufacturing expenses associated with rotate in. All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Speaker Change: In the regions, where are you going to stay? U.S., Germany, and Italy?

Speaker Change: If you remember we said we would. By Investor day communicate the timing the criteria for making a decision we're pleased to be able to share. This at the second quarter. So. The approach that. We're taking allows us to focus geographically or those three geographies. Those states, Germany, and Italy, and also to focus our efforts within those geographies. What we have seen and we've looked to be the most important thing for the success of rotate in which is the patient pull through activities, mainly at the site level. So that's why we're going to focus geographically, but also in terms of types of activity within those geographies. We're also focusing our activities from a. R&D and development perspective. Stopping <unk>. Lifecycle development activities just to focus on the current vein indication will continue to generate long term data and we're also reducing our manufacturing expenses associated with rotate in. All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Alexander Hardy: Yeah, thanks very much for the question. So we're very pleased with the clarity that we're able to provide for Octavian. If you remember, we said we would buy and invest today and communicate the timing criteria for making a decision. We're pleased to be able to share this in the second quarter.

Speaker Change: By Investor day communicate the timing the criteria for making a decision we're pleased to be able to share. This at the second quarter. So. The approach that. We're taking allows us to focus geographically or those three geographies. Those states, Germany, and Italy, and also to focus our efforts within those geographies. What we have seen and we've looked to be the most important thing for the success of rotate in which is the patient pull through activities, mainly at the site level. So that's why we're going to focus geographically, but also in terms of types of activity within those geographies. We're also focusing our activities from a. R&D and development perspective. Stopping <unk>. Lifecycle development activities just to focus on the current vein indication will continue to generate long term data and we're also reducing our manufacturing expenses associated with rotate in. All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Speaker Change: Yeah, thanks very much for the question. So,

Speaker Change: We're very pleased with the clarity that we're able to provide for Octavian.

Brian Mueller: Thanks, Gina. This is Brian. I'll start with your first question. We're not giving specific ROCTAVIAN patient or revenue guidance today. It was important to talk about the progress we're seeing in the launch that Cristin covered earlier and then this cost envelope for next year and the goal of getting to profitability. So framing it up in that way as demonstration and articulation of the strategy. So you can do that as a, again, minimum level of revenues, if you will, but not getting into further specifics at this time.

Speaker Change: So. The approach that. We're taking allows us to focus geographically or those three geographies. Those states, Germany, and Italy, and also to focus our efforts within those geographies. What we have seen and we've looked to be the most important thing for the success of rotate in which is the patient pull through activities, mainly at the site level. So that's why we're going to focus geographically, but also in terms of types of activity within those geographies. We're also focusing our activities from a. R&D and development perspective. Stopping <unk>. Lifecycle development activities just to focus on the current vein indication will continue to generate long term data and we're also reducing our manufacturing expenses associated with rotate in. All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Speaker Change: The approach that. We're taking allows us to focus geographically or those three geographies. Those states, Germany, and Italy, and also to focus our efforts within those geographies. What we have seen and we've looked to be the most important thing for the success of rotate in which is the patient pull through activities, mainly at the site level. So that's why we're going to focus geographically, but also in terms of types of activity within those geographies. We're also focusing our activities from a. R&D and development perspective. Stopping <unk>. Lifecycle development activities just to focus on the current vein indication will continue to generate long term data and we're also reducing our manufacturing expenses associated with rotate in. All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Speaker Change: If you remember, we said we would buy invest today, communicate the timing, the criteria for making a decision. We're pleased to be able to share this at the second quarter.

Speaker Change: We're taking allows us to focus geographically or those three geographies. Those states, Germany, and Italy, and also to focus our efforts within those geographies. What we have seen and we've looked to be the most important thing for the success of rotate in which is the patient pull through activities, mainly at the site level. So that's why we're going to focus geographically, but also in terms of types of activity within those geographies. We're also focusing our activities from a. R&D and development perspective. Stopping <unk>. Lifecycle development activities just to focus on the current vein indication will continue to generate long term data and we're also reducing our manufacturing expenses associated with rotate in. All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Speaker Change: Those states, Germany, and Italy, and also to focus our efforts within those geographies. What we have seen and we've looked to be the most important thing for the success of rotate in which is the patient pull through activities, mainly at the site level. So that's why we're going to focus geographically, but also in terms of types of activity within those geographies. We're also focusing our activities from a. R&D and development perspective. Stopping <unk>. Lifecycle development activities just to focus on the current vein indication will continue to generate long term data and we're also reducing our manufacturing expenses associated with rotate in. All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Alexander Hardy: So, you know, the approach that we're taking allows us to focus geographically on those three geographies, the United States, Germany, and Italy, and also to focus our efforts within those geographies on what we have seen and we've learned to be the most important thing for the success of Roktavien, which is patient pull-through activities, mainly at the site level. So that's where we're going to focus geographically, but also in terms of types of activities within those chokers.

Speaker Change: So, you know, the approach that we're taking allows us to focus geographically on those three geographies.

Speaker Change: What we have seen and we've looked to be the most important thing for the success of rotate in which is the patient pull through activities, mainly at the site level. So that's why we're going to focus geographically, but also in terms of types of activity within those geographies. We're also focusing our activities from a. R&D and development perspective. Stopping <unk>. Lifecycle development activities just to focus on the current vein indication will continue to generate long term data and we're also reducing our manufacturing expenses associated with rotate in. All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Speaker Change: United States, Germany and Italy and also to focus our efforts within those geographies on what we have seen and we've learned to be the most important thing for the success of Roktavien which is the patient pull through activities mainly at the site level.

Speaker Change: So that's why we're going to focus geographically, but also in terms of types of activity within those geographies. We're also focusing our activities from a. R&D and development perspective. Stopping <unk>. Lifecycle development activities just to focus on the current vein indication will continue to generate long term data and we're also reducing our manufacturing expenses associated with rotate in. All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Speaker Change: We're also focusing our activities from a. R&D and development perspective. Stopping <unk>. Lifecycle development activities just to focus on the current vein indication will continue to generate long term data and we're also reducing our manufacturing expenses associated with rotate in. All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Speaker Change: R&D and development perspective. Stopping <unk>. Lifecycle development activities just to focus on the current vein indication will continue to generate long term data and we're also reducing our manufacturing expenses associated with rotate in. All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Speaker Change: Stopping <unk>. Lifecycle development activities just to focus on the current vein indication will continue to generate long term data and we're also reducing our manufacturing expenses associated with rotate in. All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Cristin Hubbard: Yeah. And I'll take the second two questions. So with regard to the subinsurers, I mean, it's impossible to give you an exact time of it, but I can say that we're making progress in these discussions that we're having. And really, we're just pushing through insurer by insurer and making sure that we are addressing the needs, which, of course, are going to be about risk sharing. On the US side, you asked about the HTCs that have been treated. We've had those 3 patients treated. They were all in separate HTCs and were actually geographically dispersed across the US.

Speaker Change: So that's where we're going to focus geographically, but also in terms of types of activity.

Speaker Change: Lifecycle development activities just to focus on the current vein indication will continue to generate long term data and we're also reducing our manufacturing expenses associated with rotate in. All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Alexander Hardy: We're also focusing our activities from a R&D and development perspective. We are stopping our life cycle development activities just to focus on the current main indication. We'll continue to generate long-term data, and we're also reducing our manufacturing expenses associated with Roktavia. All of this allows us to operate in an envelope of $60 million in 2025 and underpins our confidence that we're going to be able to achieve profitability for Octavian in that time with this focus strategy.

Speaker Change: are within those geographies.

Speaker Change: We're also focusing our activities from a

Speaker Change: R&D and Development Perspective

Speaker Change: We are stopping our life cycle development activities just to focus on the current main indication. We'll continue to generate long-term data and we're also reducing our manufacturing expenses associated with Roktavion.

Speaker Change: All of this allows us to operate in an envelope of a $60 million turning 25. And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Speaker Change: And underpins our confidence that we're going to be able to achieve profitability for Octavian and that time with this focused strategy.

Speaker Change: All of this allows us to operate in an envelope of $60 million in 2025, and underpins our confidence that we're going to be able to achieve profitability for Octavian in that time with this focus strategy.

Phil Nadeau: And when will we hear the next update on the strategy? It sounds like this is a framework--an initial strategy, but you're going to continue to identify the investment and success of ROCTAVIAN. When do you think you'd be in a position to give a further confirmation of the strategy or revision of the strategy if necessary?

Gena Wang: Thank you.

Operator: Our next question comes from Costas Beloris with BMO Capital Markets. Please go ahead.

Alexander Hardy: And when will we hear the next update on the strategy? It sounds like this is a framework, an initial strategy, but you're going to continue to identify the investment and success of Roktevian. When do you think you'd be in a position to give a further confirmation of the strategy or revision of the strategy, if necessary?

Speaker Change: When do you think you'd be in a position to give a further confirmation of the strategy or a revision of the strategy if necessary.

Kostas Biliouris: Thanks for taking our question and congrats on the strong quarter. One question from us on ROCTAVIAN. Acknowledging that Hemgenix in hemophilia B was approved six months before ROCTAVIAN in the US and that the hemophilia B population is four to five times smaller than the hemophilia A, how are you benchmarking ROCTAVIAN revenues versus Hemgenix, which generated about $15 to 30 million in the first half of 2024 based on our estimations? Thank you.

Speaker Change: And when will we hear the next update on the strategy? It sounds like this is a framework, an initial strategy, but you're going to continue to identify the investment and success of Roktevian. When do you think you'd be in a position to give a further confirmation of the strategy or revision of the strategy if necessary?

Alexander Hardy: No, this is a decision of going forward for ROCTAVIAN. We will continue to update you on our progress. As we've said before, the success metric we're all focused on is patients infused and treated. And so, we'll update you on our progress on those metrics, but this is our strategy. We've reached a decision. We have confidence that ROCTAVIAN can be a contributor to Biomarin's profitability going forward.

Octavian: Going forward for Octavian. We will continue to update you on our progress. As we've said before. The success metric, we're all focused on his patients infused treated so we will update you on our progress on those metrics, but this is our strategy we've reached decision. We have confidence that <unk> can be a contributor to <unk> profitability going forward.

We will continue to update you on our progress. As we've said before. The success metric, we're all focused on his patients infused treated so we will update you on our progress on those metrics, but this is our strategy we've reached decision. We have confidence that <unk> can be a contributor to <unk> profitability going forward.

Octavian: As we've said before. The success metric, we're all focused on his patients infused treated so we will update you on our progress on those metrics, but this is our strategy we've reached decision. We have confidence that <unk> can be a contributor to <unk> profitability going forward.

Alexander Hardy: Now this is a decision about going forward for Roktavien. We will continue to update you on our progress. As we've said before, the success metric we're all focused on is patients infused and treated. And so we'll update you on our progress on those metrics. But this is our strategy. We've reached a decision. We have confidence that Roktavien can be a contributor to Biomin's profitability going forward

Octavian: The success metric, we're all focused on his patients infused treated so we will update you on our progress on those metrics, but this is our strategy we've reached decision. We have confidence that <unk> can be a contributor to <unk> profitability going forward.

Speaker Change: Now this is a decision of going forward for Octavian.

Speaker Change: We will continue to update you on our progress. As we've said before, the success metric we're all focused on.

Octavian: We have confidence that <unk> can be a contributor to <unk> profitability going forward.

Brian Mueller: Yeah. Thanks for the question, Costas. I'll jump in on that one, Brian, since I've been tracking the Hemgenix launch along the way. There's different circumstances there between the two launches. Of course, there's differences as well, but we're not getting into launch comparisons or benchmarking Hemgenix. More of a qualitative assessment. Again, we are going through similar contracting and access challenges. So there is a comparison to be made, but we don't do it at that quantitative level.

Speaker Change: His patients infused and treated and so we'll update you on our progress on those metrics, but this is our strategy We've reached a decision We have confidence that Roktavien can be a contributor to to Biomin's profitability going forward

Phil Nadeau: That's very helpful. Thanks for taking my questions.

Operator: Our next question comes from Salveen Richter with Goldman Sachs. Please go ahead.

<unk> Richter: <unk> Richter with Goldman Sachs. Please go ahead.

Operator: That's very helpful. Thanks for taking our questions. Our next question comes from Salveen Richter with Goldman Sachs. Please go ahead. Good afternoon. Thank you for taking my questions. I just want to dig in further into Octavian with regard to the break.

Salveen Richter: Good afternoon, thank you for taking my question. I just want to dig in further into ROCTAVIAN. With regard to the breakeven situation by year-end '25, could you just speak to the metrics that are giving you confidence at this time point? Is it the demand aspect? Is it 340B dynamics improving in the U.S.? And just maybe help us understand where all these moving parts are coming from to give you that conclusion.

Operator: Our next question comes from Salveen Richter of Goldman Sachs. Please go ahead.

Speaker Change: That's very helpful. Thanks for taking our questions.

Speaker Change: Our next question comes from Salveen Richter with Goldman Sachs. Please go ahead.

Speaker Change: The metrics that are giving you confidence at this time point of it. The demand aspect is at 240, <unk> dynamics improving in the U S. Just maybe help us understand where all of these moving parts are coming from to give you that conclusion.

Salveen Richter: Good afternoon. Thank you for taking my questions. I just want to dig in further into Octavian with regard to the break-even situation by urine 25. Could you just speak to

Speaker Change: The demand aspect is at 240, <unk> dynamics improving in the U S. Just maybe help us understand where all of these moving parts are coming from to give you that conclusion.

Salveen Richter: The metrics that are giving you confidence at this time point, is it, you know, the demand aspect, is it 340B dynamics improving in the U.S., and just maybe help us understand where all these moving parts are coming from to give you that conclusion.

Operator: Our next question comes from Mohit Bansal with Wells Fargo. Please go ahead.

Cristin Hubbard: Yes. Thank you so much for the question. This is Cristin Hubbard. I think that really the progress that we're seeing, and I'll be specific in the three geographies, is that in the United States, we really are encouraged with the three patients that were treated in the quarter. And really, what this is around is ensuring that the sites are operationally ready to infuse product, and we've seen a fourfold increase in this. Our commercial team is very focused here, and we've seen a fourfold increase in this just since January. But then also, as Alexander alluded to, the patient pull-through. And it's really about building comfort around working through the single case agreements that we know will be true to ensure that patients are being treated. So with that type of experience, we recognize that that will create more progress moving forward and a comfort with doing so, and we're seeing a lot of that in the U.S. today. In Italy, in particular, we are really pleased with the rapid progress that we've made in ROCTAVIAN since only receiving pricing and reimbursement since January. So we've treated a couple of patients there, and we're seeing large amounts of strong advocacy from KOLs as well as experience in the sites.

Speaker Change: I think that really the progress that we're seeing and I'll be specific in the three geographies is that in the United States. We really are encouraged with the three patients that were treated in the quarter and really what this is around is ensuring that the sites are up and operational operationally ready to infuse product and we've seen a fourfold increase unless our commercial team is. <unk> focused here and we've seen a fourfold increase in this just since January but then also as Alexandra alluded to the patient pull through and it's really about building comfort around working through the single case agreements that we know will be true too to ensure that patients are being treated so with that type of experience. We recognize that that will create more progress moving forward. And our comfort with doing so and we're seeing a lot of that in the U S. Today. In Italy in particular, we are really pleased with the rapid progress that we've made in Iraq KVM since only receiving pricing and reimbursement since January so we've treated a couple of patients there and we're seeing a large amount of strong advocacy from kols as well as well as experience in the sites.

Mohit Bansal: Sure. Thank you for taking my question. Just wanted to reconcile some of the VOXZOGO numbers. So Brian, if I understand you correctly, the $20 million number for inventory, it is all inventory, right? That was one time. And if I assume that to be inventory, you added quite a lot of patients in this first half. I mean, I think the number was about 2,600 patients by the end of the year. Now you are at 3,500. That is 35% increase. That is not reflecting in the revenue growth since the last year. So just wanted to understand if there's a pricing dynamic that is in play or a geographical mixed dynamic that we should know about.

Kristen Hubbard: Yeah, thank you so much for the question. This is Kristen Hubbard.

Salveen Richter: Yeah, thank you so much for the question. This is Kristen Hubbard. You know, I think that really the progress that we're seeing, and I'll be specific in the three geographies,

Salveen Richter: , and the United States, we are encouraged with the three patients that were treated in the quarter, and really what this is around is ensuring that the sites are operationally ready to infuse product, and we've seen a four-fold increase in this. Our commercial team is very focused here, and we've seen a four-fold increase in this.

Speaker Change: <unk> focused here and we've seen a fourfold increase in this just since January but then also as Alexandra alluded to the patient pull through and it's really about building comfort around working through the single case agreements that we know will be true too to ensure that patients are being treated so with that type of experience. We recognize that that will create more progress moving forward. And our comfort with doing so and we're seeing a lot of that in the U S. Today. In Italy in particular, we are really pleased with the rapid progress that we've made in Iraq KVM since only receiving pricing and reimbursement since January so we've treated a couple of patients there and we're seeing a large amount of strong advocacy from kols as well as well as experience in the sites.

Kristen Hubbard: Um, you know, I think that really the progress that we're seeing, and I'll be specific about the three geographies, is that in the United States, we are really encouraged by the three patients that were treated in the quarter. And, and really what this is about is ensuring that the sites are operational and operationally ready to infuse product. And we've seen a fourfold increase in this; our commercial team is very focused here, and we've seen a fourfold increase in this just since January.

Alexander Hardy: just since January, but then also, as Alexander alluded to, the patient pull-through, and it's really about building comfort around working through the single case agreements that we know will be true to ensure that patients are being treated. So with that type of experience, we recognize that that will create

Kristen Hubbard: But then also, as Alexander alluded to, the patient pulls through, and it's really about building comfort around working through the single case agreements that we know will be true to ensure that patients are being treated. So with that type of experience, we recognize that that will create more progress moving forward and more comfort with doing so. And we're seeing a lot of that in the U.S. today.

Speaker Change: And our comfort with doing so and we're seeing a lot of that in the U S. Today. In Italy in particular, we are really pleased with the rapid progress that we've made in Iraq KVM since only receiving pricing and reimbursement since January so we've treated a couple of patients there and we're seeing a large amount of strong advocacy from kols as well as well as experience in the sites.

Brian Mueller: Yeah. Thanks for the question, Mohit. And there are going to be different dynamics from quarter to quarter in terms of the patient growth rates and revenue growth rates. I tried to address that a bit in the prepared remarks, but I might even point you to last quarter where there was a significant amount of patient adds, but you didn't see that necessarily in the revenue growth. And this time around, in the second quarter here, because of that order timing that you noted, thanks to the increased supply becoming available earlier, that dynamic flipped the other way around. And this time, revenues were ahead of patients a bit and recognized also that in the second half, we could see patient growth rates exceed revenue growth rates in either Q3 or Q4. But I mean, big picture, we're in the third year of this launch.

Speaker Change: In Italy in particular, we are really pleased with the rapid progress that we've made in Iraq KVM since only receiving pricing and reimbursement since January so we've treated a couple of patients there and we're seeing a large amount of strong advocacy from kols as well as well as experience in the sites.

Alexander Hardy: More progress moving forward and a comfort with doing so and we're seeing a lot of that in the in the US today

Speaker Change: In Italy, in particular, we are really pleased with the rapid progress that we've made in in Roktavion since only receiving pricing and reimbursement since January . So we've treated a couple patients there and we're seeing large amounts of strong advocacy from KOLs as well as well as experience in the sites.

Cristin Hubbard: And then lastly, with Germany, as I said a little bit earlier, we have reached a negotiated agreement with the subinsurer, and we are happy to report that we now have approximately 25% of patient lives covered in Germany, and we will continue to push forward with progress with the subinsurers there. We know there's demand. We do have KOL advocacy in Germany, and we want to continue on that front. So, these are the types of metrics that we're looking at, and I'll pass it to Brian for more.

Speaker Change: I as I've said, a little bit earlier, we have reached a negotiated agreement with the southern shore and we are happy to report that we now have approximately 25% of patient lives covered in Germany, and we will continue to push forward with progress with the southern shares. There. We know there's demand we do have kols advocacy in Germany, and we want to continue on that front. So these are the types of metrics that we're looking at. I will pass it to Brian from yes.

Kristen Hubbard: In Italy, in particular, we are really pleased with the rapid progress that we've made in Roktavien since only receiving pricing and reimbursement since January. So we've treated a couple of patients there, and we're seeing large amounts of strong advocacy from KOLs as well, as well as experience in the sites. And then lastly, with Germany, as I said a little bit earlier, we have reached a negotiated agreement with the sub-insurer, and we are happy to report that we now have approximately 25% of patient lives covered in Germany, and we will continue to push forward with progress with the sub-insurers there.

Speaker Change: And then lastly with Germany, as I said a little bit earlier, we have reached a negotiated agreement with the sub-insurer, and we are happy to report that we now have approximately 25% of patient lives covered in Germany, and we will continue to push forward with progress with the sub-insurers there. We know there's demand, we do have KOL advocacy in Germany, and we want to continue on that front.

Speaker Change: I will pass it to Brian from yes.

Brian Mueller: Yes, hi, Salveen, this is Brian. Just to add to Cristin's comments, in addition to those signs of progress and our continued execution across those three markets with this focused strategy, that plus this revised $60 million cost envelope is what gives us confidence that we can manage the asset to be profitable next year.

Kristen Hubbard: We know there's demand; we do have KOL advocacy in Germany, and we want to continue on that front. So these are the types of metrics that we're looking at, and I'll pass this to Brian. Yeah. Hi, Salveen. This is Brian.

Brian: In addition to those guys. Signs of progress and our continued execution across those three markets with this focused strategy. That plus this revised $60 million cost envelope is what gives us confidence that we can manage the asset to be profitable next year. Okay.

Brian: Signs of progress and our continued execution across those three markets with this focused strategy. That plus this revised $60 million cost envelope is what gives us confidence that we can manage the asset to be profitable next year. Okay.

Brian Mueller: Just to add to Kristen's comments, you know, in addition to those signs of progress in our continued execution across those three markets with this focused strategy, that plus this revised $60 million cost envelope is what gives us confidence that we can manage the asset to be profitable. Our next question comes from Ellie Merle with UBS. Please go ahead. Hey guys, thanks for taking the question. Just in terms of 333, how are you thinking about the

Brian Mueller: Yeah. Hi Salveen. This is Brian.

Speaker Change: So these are the types of metrics that we're looking at, and I'll pass it to Brian for more.

Brian Mueller: We're still less than 20% penetrated with a long way to go globally. We're on track for VOXZOGO to exceed $1 billion in revenue. Yes, there's going to be differences in quarter-to-quarter timing of patients and orders. We think those two will correlate over time. But we're out there executing quarter-to-quarter. There's going to be timing differences.

Brian: That plus this revised $60 million cost envelope is what gives us confidence that we can manage the asset to be profitable next year. Okay.

Brian Mueller: Yeah, hi Salveen, this is Brian . Just to add to Kristen's comments, you know, in addition to those signs of progress in our continued execution across those three markets with this focus strategy,

Brian: Okay.

Brian Mueller: That plus this revised $60 million cost envelope is what gives us confidence that we can manage the asset to be profitable next year.

Operator: Our next question comes from Ellie Merle with UBS. Please go ahead.

Ellie Merle: Thanks for taking my question. Just in terms of 333, how are you thinking about the development of 333 relative to VOXZOGO across different indications? Are there any indication where you would move forward with 333 instead of VOXZOGO? And then just a second question on idiopathic short stature. Given the much larger prevalence, what are your plans to potentially focus on a subset of patients with ISS within that 600,000 prevalence that you've cited?

Ellie Merle: Just in terms of grade three how are you thinking about the development of phase III relative tobacco, though across different indications are there any indications where you can move forward with pretty quickly in quantum box logo. And then just a quick question. Idiopathic short Walter. As a much larger problem what are your plans to potentially bogus. Ah patients. Hum. Okay. Thank you Paul.

Mohit Bansal: Got it. Thank you.

Operator: Our next question comes from Ellie Merle with UBS. Please go ahead. Thank you, guys. Thank you.

Operator: Your next question comes from Olivia Brayer with Cantor Fitzgerald. Please go ahead.

Speaker Change: Our next question comes from Ellie Merle with UBS. Please go ahead.

Olivia Brayer: Hey. Good afternoon, guys. Thank you for the question. As you consider profitability next year and just broader uptake of ROCTAVIAN, how are you thinking about sales contribution and breakdown across the three geographies that you're focused on? I'm just trying to get a sense for whether you expect the US to really make up the majority of ROCTAVIAN sales going forward. Thank you.

Ellie Merle: Hey guys, thanks for taking the question. Just in terms of 3.3.3, how are you thinking about the development of 3.3.3 relative to VoxOgo across different indications?

Speaker Change: And then just a quick question. Idiopathic short Walter. As a much larger problem what are your plans to potentially bogus. Ah patients. Hum. Okay. Thank you Paul.

Speaker Change: Idiopathic short Walter. As a much larger problem what are your plans to potentially bogus. Ah patients. Hum. Okay. Thank you Paul.

Speaker Change: As a much larger problem what are your plans to potentially bogus. Ah patients. Hum. Okay. Thank you Paul.

Ellie Merle: Are there any indications where you would move forward with 333 instead of VoxOgo?

Speaker Change: Ah patients. Hum. Okay. Thank you Paul.

Speaker Change: Hum. Okay. Thank you Paul.

Speaker Change: Okay. Thank you Paul.

Paul: Thank you Paul.

Speaker Change: And then just a second question on idiopathic short stature.

Hank Fuchs: Hi, Ellie, this is Hank. As far as the development plans for 333, our initial thinking is, first of all, to get it into the clinic, which will start shortly. And thereafter to proceed with the most expedited path to approval, leveraging potential for superior efficacy, superior convenience or both. As regards to the program of idiopathic short stature, the opportunity there is in patients with the greatest unmet need, potentially more specifically patients who have severe stature deficiency, or patients who have been unsatisfied with available current therapy. So, I hope that addresses your questions.

Brian Mueller: Yeah. Thanks, Olivia. It's a good question. Noteworthy there just in terms of the US versus ex-US dynamic. The price of ROCTAVIAN, net revenue per patient on average in the US is going to be higher. So as we continue to make progress in the US market, those patients are going to contribute more to revenue. Again, we're focused on these three markets, both with the restructured cost envelope as well as the tactics, and this dedicated and focused business unit that will be working exclusively on ROCTAVIAN. It's going to allow the rest of the business to focus on the remainder of the portfolio. So we're viewing this as the three markets together, not getting into further details of each of those individual markets at this time. But important to know that those three markets are the focus area and that will be the contributors to revenue in 2025.

Speaker Change: Given the much larger prevalence, what are your plans to potentially focus on a subset of patients with ISF within that 600,000 prevalence that you've cited? Thanks.

Speaker Change: As far as the development plans for $3 33. Our initial thinking is. First of all to get it into the clinic, which will start shortly. And thereafter to Purdue to proceed with the most expedited path. Sure approval leveraging. Potential for superior efficacy superior convenience or both. As regards the program of idiopathic short stature. The opportunity there. Patients with the greatest. Met need. Potentially more specifically patients who have severe statue of sufficient deficiency, where patients who have been unsatisfied with available current therapy. So I hope that addresses your questions.

Speaker Change: Our initial thinking is. First of all to get it into the clinic, which will start shortly. And thereafter to Purdue to proceed with the most expedited path. Sure approval leveraging. Potential for superior efficacy superior convenience or both. As regards the program of idiopathic short stature. The opportunity there. Patients with the greatest. Met need. Potentially more specifically patients who have severe statue of sufficient deficiency, where patients who have been unsatisfied with available current therapy. So I hope that addresses your questions.

Speaker Change: First of all to get it into the clinic, which will start shortly. And thereafter to Purdue to proceed with the most expedited path. Sure approval leveraging. Potential for superior efficacy superior convenience or both. As regards the program of idiopathic short stature. The opportunity there. Patients with the greatest. Met need. Potentially more specifically patients who have severe statue of sufficient deficiency, where patients who have been unsatisfied with available current therapy. So I hope that addresses your questions.

Hank Fuchs: Hi Ellie, this is Hank. As far as the development plans for 333, you know, our initial thinking is, first of all, to get it into the clinic, which will start shortly, and thereafter to proceed with the most expedited path to approval, leveraging potential for superior efficacy, superior convenience, or both. As regards the program for idiopathic short stature, the opportunity is in patients with the greatest unmet need, potentially more specifically patients who have severe statural deficiency or patients who have been unsatisfied with available current therapy. So I hope that addresses your question.

Speaker Change: And thereafter to Purdue to proceed with the most expedited path. Sure approval leveraging. Potential for superior efficacy superior convenience or both. As regards the program of idiopathic short stature. The opportunity there. Patients with the greatest. Met need. Potentially more specifically patients who have severe statue of sufficient deficiency, where patients who have been unsatisfied with available current therapy. So I hope that addresses your questions.

Speaker Change: Hi Ellie, this is Hank. As far as the development plans for 333,

Sure approval leveraging. Potential for superior efficacy superior convenience or both. As regards the program of idiopathic short stature. The opportunity there. Patients with the greatest. Met need. Potentially more specifically patients who have severe statue of sufficient deficiency, where patients who have been unsatisfied with available current therapy. So I hope that addresses your questions.

Hank Fuchs: You know, our initial thinking is, first of all, to get it into the clinic, which will start shortly, and thereafter to proceed with the most expedited task.

Speaker Change: Potential for superior efficacy superior convenience or both. As regards the program of idiopathic short stature. The opportunity there. Patients with the greatest. Met need. Potentially more specifically patients who have severe statue of sufficient deficiency, where patients who have been unsatisfied with available current therapy. So I hope that addresses your questions.

Speaker Change: As regards the program of idiopathic short stature. The opportunity there. Patients with the greatest. Met need. Potentially more specifically patients who have severe statue of sufficient deficiency, where patients who have been unsatisfied with available current therapy. So I hope that addresses your questions.

Speaker Change: The opportunity there. Patients with the greatest. Met need. Potentially more specifically patients who have severe statue of sufficient deficiency, where patients who have been unsatisfied with available current therapy. So I hope that addresses your questions.

Hank Fuchs: to approval leveraging

Speaker Change: Potential for superior efficacy, superior convenience, or both.

Speaker Change: Patients with the greatest. Met need. Potentially more specifically patients who have severe statue of sufficient deficiency, where patients who have been unsatisfied with available current therapy. So I hope that addresses your questions.

Speaker Change: Met need. Potentially more specifically patients who have severe statue of sufficient deficiency, where patients who have been unsatisfied with available current therapy. So I hope that addresses your questions.

Speaker Change: Potentially more specifically patients who have severe statue of sufficient deficiency, where patients who have been unsatisfied with available current therapy. So I hope that addresses your questions.

Speaker Change: As regards the program of idiopathic short stature, the opportunity there is in patients with the greatest unmet need.

Speaker Change: So I hope that addresses your questions.

Speaker Change: Patients who have severe statural deficiency or patients who have been unsatisfied with available current therapy.

Ellie Merle: Great. Thanks.

Operator: Our next question--

Operator: This will be our last question. Our last question comes from Luca Issi with RBC Capital. Please go ahead.

Alexander Hardy: Ellie, we'll share more information on the development plans for VOXZOGO at Investor Day, we'll be sharing the specifics around the design of our studies and also the TAMs we're expecting to be able to achieve with those development programs.

Speaker Change: I hope that addresses your questions.

Operator: Our next question comes from Corey.

Speaker Change: Great, thanks.

Lee: Today, I will be sharing the specifics. Around the design of our studies. I'd also the times. We're expecting to be able to achieve. With those development programs.

Luca Issi: Oh, great. Thanks so much for taking my question. Congrats on the progress. Maybe two quick ones on ROCTAVIAN. Maybe you're obviously prioritizing Italy, Germany, and the US. But what's the plan for the other geographies where you still have rights? Is there any plan there to find a partner or try to monetize that in any capacity? Any color there, much appreciated. And maybe sticking to ROCTAVIAN, maybe just talk about net pricing in Italy versus the US. I believe last quarter, you reported $800,000 for the only patients treated in Italy versus today, $7.4 billion for three patients in the US and two in Italy. That would imply net pricing of $1.9 million in the US and $800,000 in Italy. Is that right? And if so, how should we think about pricing in Germany? Thanks so much.

Speaker Change: Our next question comes from Corey.

Lee: Around the design of our studies. I'd also the times. We're expecting to be able to achieve. With those development programs.

Operator: And Ellie, we'll share more information on the development plans for Boxergo at Invest today. We'll be sharing the specifics around the design of our studies and also the TAMs we're expecting to be able to achieve with those developments.

Speaker Change: I'd also the times. We're expecting to be able to achieve. With those development programs.

Speaker Change: We'll share more information on the development plans for Boxergo at Invest Today. We'll be sharing the specifics.

Lee: We're expecting to be able to achieve. With those development programs.

Lee: With those development programs.

Operator: Our next question comes from Cory Kasimov with Evercore. Please go ahead.

Speaker Change: around the design of our studies and also the TAMs we're expecting to to be able to achieve.

Cory Kasimov: Great. Thanks for taking the question. I have a two-part question on VOXZOGO. First kind of big picture. So, assuming you're successful in adding additional indications here, what are your current expectations out of Washington on the potential impact of the IRA when it comes to multi-indication products for orphan diseases? And then the second one is, we're thinking about the hypochondroplasia opportunity. Curious how this indication relates to--how it's similar, different to achondroplasia in terms of the level of market awareness and maybe the motivation on the part of the patients to get treatment as we think about kind of as you proceed through your Phase III trial? Thanks.

Operator: Our next question comes from Corey Kazimoff with Evercore. Please go ahead.

Speaker Change: with those development programs.

Speaker Change: Our next question comes from Corey Kazimoff with Evercore. Please go ahead.

Corey Kazimoff: Great, thanks for taking the question. I have a two-part question on VoxOgo.

Speaker Change: Are your current expectations out of Washington on the potential impact of the IRA when it comes to multi indication products for orphan diseases. And then the second one is as we're thinking about the hypochondriac <unk> opportunity I'm curious, how this indication relate to or how it is similar or different to two achondroplasia in terms of the level of market awareness and maybe the motivation on the part of the patient to get treatment as we think about kind of <unk>. Proceed through your phase III trials. Thanks, Laura.

Corey Kazimoff: Great. Thanks for taking the question. I have a two-part question on VoxOgo. First, kind of big picture. So, assuming you're successful in adding additional indications here, what are your current expectations out of Washington on the potential impact of the IRA when it comes to multi-indication products for orphan diseases?

Speaker Change: And then the second one is as we're thinking about the hypochondriac <unk> opportunity I'm curious, how this indication relate to or how it is similar or different to two achondroplasia in terms of the level of market awareness and maybe the motivation on the part of the patient to get treatment as we think about kind of <unk>. Proceed through your phase III trials. Thanks, Laura.

Brian Mueller: Thanks, Luca. This is Brian. I'll take that one. So first of all, in terms of the focus, it's an important question because the key element behind the driver of the $60 million cost structure for next year is focusing entirely on supporting patients in those three markets as well as the long-term overall clinical and regulatory commitments that come with ROCTAVIAN. That means that we are not investing in additional markets at this time. As we prove out and continue to gain traction and get confidence in ROCTAVIAN's overall progress over time, we'll retain the right to make select investments that are value accretive to the asset over time. But important to note that it is just those three markets commercially at this time. And on pricing, I'll share, again, it's going to be dynamic over time with variations in the global pricing.

Alexander Hardy: First, kind of the big picture, so assuming you're successful and adding additional indications here, what are your current expectations out of Washington on the potential impact of the IRA when it comes to multi-indication products for orphan diseases? And then the second one is that we're thinking about the hypochondroplasia opportunity. Curious how this indication relates to or how it's similar or different to achondroplasia in terms of the level of market awareness and maybe the motivation on the part of the patient to get treatment as we think about kind of as you proceed through your phase three trial. Thanks a lot.

Speaker Change: And then the second one is we're thinking about the hypochondroplasia opportunity, curious how this indication relates to or how it's similar or different to achondroplasia in terms of

Speaker Change: Proceed through your phase III trials. Thanks, Laura.

Alexander Hardy: Hey, Cory. Thanks very much for your questions. I'll handle the first one and then hand over to Cristin for the second one. So, the first one with regard to the potential impact of IRA. We actually think there'll be minimal impact of IRA on VOXZOGO's life cycle development plans, whilst the IRA overall from a legislation standpoint is a very significant factor for the industry in terms of impacting innovation in many important areas. We don't think it's going to have a significant impact on VOXZOGO, and that's because of the payer mix that we have for VOXZOGO with these indications where we see minimal exposure to Medicare. And I'll hand over now to Hank for the second question.

Speaker Change: The level of market awareness and maybe the motivation on the part of the patient to get treatment as we think about kind of as you proceed through your phase three trial. Thanks a lot.

Speaker Change: So the first one with regard to the potential impacted by array. We actually think there will be minimal impact. Array on books Arcos. Lifecycle development plans. Whilst the IRI overrule from a legislation standpoint is a very significant factor for the industry in terms of impacting innovation in many important areas. We don't think it's going to have a significant impact on books are gone and thats because of the payer mix. For bulk sargo with these start with these indications from legacy minimum exposure to Medicare. I'll hand over now to you. The second question.

Alexander Hardy: Hey Corey, thanks very much for your questions. I'll handle the first one and then hand over to Kristen for the second one. So the first one with regard to the potential impact of IRA, we actually think there'd be minimal impact of IRA on Boxoco's life cycle development plan. The IRA overall from a legislative standpoint is a very significant factor for the industry in terms of impacting innovation in many important areas. We don't think it's going to have a significant impact on VoxAgra, and that's because of the pay-off for Boxergo for these indications where we see minimal exposure to Medicare.

Speaker Change: We actually think there will be minimal impact. Array on books Arcos. Lifecycle development plans. Whilst the IRI overrule from a legislation standpoint is a very significant factor for the industry in terms of impacting innovation in many important areas. We don't think it's going to have a significant impact on books are gone and thats because of the payer mix. For bulk sargo with these start with these indications from legacy minimum exposure to Medicare. I'll hand over now to you. The second question.

Speaker Change: Hey Corey, thanks very much for your questions. I'll handle the first one and then hand over to Kristen for the second one.

Christian: Array on books Arcos. Lifecycle development plans. Whilst the IRI overrule from a legislation standpoint is a very significant factor for the industry in terms of impacting innovation in many important areas. We don't think it's going to have a significant impact on books are gone and thats because of the payer mix. For bulk sargo with these start with these indications from legacy minimum exposure to Medicare. I'll hand over now to you. The second question.

Christian: Lifecycle development plans. Whilst the IRI overrule from a legislation standpoint is a very significant factor for the industry in terms of impacting innovation in many important areas. We don't think it's going to have a significant impact on books are gone and thats because of the payer mix. For bulk sargo with these start with these indications from legacy minimum exposure to Medicare. I'll hand over now to you. The second question.

Christian: Whilst the IRI overrule from a legislation standpoint is a very significant factor for the industry in terms of impacting innovation in many important areas. We don't think it's going to have a significant impact on books are gone and thats because of the payer mix. For bulk sargo with these start with these indications from legacy minimum exposure to Medicare. I'll hand over now to you. The second question.

Speaker Change: So the first one with regard to the potential impact of IRA, we actually think there will be minimal impact of IRA on Bok Soko's life cycle development plans.

Christian: We don't think it's going to have a significant impact on books are gone and thats because of the payer mix. For bulk sargo with these start with these indications from legacy minimum exposure to Medicare. I'll hand over now to you. The second question.

Kristen Hubbard: Whilst the IRA overall, from a legislation standpoint, is a very significant factor for the industry in terms of impacting innovation in many important areas, we don't think it's going to have a significant impact on Voxergo, and that's because of the pay and mix that we have.

Speaker Change: For bulk sargo with these start with these indications from legacy minimum exposure to Medicare. I'll hand over now to you. The second question.

Brian Mueller: I will share that the net revenue that we recognized for those five patients across the two regions where we had sales in Q2 was consistent with our expectations and prior communications. The largest growth to net item in the US is 340B rebates at 23% and then traditional planned discounting in Italy. So I'll stand by prior comments on that. Thanks for the question.

Speaker Change: I'll hand over now to you. The second question.

Speaker Change: The second question.

Hank Fuchs: Right. And your second question, Cory, just remind me real fast?

Kristen Hubbard: for Boxergo with these with these indications where we see minimal exposure to to Medicare and I'll hand over now to to Hank for this second question.

Hank Fuchs: And I'll hand the microphone over now to Hank for the second question.

Cory Kasimov: Yes. And the difference or the similarities or differences between achondroplasia and hypochon in terms of level of market awareness of these patients and desire to seek treatment?

Hank Fuchs: Right. And your second question, Corey, just remind me real fast.

Corey: In terms of level of market awareness of these patients and desire to seek treatment.

Hank Fuchs: Right. And your second question, Corey, just remind me real fast.

Hank Fuchs: Yeah, and the differences or similarities or differences between achondroplasia and hypochondria in terms of the level of market awareness of these patients and their desire to seek treatment.

Hank Fuchs: Yes, hypochondroplasia doesn't occur as early in life for diagnostic purposes. So, it's underdiagnosed, and we're undertaking a lot of effort to increase its diagnosis. Having said that, we're really quite pleased with the rate of progress of the feeder study, and I'll give you more of an update about that. But as with a lot of genetic conditions, once a diagnosis--once the therapy is in place, then there's more diagnostic effort undertaken once there's more diagnostic efforts undertaken, there's more ambition to treat. During the development program, we're hoping to try to begin the process of increasing awareness and ultimately uptake in the hypochondroplasia population.

Speaker Change: <unk> doesn't. Occur early in life for diagnostic. Caustic purposes, so it's <unk> it's. It's under diagnosed and we're undertaking a lot of effort to increase. Having said that we're really quite pleased with the rate of progress. The theater study and I will give you more of an update about that but. As with a lot of genetic conditions once at diagnosis. Once a therapy is in place then theres more diagnostic effort undertaken whats theres more diagnostic effort undertaken there's more ambition to treat so during the development program. Hoping to try to begin the process of increasing awareness and ultimately uptake. Hypo cancer patient population.

Hank Fuchs: Yeah, and the differences or similarities or differences between achondroplasia and hypochondria in terms of level of market awareness of these patients and desire to seek treatment.

Speaker Change: Occur early in life for diagnostic. Caustic purposes, so it's <unk> it's. It's under diagnosed and we're undertaking a lot of effort to increase. Having said that we're really quite pleased with the rate of progress. The theater study and I will give you more of an update about that but. As with a lot of genetic conditions once at diagnosis. Once a therapy is in place then theres more diagnostic effort undertaken whats theres more diagnostic effort undertaken there's more ambition to treat so during the development program. Hoping to try to begin the process of increasing awareness and ultimately uptake. Hypo cancer patient population.

Speaker Change: Caustic purposes, so it's <unk> it's. It's under diagnosed and we're undertaking a lot of effort to increase. Having said that we're really quite pleased with the rate of progress. The theater study and I will give you more of an update about that but. As with a lot of genetic conditions once at diagnosis. Once a therapy is in place then theres more diagnostic effort undertaken whats theres more diagnostic effort undertaken there's more ambition to treat so during the development program. Hoping to try to begin the process of increasing awareness and ultimately uptake. Hypo cancer patient population.

Luca Issi: Got it. Thanks.

Hank Fuchs: Yeah, hypochondria doesn't occur as early in life for diagnostic purposes, so it's underdiagnosed, and we're undertaking a lot of effort to increase its diagnosis. Having said that, we're really quite pleased with the rate of progress of the feeder study, and I'll give you more of an update on that. But as with a lot of genetic conditions, once a diagnosis, once a therapy is in place Once there's more diagnostic effort undertaken, there's more ambition to treat. So during the development program, we're hoping to try to begin the process of increasing awareness and ultimately uptake in the hypochondroplasia population.

Speaker Change: It's under diagnosed and we're undertaking a lot of effort to increase. Having said that we're really quite pleased with the rate of progress. The theater study and I will give you more of an update about that but. As with a lot of genetic conditions once at diagnosis. Once a therapy is in place then theres more diagnostic effort undertaken whats theres more diagnostic effort undertaken there's more ambition to treat so during the development program. Hoping to try to begin the process of increasing awareness and ultimately uptake. Hypo cancer patient population.

Operator: I will now turn the call back to CEO Alexander Hardy for closing remarks.

Hank Fuchs: Yeah. Papachondroplasia doesn't, uh...

Speaker Change: Having said that we're really quite pleased with the rate of progress. The theater study and I will give you more of an update about that but. As with a lot of genetic conditions once at diagnosis. Once a therapy is in place then theres more diagnostic effort undertaken whats theres more diagnostic effort undertaken there's more ambition to treat so during the development program. Hoping to try to begin the process of increasing awareness and ultimately uptake. Hypo cancer patient population.

Hank Fuchs: Occur as early in life.

Alexander Hardy: Thank you very much, everybody. And thank you for joining our call for all your questions. We're very proud of the quarter and the strong growth both in revenue and profitability that BioMarin has achieved this quarter, and very excited about the outlook and raising our guidance on both the top and the bottom line. We're also very excited about the progress we've made so far this year with the priorities we set out in January. Reminder of those priorities. One was to accelerate and maximize the VOXZOGO opportunity. As you can see with these strong results, VOXZOGO is growing very, very well in just its first indication. And we're less than 20% penetrated in that. And we have a path forward with five additional indications. And we made progress so far this year in those, both in terms of regulatory feedback and progress in the development programs.

Speaker Change: The theater study and I will give you more of an update about that but. As with a lot of genetic conditions once at diagnosis. Once a therapy is in place then theres more diagnostic effort undertaken whats theres more diagnostic effort undertaken there's more ambition to treat so during the development program. Hoping to try to begin the process of increasing awareness and ultimately uptake. Hypo cancer patient population.

Speaker Change: For diagnostic purposes, so it's underdiagnosed and we're undertaking a lot of effort to increase its diagnosis.

Speaker Change: As with a lot of genetic conditions once at diagnosis. Once a therapy is in place then theres more diagnostic effort undertaken whats theres more diagnostic effort undertaken there's more ambition to treat so during the development program. Hoping to try to begin the process of increasing awareness and ultimately uptake. Hypo cancer patient population.

Speaker Change: Having said that, we're really quite pleased with the rate of progress.

Speaker Change: As with a lot of genetic conditions, once a diagnosis, once a therapy is in place,

Speaker Change: Hoping to try to begin the process of increasing awareness and ultimately uptake. Hypo cancer patient population.

Speaker Change: Then there's more diagnostic effort undertaken. Once there's more diagnostic effort undertaken, there's more ambition to treat.

Speaker Change: Hypo cancer patient population.

Cory Kasimov: Perfect. That's helpful. Thank you.

Speaker Change: During the development program, we're hoping to try to begin the process of increasing awareness and ultimately uptake in the hypochondroplasia population.

Operator: Our next question comes from Jessica Fye with JP Morgan. Please go ahead.

Speaker Change: Our next question comes from Jessica Fye with Jpmorgan. Please go ahead.

Jessica Fye: Hey guys, good afternoon. Thanks for taking my question. How are you thinking about the evolution of the competitive landscape in achondroplasia when we think about TransCon CNP and infigratinib potentially on the horizon? And maybe as a follow-up, would you ever consider evaluating VOXZOGO in combination with growth hormone in achondroplasia?

Operator: Perfect. That's very helpful, Hank.

Jessica Fye: How are you thinking about the evolution of the competitive landscape and a contemplation. When we think about transcon at AMC and integrate nib potentially on the horizon. And maybe as a follow up would you ever consider evaluating <unk> in combination with growth hormone and contemplate here. Thank you.

Speaker Change: Perfect. That's helpful, Hank. Thank you.

Operator: Our next question comes from Jessica Fye of J.P. Morgan. Please go ahead.

Operator: Thank you. Our next question comes from Jessica Fye with J.P. Morgan. Please go ahead. Hey guys, good afternoon. Thanks for taking my question. What are you thinking about the evolution?

Speaker Change: [inaudible]

Alexander Hardy: Secondly, to establish the ROCTAVIAN opportunity. Today, with this decision, we have clarity on a path forward, a position of ROCTAVIAN within the portfolio. We're excited by the signs we're seeing of progress. But we're also confident in our strategy, reducing the expenditure down to $60 million in 2025, and confident in our ability for this product to be profitable in that timeframe. Thirdly, as you can see so far this year, we've prioritized our R&D efforts. We focus on three programs that we're most excited about. And we've made consequential decisions around stopping programs that don't meet the high bar for innovation and value creation for shareholders that we have at BioMarin. And fourthly, we're really excited and proud of the progress we've made on increasing profitability faster than revenue. You can see that with our results this quarter and our increase in guidance.

Speaker Change: Our next question comes from Jessica Fye with JP Morgan. Please go ahead.

Speaker Change: When we think about transcon at AMC and integrate nib potentially on the horizon. And maybe as a follow up would you ever consider evaluating <unk> in combination with growth hormone and contemplate here. Thank you.

Jessica Fye: Hey guys, good afternoon. Thanks for taking my questions.

Speaker Change: And maybe as a follow up would you ever consider evaluating <unk> in combination with growth hormone and contemplate here. Thank you.

Jessica Fye: How are you thinking about the evolution of the competitive landscape in achondroplasia when we think about transconstancy and infogratinib potentially on the horizon? And maybe as a follow-up, would you ever consider evaluating doxogo in combination with growth hormone in achondroplasia? Thank you.

Alexander Hardy: Thanks very much for your question, Jessica. So overall, we feel very confident about our competitive profile in achondroplasia and in the subsequent indication. As you know, CNP pathway is amenable to all the five different indications that we're now pursuing after achondroplasia. And even with achondroplasia, we think with the movement in achondroplasia to treat the youngest infants, zero to five population, which is very, very part of the growth, but also very important in terms of optimal patient outcomes. The early start treatment, the greatest possible impact on the child's health outcomes as well as growth. So, we think that's a really compelling position in achondroplasia. The lead we have the safety profile of the durable efficacy, we feel very, very good about achondroplasia. And then obviously, in the subsequent find indications where we think we're going to be very competitive. With regard to your second question, I will hand it over to Hank to answer the possibility of the combination approach.

Speaker Change: Overall, we feel very confident about our competitive profile. Achondroplasia and ended in a subsequent indications as you know CMP pathway. It's amenable to all five different. The patients that we're now pursuing after achondroplasia and even with achondroplasia, we think. The movement in achondroplasia to treat. The youngest stemming from that. The zero to five population, which is very very part. Part of the growth. But also very important in terms of optimal patient outcomes. Stock treatment. The greatest possible impact on the child's health outcomes as well as growth. So we think that's a really compelling. Physician in Achondroplasia I believe we have. The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: Achondroplasia and ended in a subsequent indications as you know CMP pathway. It's amenable to all five different. The patients that we're now pursuing after achondroplasia and even with achondroplasia, we think. The movement in achondroplasia to treat. The youngest stemming from that. The zero to five population, which is very very part. Part of the growth. But also very important in terms of optimal patient outcomes. Stock treatment. The greatest possible impact on the child's health outcomes as well as growth. So we think that's a really compelling. Physician in Achondroplasia I believe we have. The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: Thanks very much for your question, Jessica. So overall, we feel very confident about our competitive profile in achondroplasia and in the subsequent indications, as you know.

Speaker Change: It's amenable to all five different. The patients that we're now pursuing after achondroplasia and even with achondroplasia, we think. The movement in achondroplasia to treat. The youngest stemming from that. The zero to five population, which is very very part. Part of the growth. But also very important in terms of optimal patient outcomes. Stock treatment. The greatest possible impact on the child's health outcomes as well as growth. So we think that's a really compelling. Physician in Achondroplasia I believe we have. The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: The patients that we're now pursuing after achondroplasia and even with achondroplasia, we think. The movement in achondroplasia to treat. The youngest stemming from that. The zero to five population, which is very very part. Part of the growth. But also very important in terms of optimal patient outcomes. Stock treatment. The greatest possible impact on the child's health outcomes as well as growth. So we think that's a really compelling. Physician in Achondroplasia I believe we have. The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: The movement in achondroplasia to treat. The youngest stemming from that. The zero to five population, which is very very part. Part of the growth. But also very important in terms of optimal patient outcomes. Stock treatment. The greatest possible impact on the child's health outcomes as well as growth. So we think that's a really compelling. Physician in Achondroplasia I believe we have. The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: The CMP pathway is amenable to all the five different indications that we're now pursuing after achondroplasia. And even with achondroplasia, we think with the movement in achondroplasia to treat the youngest infants,

Speaker Change: The youngest stemming from that. The zero to five population, which is very very part. Part of the growth. But also very important in terms of optimal patient outcomes. Stock treatment. The greatest possible impact on the child's health outcomes as well as growth. So we think that's a really compelling. Physician in Achondroplasia I believe we have. The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: The zero to five population, which is very very part. Part of the growth. But also very important in terms of optimal patient outcomes. Stock treatment. The greatest possible impact on the child's health outcomes as well as growth. So we think that's a really compelling. Physician in Achondroplasia I believe we have. The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: Part of the growth. But also very important in terms of optimal patient outcomes. Stock treatment. The greatest possible impact on the child's health outcomes as well as growth. So we think that's a really compelling. Physician in Achondroplasia I believe we have. The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: But also very important in terms of optimal patient outcomes. Stock treatment. The greatest possible impact on the child's health outcomes as well as growth. So we think that's a really compelling. Physician in Achondroplasia I believe we have. The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Alexander Hardy: So we're looking forward to seeing you all at Investor Day to share our outlook for the future of BioMarin. It's a very, very exciting perspective we're going to share. We have a lot of confidence in that corporate strategy. We're looking forward to seeing you all in New York, either live or online, and seeing our plans for BioMarin. With that, thank you very much for joining us. I wish you a good rest of your day.

Speaker Change: Stock treatment. The greatest possible impact on the child's health outcomes as well as growth. So we think that's a really compelling. Physician in Achondroplasia I believe we have. The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: The greatest possible impact on the child's health outcomes as well as growth. So we think that's a really compelling. Physician in Achondroplasia I believe we have. The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: The Zero to Five Population, which is very, very part of the growth.

Hank Fuchs: But also very important in terms of optimal patient outcomes, the earlier you start treatment, the greatest possible impact on the child's health outcomes as well as growth. So we think that's really compelling. This is a very good position in achondroplasia. The lead we have there, the safety profile, the durable efficacy, we feel very, very good about achondroplasia. And then, obviously, in the subsequent five indications where we think we're going to be very competitive. With regard to your second question. I will hand it over to Hank to answer the possibility of a combined approach. Yeah, thanks, Alexander.

Speaker Change: So we think that's a really compelling. Physician in Achondroplasia I believe we have. The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: But also very important in terms of optimal patient outcomes, the early you start treatment, the greatest possible impact on the child's health outcomes as well as growth. So we think that's a really compelling point.

Speaker Change: Physician in Achondroplasia I believe we have. The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: The safety profile of the durable efficacy, we feel very very good about. Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: Got it in place yet. And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: And then obviously in the subsequent five indications. We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: The lead we have there, the safety profile, the durable efficacy, we feel very, very good about achondroplasia. And then, obviously, in the subsequent five indications, where we think we're going to be very competitive. With regard to your second question.

Operator: This concludes today's conference. You may now disconnect.

Speaker Change: We think we're going to be very competitive. With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: With regard to your second question. I will hand, it over to Frank. To answer the possibility of a combination approaches.

Speaker Change: I will hand, it over to Frank. To answer the possibility of a combination approaches.

Frank: To answer the possibility of a combination approaches.

Hank Fuchs: Yeah, thanks, Jessica. VOXZOGO restores growth to about 90% of the average stature population. And if we have a strategy to even further optimize efficacy, it would be more focused on 333 by virtue of offering the three advantages would be potentially for superior efficacy, potentially for superior convenience. And as part of that superior convenience, the ability to use one drug, not two drugs. So, we think that CMP franchise ultimately can do it all.

Frank: Jessica. But some of the stores. Growth to about 90% of the average stature population. And if we have a strategy to even further optimize efficacy it would be more focused. 333 by virtue of operating the dual <unk>. Three advantages would be potentially. Potentially for superior efficacy potentially for superior convenience and. As part of that severity of disease, the ability to use one drug two drugs. So we think the CMP franchise ultimately can do it all.

Frank: But some of the stores. Growth to about 90% of the average stature population. And if we have a strategy to even further optimize efficacy it would be more focused. 333 by virtue of operating the dual <unk>. Three advantages would be potentially. Potentially for superior efficacy potentially for superior convenience and. As part of that severity of disease, the ability to use one drug two drugs. So we think the CMP franchise ultimately can do it all.

Speaker Change: I will hand it over to Hank to answer the possibility of a combination approach.

Frank: Growth to about 90% of the average stature population. And if we have a strategy to even further optimize efficacy it would be more focused. 333 by virtue of operating the dual <unk>. Three advantages would be potentially. Potentially for superior efficacy potentially for superior convenience and. As part of that severity of disease, the ability to use one drug two drugs. So we think the CMP franchise ultimately can do it all.

Hank Fuchs: Yeah, thanks, Alexander and Jessica. You know that Voxogo restores Growth to about 90% of the average stature population. And if we had a strategy to even further optimize efficacy, it would be more focused on 333 by virtue of offering the dual or the three advantages would be potentially for superior efficacy, potentially for superior convenience, and, [inaudible] Our next question comes from Paul Matteis with Stiefel. Please go ahead. Great, thanks so much for taking my question. As we think about the next leg of growth for BoxoGo, I

Frank: And if we have a strategy to even further optimize efficacy it would be more focused. 333 by virtue of operating the dual <unk>. Three advantages would be potentially. Potentially for superior efficacy potentially for superior convenience and. As part of that severity of disease, the ability to use one drug two drugs. So we think the CMP franchise ultimately can do it all.

Hank Fuchs: Yeah, thanks Alexander and Jessica. You know that VOXOGO restores...

Frank: 333 by virtue of operating the dual <unk>. Three advantages would be potentially. Potentially for superior efficacy potentially for superior convenience and. As part of that severity of disease, the ability to use one drug two drugs. So we think the CMP franchise ultimately can do it all.

Hank Fuchs: Growth to about 90% of the average stature population.

Frank: Three advantages would be potentially. Potentially for superior efficacy potentially for superior convenience and. As part of that severity of disease, the ability to use one drug two drugs. So we think the CMP franchise ultimately can do it all.

Hank Fuchs: And if we have a strategy to even...

Frank: Potentially for superior efficacy potentially for superior convenience and. As part of that severity of disease, the ability to use one drug two drugs. So we think the CMP franchise ultimately can do it all.

Hank Fuchs: Further optimized efficacy, it would be more focused on...

As part of that severity of disease, the ability to use one drug two drugs. So we think the CMP franchise ultimately can do it all.

Hank Fuchs: 333 by virtue of offering the dual or the three advantages would be potentially for superior efficacy potentially for superior convenience and as part of that superior convenience you know the ability to use one drug not two drugs

Jessica Fye: Thank you.

Operator: Our next question comes from Paul Matteis with Stifel. Please go ahead.

Hank Fuchs: So we think that CMP franchise ultimately can do it all.

Paul Matteis: Great. Thanks so much for taking my question. As you think about the next leg of growth for VOXZOGO, I was curious, in the untreated population, what percent are treated by a specialist today? And I say this because anecdotally, we've been hearing from some of the key opinion leaders that the incremental patient adds are coming from primary care referrals and that the next leg of growth here might be driven by diagnosis. So maybe you can give some context there and just speak to from a marketing effort perspective. What's the kind of incremental effort for the next leg--of the next 500 patients versus the last 500? Thanks so much.

As we think about the next leg of growth for Bob I was curious when you speak of population what percent are treated by our specialists today and I say this because anecdotally we've been hearing from some of the key opinion leaders that the incremental patient adds are coming from primary care referrals and that the next leg of growth here might be driven by diagnosis. So maybe you can. Give some context, there and just speak to from a marketing effort perspective, whats the kind of incremental effort for the next leg of the next 500 patients versus the last 500. Thanks so much.

Operator: Our next question comes from Paul Matteis with Stiefel. Please go ahead.

Speaker Change: Thank you.

Speaker Change: Our next question comes from Paul Matteis with Stiefel. Please go ahead.

Paul Matisse: Great, thanks so much for taking my question. As we think about the next leg of growth for VoxOgo, I was curious, in the untreated population, what percent are treated by a specialist today? And I say this because anecdotally, we've been hearing from some of the key opinion leaders that the incremental patient ads are coming from primary care referrals, and that the next leg of growth here might be driven by diagnosis. So maybe you can give some context there and just speak to from a marketing effort perspective, you know, what's the kind of incremental effort for the next leg of the next 500 patients versus the last 500?

Speaker Change: Give some context, there and just speak to from a marketing effort perspective, whats the kind of incremental effort for the next leg of the next 500 patients versus the last 500. Thanks so much.

Cristin Hubbard: Yes. Thanks, such for the question, Paul. I will say, I don't know the exact numbers the actual treaters per say that you're referring to, but I will say where our efforts are focused. So in the United States, for instance, where we believe there's going to be a large growth opportunity for us. This is really an area where we do see more decentralized care. And so, what I mean by that is you certainly have it treated by some geneticists in some offices and in fact, some pediatric endocrinologists as well. But what we're finding is a lot of these patients are also sitting with general pediatricians. And so what we're really focused on is ensuring that we have awareness around VOXZOGO in that general peed population and making sure that they know we're a local treater, namely in your pediatric endocrinology offices may be. So we're really trying to build those connection points between where we know those patients might be to those who are going to be treated.

Kristen Hubbard: Yeah, thanks so much for the question, Paul. I will say, I don't know the exact numbers of the actual treaters, per se, that you're referring to. But I will say where our efforts are focused. So in the United States, for instance, where we believe there to be a large growth opportunity before us, this is really an area where we do see more decentralized care. And so what I mean by that is that you certainly have it, you know, being treated by some geneticists in some offices and, in fact, some pediatric endocrinologists as well.

Speaker Change: Thanks so much.

Speaker Change: Yeah, thanks so much for the question, Paul. I will say, I don't know the exact numbers of the actual treaters per se that you're referring to, but I will say where our efforts are focused. So, in the United States, for instance, where we believe there to be a large growth opportunity before us,

Speaker Change: This is really an area, where we do see more decentralized care and so what I mean by that is that you certainly have it treated by some geneticists in some offices and in fact, some pediatric endocrinologists as well, but what we're finding is a lot of these patients are also sitting with general pediatricians and so what we're really focused on is ensuring that we have awareness around upsell, though in that general. <unk> population and making sure that they know where our local trigger namely in your pediatric endocrinology offices, maybe so we're really trying to build those connection points between where we know those patients might be to those who are going to be treated

Speaker Change: This is really an area where we do see more decentralized care and so what I mean by that is that you certainly have it you know treated by some geneticists in some offices and in fact some pediatric endocrinologists as well but what we're finding is a lot of these patients are also sitting with general pediatricians.

Speaker Change: <unk> population and making sure that they know where our local trigger namely in your pediatric endocrinology offices, maybe so we're really trying to build those connection points between where we know those patients might be to those who are going to be treated now in ex U S. We do see much more of a centralized care system, there where they're treated in centers that specialize in.

<unk> population and making sure that they know where our local trigger namely in your pediatric endocrinology offices, maybe so we're really trying to build those connection points between where we know those patients might be to those who are going to be treated

Kristen Hubbard: But what we're finding is that a lot of these patients are also sitting with the general pediatricians. And so what we're really focused on is ensuring that we have awareness around Voxelgo in that general pediatric population and making sure that they know where a local specialist, namely in your pediatric endocrinology offices, may be. So we're really trying to build those connection points between where we know those patients might be and those who are going to be treated.

Speaker Change: And so what we're really focused on is ensuring that we have awareness around VoxOgo in that general PEED population and making sure that they know where a local treater, namely in your pediatric endocrinology offices, may be.

Cristin Hubbard: Now in ex-U.S., we do see much more of a centralized care system there where they are treated in centers that specialize in this area and so in skeletal dysplasias. So of course, we've seen faster uptake in those areas where they--you know where those patients are and how to get them. And importantly, while we've seen really good penetration in those countries. There's still room to grow, and that's where we're focused as well. So, I'd say that our focus areas are really going to be around that referral pattern in the U.S. continuing our growth in those kind of highly penetrated strategic markets and importantly, opening up in new markets where we might have lower penetration or we--we've not yet opened up the market. So, focus on all those areas.

Kristen Hubbard: Now, in the ex-US, we do see much more of a centralized care system there, where they're treated in centers that specialize in this area. And so for skeletal dysplasias, so, of course, we've seen faster uptake in those areas where they know, you know, where those patients are and how to get them.

Speaker Change: And this area and so in skeletal dysplasia. Of course, we've seen faster uptake in those areas, where they you know where those patients on how to get them and importantly, while we've seen really good penetration those countries, there's still room to grow and that's where we're focused as well. So I'd say that our focus areas are really going to be around that referral pattern in the U S. Continuing our growth in those kind of highly penetrated strategic markets. And importantly, opening up in new markets, where we might have lower penetration or are we think we have not yet. Opened up the market so focused on all of those areas.

Speaker Change: So we're really trying to build those connection points between where we know those patients might be to those who are going to be treated.

Speaker Change: Of course, we've seen faster uptake in those areas, where they you know where those patients on how to get them and importantly, while we've seen really good penetration those countries, there's still room to grow and that's where we're focused as well. So I'd say that our focus areas are really going to be around that referral pattern in the U S. Continuing our growth in those kind of highly penetrated strategic markets. And importantly, opening up in new markets, where we might have lower penetration or are we think we have not yet. Opened up the market so focused on all of those areas.

Speaker Change: Now, in Ex-US, we do see much more of a centralized care.

Speaker Change: systems there where they're treated in centers that specialize in this area and so in skeletal dysplasias.

Speaker Change: So, of course, we've seen faster uptake in those areas where they, you know, you know where those patients are and how to get them.

Speaker Change: And importantly, while we've

Kristen Hubbard: And importantly, while we've seen really good penetration in those countries, there's still room to grow, and that's where we're focused as well. So I'd say that our focus areas are really going to be around that referral pattern in the US, continuing our growth in those kind of highly penetrated strategic markets, and importantly, opening up new markets where we might have lower penetration, or we see we've not yet opened up the market. So, focused on all of those areas.

Speaker Change: And importantly, opening up in new markets, where we might have lower penetration or are we think we have not yet. Opened up the market so focused on all of those areas.

Speaker Change: Seeing really good penetration in those countries, there's still room to grow and that's where we're focused as well.

Speaker Change: So I'd say that our focus areas are really going to be around that referral pattern in the U.S., continuing our growth in those kind of highly penetrated strategic markets, and importantly, opening up in new markets where we might have lower penetration or we've not yet opened up the markets. So focused in all of those areas.

Speaker Change: Opened up the market so focused on all of those areas.

Operator: Our next question comes from Joseph Schwartz with Leerink Partners. Please go ahead.

Joseph P. Schwartz: Thank you. I'll ask about a couple of your priority pipeline programs. First, on BMN 333, I was hoping you could give us some insight into the long-acting technology that's used there. And also help us envision the expected time to market for BMN 333. And then, what are you hoping to see for BMN 351 at the target dose or doses since it's moving behind several other next-gen next gen Exon skippers, it would be helpful to hear what you hope to see efficacy wise at different dose levels? Thank you.

Joseph Schwartz: Priority pipeline programs first on <unk> 333, I was hoping you could give us some insight into the long acting technology that's used there. So. Help us. Envision that you expect and time to market for <unk> 333, and then what are you hoping to see from <unk> $3 51 at the target dose or doses since it's moving behind several other nextgen exon skippers. It would be helpful to hear what you hope to see efficacy wise at different dose levels. Thank you.

Operator: Our next question comes from Joseph Schwartz with Lerink Partners. Please go ahead.

Speaker Change: Our next question comes from Joseph Schwartz with Lerink Partners. Please go ahead.

Joseph Schwartz: Thank you. I'll ask you about a couple of your priority pipeline programs. First, on BMN-333, I was hoping you could give us some insight into the long-acting technology that's used there and also help us envision the expected time to market for BMN-333. And then, what are you hoping to see for BMN-351 at the target dose or doses since it's moving behind several other next-gen exon skippers? It would be helpful to hear what you hope to see efficacy-wise at different dose levels. Thank you. Hi Joe.

Joseph Schwartz: So. Help us. Envision that you expect and time to market for <unk> 333, and then what are you hoping to see from <unk> $3 51 at the target dose or doses since it's moving behind several other nextgen exon skippers. It would be helpful to hear what you hope to see efficacy wise at different dose levels. Thank you.

Joseph Schwartz: Thank you. I'll ask about a couple of your priority pipeline programs. First, on BMN 333, I was hoping you could give us some insight

Speaker Change: Help us. Envision that you expect and time to market for <unk> 333, and then what are you hoping to see from <unk> $3 51 at the target dose or doses since it's moving behind several other nextgen exon skippers. It would be helpful to hear what you hope to see efficacy wise at different dose levels. Thank you.

Envision that you expect and time to market for <unk> 333, and then what are you hoping to see from <unk> $3 51 at the target dose or doses since it's moving behind several other nextgen exon skippers. It would be helpful to hear what you hope to see efficacy wise at different dose levels. Thank you.

Joseph Schwartz: into the long-acting technology that's used there.

Speaker Change: Also, help us.

Joseph Schwartz: Envision the expected time to market for...

Speaker Change: BMN 333 and then what are you hoping to see for BMN 351 at the target dose or doses?

Hank Fuchs: Hi, Joe. As far as the specifics about 333 in terms of design, we really tried to leverage a lot of safety and information that's already available on linking chemistry. I'll get into that in a little bit more detail at Investor Day. Obviously, one of the other critical aspects of the program is to ensure sustained exposure without a lot of thoroughly high exposure or dumping is sometimes is referred to in drive delivery circles. So, I'll get into a little bit more details, but really a big orientation around safety and consistency of exposure. On 351, acknowledging that there are a lot of competitors, it doesn't seem like anybody is really able to get to high levels of dystrophin, either by conjugating polymorphelino with CAGS, or with transferrin receptor antibodies. And so, what our strategy has been all along, is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre-mRNA processing.

Speaker Change: As far as specifics about $3 33. In terms of design, we really tried to leverage a lot. Safety. Information Thats already available on Lincoln chemistry, I'll get into that in a little bit more detail at. At Investor Day, obviously, one of the other critical aspects of the program is to ensure. Stained exposure without a lot of it. Early high exposure. We're dumping is sometimes referred to in drug delivery circles. So. Get into a little bit more details, but really our big orientation around safety inconsistency of exposure. On 351, acknowledging that there are a lot of competitors. It doesn't seem like anybody is really able to get. High levels of dystrophin. Either by conjugated Polymorph Lino. With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: Since it's moving behind several other next-gen exon skippers, it would be helpful to hear what you hope to see efficacy-wise at different dose levels. Thank you.

Speaker Change: In terms of design, we really tried to leverage a lot. Safety. Information Thats already available on Lincoln chemistry, I'll get into that in a little bit more detail at. At Investor Day, obviously, one of the other critical aspects of the program is to ensure. Stained exposure without a lot of it. Early high exposure. We're dumping is sometimes referred to in drug delivery circles. So. Get into a little bit more details, but really our big orientation around safety inconsistency of exposure. On 351, acknowledging that there are a lot of competitors. It doesn't seem like anybody is really able to get. High levels of dystrophin. Either by conjugated Polymorph Lino. With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Hank Fuchs: Hi Joe. As far as the specifics about 333, in terms of design, we really tried to leverage a lot of safety and information that's already available on Linking Chemistry. I'll get into that in a little bit more detail. And investor day, obviously, one of the other critical aspects of the program is to ensure sustained exposure without a lot of Thoroughly high exposure or dumping, as it is sometimes referred to in drug delivery circles.

Speaker Change: Safety. Information Thats already available on Lincoln chemistry, I'll get into that in a little bit more detail at. At Investor Day, obviously, one of the other critical aspects of the program is to ensure. Stained exposure without a lot of it. Early high exposure. We're dumping is sometimes referred to in drug delivery circles. So. Get into a little bit more details, but really our big orientation around safety inconsistency of exposure. On 351, acknowledging that there are a lot of competitors. It doesn't seem like anybody is really able to get. High levels of dystrophin. Either by conjugated Polymorph Lino. With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: Information Thats already available on Lincoln chemistry, I'll get into that in a little bit more detail at. At Investor Day, obviously, one of the other critical aspects of the program is to ensure. Stained exposure without a lot of it. Early high exposure. We're dumping is sometimes referred to in drug delivery circles. So. Get into a little bit more details, but really our big orientation around safety inconsistency of exposure. On 351, acknowledging that there are a lot of competitors. It doesn't seem like anybody is really able to get. High levels of dystrophin. Either by conjugated Polymorph Lino. With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: Hi, Joe. As far as the specifics about 333,

Speaker Change: In terms of design, we really tried to leverage

Speaker Change: At Investor Day, obviously, one of the other critical aspects of the program is to ensure. Stained exposure without a lot of it. Early high exposure. We're dumping is sometimes referred to in drug delivery circles. So. Get into a little bit more details, but really our big orientation around safety inconsistency of exposure. On 351, acknowledging that there are a lot of competitors. It doesn't seem like anybody is really able to get. High levels of dystrophin. Either by conjugated Polymorph Lino. With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: A lot of safety.

Speaker Change: Stained exposure without a lot of it. Early high exposure. We're dumping is sometimes referred to in drug delivery circles. So. Get into a little bit more details, but really our big orientation around safety inconsistency of exposure. On 351, acknowledging that there are a lot of competitors. It doesn't seem like anybody is really able to get. High levels of dystrophin. Either by conjugated Polymorph Lino. With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: Information that's already available on Linking Chemistry. I'll get into that in a little bit more detail.

Speaker Change: Early high exposure. We're dumping is sometimes referred to in drug delivery circles. So. Get into a little bit more details, but really our big orientation around safety inconsistency of exposure. On 351, acknowledging that there are a lot of competitors. It doesn't seem like anybody is really able to get. High levels of dystrophin. Either by conjugated Polymorph Lino. With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: We're dumping is sometimes referred to in drug delivery circles. So. Get into a little bit more details, but really our big orientation around safety inconsistency of exposure. On 351, acknowledging that there are a lot of competitors. It doesn't seem like anybody is really able to get. High levels of dystrophin. Either by conjugated Polymorph Lino. With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: and Investor Day, obviously one of the other.

Speaker Change: The critical aspects of the program is to ensure sustained exposure without a lot of early high exposure.

Speaker Change: Get into a little bit more details, but really our big orientation around safety inconsistency of exposure. On 351, acknowledging that there are a lot of competitors. It doesn't seem like anybody is really able to get. High levels of dystrophin. Either by conjugated Polymorph Lino. With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: On 351, acknowledging that there are a lot of competitors. It doesn't seem like anybody is really able to get. High levels of dystrophin. Either by conjugated Polymorph Lino. With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: or dumping as sometimes is referred to in drug delivery circles. So I'll get into a little bit more details but really a big orientation around safety and consistency of exposure.

Hank Fuchs: So, I'll get into a little bit more detail, but really a big orientation around safety and consistency of exposure. On 351, acknowledging that there are a lot of competitors, it doesn't seem like anybody is really able to get to high levels of dystrophin, either by conjugating polymorpholino with TAGs or with transferrin receptor antibodies.

Speaker Change: It doesn't seem like anybody is really able to get. High levels of dystrophin. Either by conjugated Polymorph Lino. With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: High levels of dystrophin. Either by conjugated Polymorph Lino. With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: Either by conjugated Polymorph Lino. With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: I'm 351 acknowledging that there are a lot of competitors.

Speaker Change: With. Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: Kids or with the transferrin receptor antibodies. So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: It doesn't seem like anybody is really able to get to high levels of dystrophin, either by conjugating polymorpholino

Speaker Change: So our. Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: Our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre mrna processing.

Speaker Change: with CADs or with transferrin receptor antibodies.

Hank Fuchs: The advantage of that approach has been twofold: One in preclinical miles. We can get dystrophin expression up to 40% in not just skeletal muscle, but also in high levels and diaphragm, high levels in heart. And the second is that we can do that at levels where we have confidence about the safety profile, given our experience with [inaudible]. I'll give more detailed updates on status of the program and expectations to when you're going to see data at Investor Day, but we're very encouraged by the progress that we're making in 351.

Speaker Change: And so, what our strategy has been all along is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre-mRNA processing. The advantage of that approach has been...

Speaker Change: The high levels and diaphragm high levels alright. And the second is that we can do that at levels, where we have confidence about the safety profile given our experience with drives the person I will give more detailed updates on status of the program and expectations for when Youre going to see data at Investor day, but we're very encouraged. By by the progress, we're making with 351.

Speaker Change: And the second is that we can do that at levels, where we have confidence about the safety profile given our experience with drives the person I will give more detailed updates on status of the program and expectations for when Youre going to see data at Investor day, but we're very encouraged. By by the progress, we're making with 351.

Speaker Change: 2-fold 1 in preclinical miles, we can get dystrophin expression up to 40% in not just skeletal muscle, but also in high levels in diaphragm, high levels in heart.

Hank Fuchs: And the second is that we can do that at levels where we have confidence about the safety profile given our experience with dry as a person. I'll give more detailed updates on the status of the program and expectations for when you're going to see data at investor day, but we're very encouraged by the progress that we're making with 351. Great, I'll speak to you in a minute. Our next question comes from Akash Tewari with Jeffreys. Please go ahead. Hi, this is Phoebe on behalf of Akash.

Speaker Change: And the second is that we can do that at levels where we have confidence about the safety profile given our experience with dry as a person.

Speaker Change: By by the progress, we're making with 351.

Speaker Change: I'll give more detailed updates on status of the program and expectations for when you're going to see data at Investor Day, but we're very encouraged by the progress that we're making with 351.

Joseph P. Schwartz: Great. [inaudible].

Operator: Our next question comes from Akash Tewari with Jefferies. Please go ahead.

Arash: Our next question comes from our cash <unk> with Jefferies. Please go ahead.

Akash Tewari: This is Phoebe on for Akash. So, on Biomarin 351 for DMD, how should we think about the translation from animal to human models when it comes to dystrophin expression? In mice models, you showed normal dystrophin levels as high as 98%. I guess, how well does that data translate to humans perfect normal dystrophin? And also for borrower success, what would you need in order to move forward? Thank you.

Operator: Our next question comes from Akash Tewari with Jeffreys. Please go ahead. Hi, this is Phoebe.

Speaker Change: Great. I'll stay tuned.

Speaker Change: Our next question comes from Akash Tewari with Jeffreys. Please go ahead.

Speaker Change: Hi, this is Phoebe on for Akash. Also on Biomarin 351 for DMD, how should we think about the translation from animal to human models when it comes to dystrophin expression? In mice models, you showed normal dystrophin levels as high as 98%. I guess, how well does that data translate to humans, percent normal dystrophin?

Speaker Change: And also from borrowers exhaust what would you need in order to move forward.

Hank Fuchs: Yes. Well, I think one of the challenges of translation is that there hasn't been a lot in humans that's really moved the needle in terms of dystrophin inspection. So, a little bit of that question is TBD. What we talked about at last year's R&D Day was based on our animal model where if we can achieve the tissue concentrations of 351 that we achieve [inaudible], we should be in a very much higher level of dystrophin expression and partly we brd it on a relatively unique animal model, which carries the human equivalent of the gene that is to be skipped. And so, we have a very fulsome assessment in vivo skipping potential. And we've got a lot of safety data, both from rodents on human primates and humans on our class of compounds. And so, we're optimistic, based on that, that we'll be able to see much more meaningful levels of dystrophin expression. And of course, it's really the near full-length dystrophin that motorizes the muscle, if you will, that will enable much greater function of the muscle. And so far, that hasn't been achieved. And that's what we're looking to achieve with 351.

Speaker Change: Yes. What are the challenges of translation is that there hasn't been. A lot in humans, that's really move the needle in terms of dystrophin expression. So a little bit of that question is TBD. What we talked about at last year's R&D day was based on our animal model work. If we can achieve the tissue concentrations of $3 51 that we achieved were drives a person. Should be in a very much higher levels of dystrophin expression and partly we base that on a relatively unique animal model, which carries the human equivalent. The gene that needs to be skipped. And so we have a very. Fulsome assessment in vivo. Skipping potential and we've got a lot of safety data, both from rodents nonhuman primates and humans. Our class of compounds. So we're optimistic. Based on that that will be able to see much more meaningful levels of dystrophin expression and of course, it's really. The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Speaker Change: What are the challenges of translation is that there hasn't been. A lot in humans, that's really move the needle in terms of dystrophin expression. So a little bit of that question is TBD. What we talked about at last year's R&D day was based on our animal model work. If we can achieve the tissue concentrations of $3 51 that we achieved were drives a person. Should be in a very much higher levels of dystrophin expression and partly we base that on a relatively unique animal model, which carries the human equivalent. The gene that needs to be skipped. And so we have a very. Fulsome assessment in vivo. Skipping potential and we've got a lot of safety data, both from rodents nonhuman primates and humans. Our class of compounds. So we're optimistic. Based on that that will be able to see much more meaningful levels of dystrophin expression and of course, it's really. The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Speaker Change: A lot in humans, that's really move the needle in terms of dystrophin expression. So a little bit of that question is TBD. What we talked about at last year's R&D day was based on our animal model work. If we can achieve the tissue concentrations of $3 51 that we achieved were drives a person. Should be in a very much higher levels of dystrophin expression and partly we base that on a relatively unique animal model, which carries the human equivalent. The gene that needs to be skipped. And so we have a very. Fulsome assessment in vivo. Skipping potential and we've got a lot of safety data, both from rodents nonhuman primates and humans. Our class of compounds. So we're optimistic. Based on that that will be able to see much more meaningful levels of dystrophin expression and of course, it's really. The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Speaker Change: And also, for Barra's success, what would you need in order to move forward? Thank you.

Operator: Yeah. Well, I think one of the challenges of translation is that there hasn't been a lot in humans that have really moved the needle in terms of dystrophin expression. So a little bit of that question is TBD.

Speaker Change: Yeah, well, I think one of the challenges of translation is that there hasn't been...

Speaker Change: What we talked about at last year's R&D day was based on our animal model work. If we can achieve the tissue concentrations of $3 51 that we achieved were drives a person. Should be in a very much higher levels of dystrophin expression and partly we base that on a relatively unique animal model, which carries the human equivalent. The gene that needs to be skipped. And so we have a very. Fulsome assessment in vivo. Skipping potential and we've got a lot of safety data, both from rodents nonhuman primates and humans. Our class of compounds. So we're optimistic. Based on that that will be able to see much more meaningful levels of dystrophin expression and of course, it's really. The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Speaker Change: A lot in humans that's really moved the needle in terms of

Speaker Change: If we can achieve the tissue concentrations of $3 51 that we achieved were drives a person. Should be in a very much higher levels of dystrophin expression and partly we base that on a relatively unique animal model, which carries the human equivalent. The gene that needs to be skipped. And so we have a very. Fulsome assessment in vivo. Skipping potential and we've got a lot of safety data, both from rodents nonhuman primates and humans. Our class of compounds. So we're optimistic. Based on that that will be able to see much more meaningful levels of dystrophin expression and of course, it's really. The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Hank Fuchs: What we talked about at last year's R&D day was based on our animal model work. If we can achieve the tissue concentrations of 351 that we achieve for dry as a person, we should be at a very much higher level of dystrophin expression. And partly, we base that on a relatively unique animal model that carries the human equivalent of the gene that is to be skipped. And so we have a very full assessment in vivo of skipping potential.

Speaker Change: Distrophin Expression. So a little bit of that question is TBD. What we talked about at last year's R&D day was based on our animal model work.

Speaker Change: Should be in a very much higher levels of dystrophin expression and partly we base that on a relatively unique animal model, which carries the human equivalent. The gene that needs to be skipped. And so we have a very. Fulsome assessment in vivo. Skipping potential and we've got a lot of safety data, both from rodents nonhuman primates and humans. Our class of compounds. So we're optimistic. Based on that that will be able to see much more meaningful levels of dystrophin expression and of course, it's really. The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Speaker Change: If we can achieve the tissue concentrations of 351 that we achieve for dry as a person, we should be in a very much higher level of dystrophin expression, and partly we base that on a relatively unique animal model,

Speaker Change: The gene that needs to be skipped. And so we have a very. Fulsome assessment in vivo. Skipping potential and we've got a lot of safety data, both from rodents nonhuman primates and humans. Our class of compounds. So we're optimistic. Based on that that will be able to see much more meaningful levels of dystrophin expression and of course, it's really. The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Speaker Change: And so we have a very. Fulsome assessment in vivo. Skipping potential and we've got a lot of safety data, both from rodents nonhuman primates and humans. Our class of compounds. So we're optimistic. Based on that that will be able to see much more meaningful levels of dystrophin expression and of course, it's really. The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Speaker Change: Fulsome assessment in vivo. Skipping potential and we've got a lot of safety data, both from rodents nonhuman primates and humans. Our class of compounds. So we're optimistic. Based on that that will be able to see much more meaningful levels of dystrophin expression and of course, it's really. The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Speaker Change: Skipping potential and we've got a lot of safety data, both from rodents nonhuman primates and humans. Our class of compounds. So we're optimistic. Based on that that will be able to see much more meaningful levels of dystrophin expression and of course, it's really. The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Speaker Change: which carries the human equivalent of the gene that is to be skipped.

Speaker Change: and so we have you know a very fulsome assessment in vivo of skipping potential and we've got a lot of safety data both

Speaker Change: Our class of compounds. So we're optimistic. Based on that that will be able to see much more meaningful levels of dystrophin expression and of course, it's really. The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Hank Fuchs: And we've got a lot of safety data, both from rodents, non-human primates, and humans, on our class of compounds. And so, based on that, we're optimistic that we'll be able to see much more meaningful levels of dystrophin expression. And of course, it's really the near full-length dystrophin that motorizes the muscle, if you will, that will enable much greater function of the muscle. And so far, that hasn't been achieved. And that's what we're looking to achieve with 351.

Speaker Change: So we're optimistic. Based on that that will be able to see much more meaningful levels of dystrophin expression and of course, it's really. The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Speaker Change: Based on that that will be able to see much more meaningful levels of dystrophin expression and of course, it's really. The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Speaker Change: from Rodenstein Human Primates and Humans on our class of compounds, and so we're optimistic.

Speaker Change: The near full length dystrophin that motor rises the muscle if you will that that will enable much greater function of the muscle. And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Speaker Change: Based on that, that we'll be able to see much more meaningful levels of dystrophin expression. And of course, it's really the near full length dystrophin that motorizes the muscle, if you will, that that will enable much greater function of the muscle. And so far, that hasn't been achieved. And that's what we're looking to achieve with 351.

Speaker Change: And so far that Hasnt been achieved and that's what we're looking to achieve with $3 51.

Operator: Our next question comes from Chris Raymond with Piper Sandler. Please go ahead.

Speaker Change: Our next question comes from Chris Raymond with Piper Sandler. Please go ahead.

Christopher J. Raymond: Thanks. Just maybe two questions. VOXZOGO in hypochondroplasia, just Hank, just listening to your comments on how hypochondroplasia is diagnosed maybe later in life. Just noticing that the pivotal study includes patients aged 3 to 18, should we expect maybe that you won't have to go back for earlier age in the label once approved? Or are--is the diagnostic journey be different among hypo and achondroplasia? And then also maybe another sort of pipeline question of 349, just noting that you had a competitor discontinue their oral AAT corrector program. I'm just wondering if you could call at any points of differentiation between 349 in that molecule. And just walk us through what gives you confidence here, comparatively?

Speaker Change: Maybe two questions on <unk> and Hypo contemplated Hank if you just listen to your comments on how. Hypochondriac wages diagnosed maybe later in life. Just noticing that the pivotal study includes patients age three to 18. Should we expect maybe. That you won't have to go back for earlier. H in the label once approved or is. Our. Is the diagnostic journey may be different among hypo in achondroplasia. And then also maybe another sort of a pipeline question on 349, just noting that you had. Competitor discontinue their oral EAP program, correct or program I'm, just wondering if you could call attention any points of differentiation between $3 49 and that molecule. Walk us through what gives you confidence here comparatively.

Operator: Our next question comes from Chris Raymond with Piper Sandler. Please go ahead.

Speaker Change: Our next question comes from Chris Raymond with Piper Sandler. Please go ahead.

Speaker Change #100: Hypochondriac wages diagnosed maybe later in life. Just noticing that the pivotal study includes patients age three to 18. Should we expect maybe. That you won't have to go back for earlier. H in the label once approved or is. Our. Is the diagnostic journey may be different among hypo in achondroplasia. And then also maybe another sort of a pipeline question on 349, just noting that you had. Competitor discontinue their oral EAP program, correct or program I'm, just wondering if you could call attention any points of differentiation between $3 49 and that molecule. Walk us through what gives you confidence here comparatively.

Chris Raymond: Thanks. Maybe I have two questions on Voxogo and hypochondroplasia. Hank, just listening to your comments on how hypochondroplasia is diagnosed maybe later in life. I'm just noticing that the pivotal study includes patients age 3 to 18. Should we expect maybe that you won't have to go back for earlier age in the label once approved, or is the diagnostic journey maybe different among hypo and achondroplasia? And then also maybe another sort of pipeline question at 349, just noting that you had a Competitor discontinued their oral AAT corrector program. Just wondering if you could call attention to any points of differentiation between 349 and that molecule.

Just noticing that the pivotal study includes patients age three to 18. Should we expect maybe. That you won't have to go back for earlier. H in the label once approved or is. Our. Is the diagnostic journey may be different among hypo in achondroplasia. And then also maybe another sort of a pipeline question on 349, just noting that you had. Competitor discontinue their oral EAP program, correct or program I'm, just wondering if you could call attention any points of differentiation between $3 49 and that molecule. Walk us through what gives you confidence here comparatively.

Chris Raymond: Thanks. Just maybe two questions on Voxogo and hypochondroplasia, just Hank, just listening to your comments on how hypochondroplasia is diagnosed maybe later in life,

Speaker Change #101: Should we expect maybe. That you won't have to go back for earlier. H in the label once approved or is. Our. Is the diagnostic journey may be different among hypo in achondroplasia. And then also maybe another sort of a pipeline question on 349, just noting that you had. Competitor discontinue their oral EAP program, correct or program I'm, just wondering if you could call attention any points of differentiation between $3 49 and that molecule. Walk us through what gives you confidence here comparatively.

Speaker Change #101: That you won't have to go back for earlier. H in the label once approved or is. Our. Is the diagnostic journey may be different among hypo in achondroplasia. And then also maybe another sort of a pipeline question on 349, just noting that you had. Competitor discontinue their oral EAP program, correct or program I'm, just wondering if you could call attention any points of differentiation between $3 49 and that molecule. Walk us through what gives you confidence here comparatively.

Speaker Change #102: H in the label once approved or is. Our. Is the diagnostic journey may be different among hypo in achondroplasia. And then also maybe another sort of a pipeline question on 349, just noting that you had. Competitor discontinue their oral EAP program, correct or program I'm, just wondering if you could call attention any points of differentiation between $3 49 and that molecule. Walk us through what gives you confidence here comparatively.

Speaker Change: Just noticing that the pivotal study includes patients age 3 to 18.

Speaker Change #101: Our. Is the diagnostic journey may be different among hypo in achondroplasia. And then also maybe another sort of a pipeline question on 349, just noting that you had. Competitor discontinue their oral EAP program, correct or program I'm, just wondering if you could call attention any points of differentiation between $3 49 and that molecule. Walk us through what gives you confidence here comparatively.

Speaker Change #103: Is the diagnostic journey may be different among hypo in achondroplasia. And then also maybe another sort of a pipeline question on 349, just noting that you had. Competitor discontinue their oral EAP program, correct or program I'm, just wondering if you could call attention any points of differentiation between $3 49 and that molecule. Walk us through what gives you confidence here comparatively.

Speaker Change: Should we expect maybe that you won't have to go back for earlier age in the label once approved or is the diagnostic journey maybe different among hypo and achondroplasia?

Speaker Change #104: And then also maybe another sort of a pipeline question on 349, just noting that you had. Competitor discontinue their oral EAP program, correct or program I'm, just wondering if you could call attention any points of differentiation between $3 49 and that molecule. Walk us through what gives you confidence here comparatively.

Speaker Change #105: Competitor discontinue their oral EAP program, correct or program I'm, just wondering if you could call attention any points of differentiation between $3 49 and that molecule. Walk us through what gives you confidence here comparatively.

Speaker Change: And then also maybe another sort of pipeline question on 349, just noting that you had a

Walk us through what gives you confidence here comparatively.

Hank Fuchs: Sure thing, Chris. The challenge oftentimes in genetic diseases is the diagnostic odyssey and the delay. And we hope that during our development program, we can overcome some of those barriers and start to teach our colleagues that early diagnosis is really the path to improve overall outcomes in genetic conditions. As to regulatory requirements for the under three population, that's a TBD. The one thing I'd say about that is, we've got a lot of wind at our back with health authorities around the world who recognize the safety and the efficacy of VOXZOGO in children under three years of age essentially approved from infancy around the world in achondroplasia, that's a very good platform to develop even further confidence of VOXZOGO in very young children. So, stay tuned for how the diagnostic Odyssey is going to unfold, how the clinical program will unfold and how the regulatory proceedings might unfold. As Alexander said earlier, we just got BRINEURA approved for the very young population. So, I think the agencies are now quite familiar with Biomarin and the strategy of obtaining earlier and earlier approvals and the rising confidence in the safety VOXZOGO efficacy can only help us.

Speaker Change: Competitor discontinued their oral AAT corrector program. I'm just wondering if you could call attention any points of differentiation between 349 and that molecule and just walk us through what gives you confidence here comparatively. Thank you.

Speaker Change #106: Sure. Thanks, Chris. The the challenge oftentimes in genetic diseases. The diagnostic Odyssey and the delay. And we. We hope that during our development program. We can't overcome. Some of those barriers and start to teach. Colleagues that early diagnosis is. Really the path to improved overall outcomes. In genetic conditions as to regulatory requirements for the under three population that's a TBD, but one thing I would say about that is we've got a lot of wind at our back with health authorities around the world, who recognize the safety and the efficacy of OXXO go in children under three years of agents. Essentially approved from infancy around the world in Achondroplasia. Very good platform to develop even further confidence. So go. In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Speaker Change #107: The the challenge oftentimes in genetic diseases. The diagnostic Odyssey and the delay. And we. We hope that during our development program. We can't overcome. Some of those barriers and start to teach. Colleagues that early diagnosis is. Really the path to improved overall outcomes. In genetic conditions as to regulatory requirements for the under three population that's a TBD, but one thing I would say about that is we've got a lot of wind at our back with health authorities around the world, who recognize the safety and the efficacy of OXXO go in children under three years of agents. Essentially approved from infancy around the world in Achondroplasia. Very good platform to develop even further confidence. So go. In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Hank Fuchs: This foot walk will give you confidence here, comparatively.

Speaker Change #108: The diagnostic Odyssey and the delay. And we. We hope that during our development program. We can't overcome. Some of those barriers and start to teach. Colleagues that early diagnosis is. Really the path to improved overall outcomes. In genetic conditions as to regulatory requirements for the under three population that's a TBD, but one thing I would say about that is we've got a lot of wind at our back with health authorities around the world, who recognize the safety and the efficacy of OXXO go in children under three years of agents. Essentially approved from infancy around the world in Achondroplasia. Very good platform to develop even further confidence. So go. In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Hank Fuchs: Sure thing, Chris. You know, the challenge with genetic diseases is often the diagnostic odyssey and the delay. And we hope that during our development program, we can overcome some of those barriers and start to teach our colleagues that early diagnosis is really the path to improved overall outcomes in genetic conditions. As to regulatory requirements for the under-three population, that's TBD. The one thing I'd say about that is we've got a lot of wind at our backs with health authorities around the world who recognize the safety and the efficacy of VoxOgo in children under three years of age. It's essentially approved for infancy around the world for achondroplasia. That's a very good platform to develop even further confidence in VoxOgo in very young children.

And we. We hope that during our development program. We can't overcome. Some of those barriers and start to teach. Colleagues that early diagnosis is. Really the path to improved overall outcomes. In genetic conditions as to regulatory requirements for the under three population that's a TBD, but one thing I would say about that is we've got a lot of wind at our back with health authorities around the world, who recognize the safety and the efficacy of OXXO go in children under three years of agents. Essentially approved from infancy around the world in Achondroplasia. Very good platform to develop even further confidence. So go. In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Speaker Change #108: We hope that during our development program. We can't overcome. Some of those barriers and start to teach. Colleagues that early diagnosis is. Really the path to improved overall outcomes. In genetic conditions as to regulatory requirements for the under three population that's a TBD, but one thing I would say about that is we've got a lot of wind at our back with health authorities around the world, who recognize the safety and the efficacy of OXXO go in children under three years of agents. Essentially approved from infancy around the world in Achondroplasia. Very good platform to develop even further confidence. So go. In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Speaker Change: Sure thing, Chris. You know, the challenge oftentimes in genetic diseases is...

Speaker Change #108: We can't overcome. Some of those barriers and start to teach. Colleagues that early diagnosis is. Really the path to improved overall outcomes. In genetic conditions as to regulatory requirements for the under three population that's a TBD, but one thing I would say about that is we've got a lot of wind at our back with health authorities around the world, who recognize the safety and the efficacy of OXXO go in children under three years of agents. Essentially approved from infancy around the world in Achondroplasia. Very good platform to develop even further confidence. So go. In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Some of those barriers and start to teach. Colleagues that early diagnosis is. Really the path to improved overall outcomes. In genetic conditions as to regulatory requirements for the under three population that's a TBD, but one thing I would say about that is we've got a lot of wind at our back with health authorities around the world, who recognize the safety and the efficacy of OXXO go in children under three years of agents. Essentially approved from infancy around the world in Achondroplasia. Very good platform to develop even further confidence. So go. In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Speaker Change: The Diagnostic Odyssey and the Delay, and we hope that during our development program

Speaker Change #108: Colleagues that early diagnosis is. Really the path to improved overall outcomes. In genetic conditions as to regulatory requirements for the under three population that's a TBD, but one thing I would say about that is we've got a lot of wind at our back with health authorities around the world, who recognize the safety and the efficacy of OXXO go in children under three years of agents. Essentially approved from infancy around the world in Achondroplasia. Very good platform to develop even further confidence. So go. In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Speaker Change #108: Really the path to improved overall outcomes. In genetic conditions as to regulatory requirements for the under three population that's a TBD, but one thing I would say about that is we've got a lot of wind at our back with health authorities around the world, who recognize the safety and the efficacy of OXXO go in children under three years of agents. Essentially approved from infancy around the world in Achondroplasia. Very good platform to develop even further confidence. So go. In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Speaker Change: We can overcome some of those barriers and start to teach our colleagues that early diagnosis is really the path to improved overall outcomes.

Speaker Change #109: In genetic conditions as to regulatory requirements for the under three population that's a TBD, but one thing I would say about that is we've got a lot of wind at our back with health authorities around the world, who recognize the safety and the efficacy of OXXO go in children under three years of agents. Essentially approved from infancy around the world in Achondroplasia. Very good platform to develop even further confidence. So go. In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Speaker Change: Ingenetic Conditions.

Speaker Change: As to regulatory requirements for the under-3 population, that's a TBD. The one thing I'd say about that is...

Speaker Change #109: Essentially approved from infancy around the world in Achondroplasia. Very good platform to develop even further confidence. So go. In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Speaker Change: We've got a lot of wind at our back with health authorities around the world who recognize the safety and the efficacy of VoxOgo in children under three years of age. It's essentially approved from infancy around the world in achondroplasia. That's a very good platform to develop even further confidence of...

Speaker Change #109: Very good platform to develop even further confidence. So go. In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Speaker Change #109: So go. In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Speaker Change #109: In very young children. So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Speaker Change #109: So stay tuned for how the diagnosis. Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Speaker Change #109: Obviously, it's going to unfold. <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Hank Fuchs: So, stay tuned for how the diagnostic odyssey is going to unfold, how the clinical program will unfold, and how the regulatory proceedings might unfold. You know, as Alexander said earlier, we just got Brinura approved for the very young population. So, I think the agencies are now quite familiar with Biomarin, and the strategy of obtaining earlier and earlier approvals and the rising confidence in the safety of VoxOgo and its efficacy can only help us.

Speaker Change #109: <unk> program, Orla unfold and how the regulatory proceedings. Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Speaker Change: Vatsoga in in very young children.

Speaker Change #109: Until it's Alexander said earlier, we just got we just got Britain Europe proved for the very young population. So I think the agencies are now quite familiar with biomarin in the strategy of obtaining earlier and earlier approvals. And the rising confidence in the safety of OXXO than that because you can only help us.

Alexander Hardy: So, stay tuned for how the diagnostic odyssey is going to unfold, how the clinical program will unfold, and how the regulatory proceedings might unfold. You know, as Alexander said earlier, we just got Brenura approved for the very young population, so I think the agencies are now quite familiar with Biomarin and the strategy of obtaining earlier and earlier approvals.

Speaker Change #110: And the rising confidence in the safety of OXXO than that because you can only help us.

Hank Fuchs: As regards to 349 and Vertex's most recently announced decision, I would just contrast two very different approaches that have been undertaken. Vertex, I think, was kind of shooting for the stars to do two very difficult things simultaneously. One was to restore antitryptic activity and the other was to prevent polymerization, which causes the liver disease. And they've had now a couple of bites at that apple. And have not so far succeeded and maybe they'll gain further traction, preclinically, don't know. What we on is a much more single-minded focus which is to stop one of the two big problems. And actually, it's the currently untreated problem than alpha-1 antitrypsin. As you know, there's replacement therapy for the lung disease, the loss of function mutations. The problem that we're addressing is a different molecular problem, which is the gain of function mutation, which causes the alpha-1 mutant proteins to polymerize in the liver, and that is currently unaddressed medically. Doing so has the advantage of--and what we've shown preclinically is that we can solubilize these polymers, increase their exclusions in the liver and then restore liver health as a consequence.

Speaker Change #111: I would just contrast, two very different approaches that have been undertaken vertex I think. Shooting for the stars to do. Two very difficult things simultaneously one was. To restore anti terrific activity and the other was to prevent polymerization, which causes the liver disease. And they've had a couple of bites of that Apple. Have not so far succeeded. And maybe they'll gain further traction pre clinically. No. What we've done is a much more single minded focus which is to stop one of the two big big problems and actually it's the currently untreated problem that Alpha one antitrypsin as you know there's replacement therapy for the lung disease the loss of function mutation. We're addressing a different molecular problem, which is the gain of function mutation, which causes the alpha one mutant proteins to polymerize in the liver and that is currently unaddressed medically doing so. The image. And what we've shown pre clinically is that we can utilize these polymers increase their expansions in the liver and then restore liver health as a consequence the.

Speaker Change: and the rising confidence in the safety of Oxhogan efficacy can only help us.

Hank Fuchs: As regards 349 and Virtex's most recently announced decision, I would just contrast two very different approaches that have been undertaken. Virtex, I think, was kind of shooting for the stars to do two very difficult things simultaneously.

Speaker Change #112: Shooting for the stars to do. Two very difficult things simultaneously one was. To restore anti terrific activity and the other was to prevent polymerization, which causes the liver disease. And they've had a couple of bites of that Apple. Have not so far succeeded. And maybe they'll gain further traction pre clinically. No. What we've done is a much more single minded focus which is to stop one of the two big big problems and actually it's the currently untreated problem that Alpha one antitrypsin as you know there's replacement therapy for the lung disease the loss of function mutation. We're addressing a different molecular problem, which is the gain of function mutation, which causes the alpha one mutant proteins to polymerize in the liver and that is currently unaddressed medically doing so. The image. And what we've shown pre clinically is that we can utilize these polymers increase their expansions in the liver and then restore liver health as a consequence the.

Speaker Change: As regards 349 and Vertex's most recently announced decision, I would just contrast two very different approaches that have been undertaken. Vertex, I think, was...

Speaker Change #112: Two very difficult things simultaneously one was. To restore anti terrific activity and the other was to prevent polymerization, which causes the liver disease. And they've had a couple of bites of that Apple. Have not so far succeeded. And maybe they'll gain further traction pre clinically. No. What we've done is a much more single minded focus which is to stop one of the two big big problems and actually it's the currently untreated problem that Alpha one antitrypsin as you know there's replacement therapy for the lung disease the loss of function mutation. We're addressing a different molecular problem, which is the gain of function mutation, which causes the alpha one mutant proteins to polymerize in the liver and that is currently unaddressed medically doing so. The image. And what we've shown pre clinically is that we can utilize these polymers increase their expansions in the liver and then restore liver health as a consequence the.

Speaker Change #112: To restore anti terrific activity and the other was to prevent polymerization, which causes the liver disease. And they've had a couple of bites of that Apple. Have not so far succeeded. And maybe they'll gain further traction pre clinically. No. What we've done is a much more single minded focus which is to stop one of the two big big problems and actually it's the currently untreated problem that Alpha one antitrypsin as you know there's replacement therapy for the lung disease the loss of function mutation. We're addressing a different molecular problem, which is the gain of function mutation, which causes the alpha one mutant proteins to polymerize in the liver and that is currently unaddressed medically doing so. The image. And what we've shown pre clinically is that we can utilize these polymers increase their expansions in the liver and then restore liver health as a consequence the.

Speaker Change: I'm kind of shooting for the stars to do two very difficult things simultaneously. One was...

Hank Fuchs: One was to restore antitrypic activity, and the other was to prevent polymerization, which causes liver disease. And they've had a couple of bites at that apple and have not so far succeeded, and maybe they'll gain further traction preclinically. I don't know.

Speaker Change #114: And they've had a couple of bites of that Apple. Have not so far succeeded. And maybe they'll gain further traction pre clinically. No. What we've done is a much more single minded focus which is to stop one of the two big big problems and actually it's the currently untreated problem that Alpha one antitrypsin as you know there's replacement therapy for the lung disease the loss of function mutation. We're addressing a different molecular problem, which is the gain of function mutation, which causes the alpha one mutant proteins to polymerize in the liver and that is currently unaddressed medically doing so. The image. And what we've shown pre clinically is that we can utilize these polymers increase their expansions in the liver and then restore liver health as a consequence the.

Speaker Change #114: Have not so far succeeded. And maybe they'll gain further traction pre clinically. No. What we've done is a much more single minded focus which is to stop one of the two big big problems and actually it's the currently untreated problem that Alpha one antitrypsin as you know there's replacement therapy for the lung disease the loss of function mutation. We're addressing a different molecular problem, which is the gain of function mutation, which causes the alpha one mutant proteins to polymerize in the liver and that is currently unaddressed medically doing so. The image. And what we've shown pre clinically is that we can utilize these polymers increase their expansions in the liver and then restore liver health as a consequence the.

Speaker Change: to restore antitrypic activity, and the other was to prevent polymerization, which causes the liver disease.

Speaker Change #114: And maybe they'll gain further traction pre clinically. No. What we've done is a much more single minded focus which is to stop one of the two big big problems and actually it's the currently untreated problem that Alpha one antitrypsin as you know there's replacement therapy for the lung disease the loss of function mutation. We're addressing a different molecular problem, which is the gain of function mutation, which causes the alpha one mutant proteins to polymerize in the liver and that is currently unaddressed medically doing so. The image. And what we've shown pre clinically is that we can utilize these polymers increase their expansions in the liver and then restore liver health as a consequence the.

Speaker Change #114: No. What we've done is a much more single minded focus which is to stop one of the two big big problems and actually it's the currently untreated problem that Alpha one antitrypsin as you know there's replacement therapy for the lung disease the loss of function mutation. We're addressing a different molecular problem, which is the gain of function mutation, which causes the alpha one mutant proteins to polymerize in the liver and that is currently unaddressed medically doing so. The image. And what we've shown pre clinically is that we can utilize these polymers increase their expansions in the liver and then restore liver health as a consequence the.

Speaker Change #114: What we've done is a much more single minded focus which is to stop one of the two big big problems and actually it's the currently untreated problem that Alpha one antitrypsin as you know there's replacement therapy for the lung disease the loss of function mutation. We're addressing a different molecular problem, which is the gain of function mutation, which causes the alpha one mutant proteins to polymerize in the liver and that is currently unaddressed medically doing so. The image. And what we've shown pre clinically is that we can utilize these polymers increase their expansions in the liver and then restore liver health as a consequence the.

Speaker Change: And they've had now a couple of bites of that apple and...

Speaker Change: have not so far succeeded.

Speaker Change: and maybe they'll gain further traction preclinically.

Hank Fuchs: What we've done is a much more single-minded focus, which is to stop one of the two big problems, and actually, it's the currently untreated problem in alpha-1 antitrypsin. As you know, there's replacement therapy for the lung disease, the loss of function mutation. The problem that we're addressing is a different molecular problem, which is the gain-of-function mutation, which causes the alpha-1 mutant proteins to polymerize in the liver, and that is currently unaddressed medically.

Speaker Change: I don't know. What we've done is a much more single-minded focus.

Speaker Change: which is to stop one of the two big, big problems and actually it's the currently untreated problem in Alpha-1 antitrypsin. As you know, there's replacement therapy for the lung disease, the loss of function in patients.

Speaker Change #114: We're addressing a different molecular problem, which is the gain of function mutation, which causes the alpha one mutant proteins to polymerize in the liver and that is currently unaddressed medically doing so. The image. And what we've shown pre clinically is that we can utilize these polymers increase their expansions in the liver and then restore liver health as a consequence the.

Speaker Change: The problem that we're addressing is a different...

Speaker Change #114: The image. And what we've shown pre clinically is that we can utilize these polymers increase their expansions in the liver and then restore liver health as a consequence the.

Speaker Change: Molecular problem, which is the gain-of-function mutation.

Speaker Change #114: And what we've shown pre clinically is that we can utilize these polymers increase their expansions in the liver and then restore liver health as a consequence the.

Speaker Change: which causes the Alpha-1 mutant proteins to polymerize.

Hank Fuchs: Doing so has the advantage of – and what we've shown preclinically is that we can solubilize these polymers, increase their excretion from the liver, and then restore liver health as a consequence. The related result of that is that in patients who have only one genocopy of the mutant protein, we can reduce the Z polymerization and leave the M protein unaffected and, therefore, preserve its antitryptic activity. The result of all of that in the competitive landscape is having an oral and, therefore, better titratable product and also a product that's potentially much more broadly deliverable. So we're very excited about the 349 program. Again, I'll update you further on the progress that we're making in InVEST today.

Speaker Change: and that is currently unaddressed medically. Doing so has the advantage of and what we've shown preclinically is that we can salubilize these polymers, increase their excretion from the liver and then restore liver health as a consequence.

Hank Fuchs: The related result of that is that in patients who have only one genome copy of the mutant protein that we can--but we can reduce the Z polymerization and leave the M protein unaffected and therefore, preserve its antitryptic activity. Result of all of that in the competitive landscape is having an oral and therefore, better titratable product and also a product that's potentially much more broadly deliverable. So, we're very excited about the 349 program. And again, I'll update you further on progress that we're making at Investor Day.

Speaker Change: The related result of that is that in patients who have only one

Speaker Change: Genocopy of the mutant protein that we can, but we can reduce the Z polymerization and leave the M protein unaffected and therefore preserve its antitryptic activity.

Speaker Change #114: Before better Titratable product and also a product that potentially much more broadly deliverable. So we're very excited about the 249 program and again I'll update you further on progress that we're making in the day.

Speaker Change: The result of all of that in the competitive landscape is having an oral and therefore better titratable product, and also a product that's potentially much more broadly deliverable. So we're very excited about the 349 program. And again, I'll update you further on progress that we're making in InVEST today.

Christopher J. Raymond: Thank you.

Operator: Our next question comes from Gena Wang with Barclays. Please go ahead.

Gena Wang: Thank you. I will have a few questions regarding ROCTAVIAN. Given your comments on 2025, is it fair to assume that you--actually your assumption is about 30, 40 patients that will be treated in 2025 in order to make breakeven? And then I have one question regarding the Germany. That 75% of the insurance, why--what's the reason they didn't cover now? And when do you expect to cover will be in place? In the U.S., the three patients that treated were these from three different sites? How many is now past contracting phase and in the process of payer discussion?

Operator: Our next question comes from Gina Wang with Barclays. Please go ahead. Thank you.

Gena Wang: Given your comments on 2025 is it fair to assume that you actually your assumption is about 30 40 patients that will be treated in 2025 in order to make. Breakeven and then. One question regarding the Germany that <unk>. 75% of the insurance of why what's the reason they didn't cover now and when do you expect to cover. Sure. We will be in place in the U S. The three patients treated with visa from three different sites, how many sites now past contracting phase and in the process of peer discussion.

Speaker Change: Thank you.

Speaker Change: Our next question comes from Gina Wang with Barclays. Please go ahead.

Gina Wang: Thank you. I will have a few questions regarding Roktavien. Given your comments on 2025,

Gina Wang: Given your comments on 2025, is it fair to assume that you actually assume about 30-40 patients that will be treated in 2025 in order to make a break even? And then I have one question regarding Germany, that 75% of the insurance, why, what's the reason they didn't cover it now, and when do you expect the cover to be in place? In the U.S., the three patients that were treated, were these from three different sites? How many sites have now passed the contracting phase and are in the process of a peer discussion?

Speaker Change #116: Breakeven and then. One question regarding the Germany that <unk>. 75% of the insurance of why what's the reason they didn't cover now and when do you expect to cover. Sure. We will be in place in the U S. The three patients treated with visa from three different sites, how many sites now past contracting phase and in the process of peer discussion.

Speaker Change #117: One question regarding the Germany that <unk>. 75% of the insurance of why what's the reason they didn't cover now and when do you expect to cover. Sure. We will be in place in the U S. The three patients treated with visa from three different sites, how many sites now past contracting phase and in the process of peer discussion.

Gina Wang: Is it fair to assume that you actually...

Speaker Change #118: 75% of the insurance of why what's the reason they didn't cover now and when do you expect to cover. Sure. We will be in place in the U S. The three patients treated with visa from three different sites, how many sites now past contracting phase and in the process of peer discussion.

Speaker Change: Your assumption is about a 30-40 patient that will be treated in 2025 in order to make a break even. And then I have one question regarding Germany, that 75% of the insurance — why?

Speaker Change #117: Sure. We will be in place in the U S. The three patients treated with visa from three different sites, how many sites now past contracting phase and in the process of peer discussion.

Speaker Change #119: We will be in place in the U S. The three patients treated with visa from three different sites, how many sites now past contracting phase and in the process of peer discussion.

Speaker Change: What's the reason they didn't cover now and when do you expect the cover will be in place? In the U.S., the three patients that treated, were these from three different sites? How many sites now passed contracting phase and in the process of a peer discussion?

Brian Mueller: Thanks, Gena. This is Brian. I'll start with your first question. We're not giving specific ROCTAVIAN patient or revenue guidance today. It was important to talk about the progress we're seeing in the launch that Cristin covered earlier. And then this cost envelope for next year and the goal of getting to profitability. So, framing it up in that way as demonstration and articulation of the strategy, so you can view that as, again, minimum level of revenues, if you will, but not getting into further specifics at this time.

Speaker Change #120: We're not giving specific brac JV in patient or revenue guidance today. It was important to talk about the progress we're seeing in the launch. Yes Christian covered earlier. And then. Cost envelope for next year and the goal of getting to profitability. So framing it up in that way as demonstration. Demonstration and articulation of the strategy. So you can view that as a minimum. A minimum level of revenues, if you will but not getting into further specifics at this time.

Brian Mueller: Thanks, Gina. This is Brian.

Speaker Change #120: It was important to talk about the progress we're seeing in the launch. Yes Christian covered earlier. And then. Cost envelope for next year and the goal of getting to profitability. So framing it up in that way as demonstration. Demonstration and articulation of the strategy. So you can view that as a minimum. A minimum level of revenues, if you will but not getting into further specifics at this time.

Brian Mueller: I'll start with your first question. We're not giving specific Roktavian patient or revenue guidance today. It was important to talk about the progress we're seeing in the launch, which Kristen covered earlier, and then this cost envelope for next year and the goal of getting to profitability. So framing it up in that way as, demonstration and articulation of the strategy, so you can do that as a, again, minimum level of revenues, if you will, but not getting into further specifics at this point. Yeah, and I'll take the second two questions. So with

Speaker Change: Thanks Gina. This is Brian . I'll start with your first question.

Christian: Yes Christian covered earlier. And then. Cost envelope for next year and the goal of getting to profitability. So framing it up in that way as demonstration. Demonstration and articulation of the strategy. So you can view that as a minimum. A minimum level of revenues, if you will but not getting into further specifics at this time.

Speaker Change: We're not giving specific Roctavian patient or revenue guidance today. It was important to talk about the progress we're seeing in the launch.

Speaker Change #121: And then. Cost envelope for next year and the goal of getting to profitability. So framing it up in that way as demonstration. Demonstration and articulation of the strategy. So you can view that as a minimum. A minimum level of revenues, if you will but not getting into further specifics at this time.

Speaker Change #122: Cost envelope for next year and the goal of getting to profitability. So framing it up in that way as demonstration. Demonstration and articulation of the strategy. So you can view that as a minimum. A minimum level of revenues, if you will but not getting into further specifics at this time.

Christian: So framing it up in that way as demonstration. Demonstration and articulation of the strategy. So you can view that as a minimum. A minimum level of revenues, if you will but not getting into further specifics at this time.

Speaker Change: that Kristen covered earlier, and then this cost envelope for next year and the goal of getting to profitability. So, framing it up in that way as...

Christian: Demonstration and articulation of the strategy. So you can view that as a minimum. A minimum level of revenues, if you will but not getting into further specifics at this time.

Christian: So you can view that as a minimum. A minimum level of revenues, if you will but not getting into further specifics at this time.

Christian: A minimum level of revenues, if you will but not getting into further specifics at this time.

Cristin Hubbard: Yes. And I'll take the second two questions. So, with regard to the subinsurers, I mean, it's impossible to give you an exact time of it, but I can say that we're making progress in these discussions that we're having. And really, we're just pushing through insurer by insurer and making sure that we are addressing the needs, which, of course, are going to be about risk carrying. On the U.S. side, you asked about the HTCs that have been treated. We've had those three patients treated. They were all in separate HTCs and we're actually geographically dispersed across the U.S.

Speaker Change: Demonstration and articulation of the strategy so you can do that as a again minimum level of revenues if you will but but not getting into further specifics at this time.

Speaker Change #123: It's impossible to give you an exact time of it but I can say that we're making progress in these in these discussions that we're having and really we're just pushing through insurer adventure and making sure that we are addressing the needs which of course are going to be about risk sharing. On the U S side, you asked about the HTC as that have been treated we've had those three patients treated they were all in separate HTC CS and were actually geographically disbursed across the U S.

Kristen Hubbard: Yeah, and I'll take the second and third questions. So, with regard to the subinsurers, I mean, it's, I, you know, it's, it's impossible to give you an exact time, but I can say that we're making progress in these, in these discussions that we're having, and really, we're just pushing through insurer by insurer and making sure that we are addressing the needs, which, of course, are going to be about risk sharing on the US side. Our next question comes from Kostas Biliouris of BMO Capital Markets. Please go ahead. Thanks for taking our question and congrats on a strong quarter. One question from us:

Speaker Change: Yeah, and I'll take the second two questions. So with regard to the signatures, I mean, it's I you know, it's it's impossible to give you an exact time of it, but I can say that we're making progress.

Speaker Change #124: On the U S side, you asked about the HTC as that have been treated we've had those three patients treated they were all in separate HTC CS and were actually geographically disbursed across the U S.

Speaker Change: In these discussions that we're having and really we're just

Speaker Change: On the US side, you asked about the HTCs that have been treated. We've had those three patients treated. They were all in separate HTCs and were actually geographically dispersed across the US.

Gena Wang: Thank you.

Operator: Our next question comes from Kostas Biliouris with BMO Capital Markets. Please go ahead.

Costi Polaris: Our next question comes from cost of Polaris with BMO capital markets. Please go ahead.

Kostas Biliouris: Thanks for taking our question and congrats on the strong quarter. One question from us on ROCTAVIAN, acknowledging that HEMGENIX in hemophilia B was approved six months before ROCTAVIAN in the U.S. and that the hemophilia depopulation is 4x to 5x smaller than the hemophilia A. How are you benchmarking ROCTAVIAN revenues versus HEMGENIX, which generated about $15 million to $30 million in the first half of 2024 based on our estimations. Thank you.

Operator: Our next question comes from Kostas Biliouris with BMO Capital Markets. Please go ahead. Thanks for taking our call.

Speaker Change: Thank you.

Speaker Change #128: Yes, or no leading that and Dan mixing hemophilia B was approved six months before Octavian in the U S and that the hemophilia B population is four to five times smaller on the hemophilia a how long do benchmarking avian revenues versus <unk>, which generated about 15 to 30. In the first half of 2024 based on our estimations. Thank you.

Speaker Change: Our next question comes from Konstantinos Biliouris with BMO Capital Markets. Please go ahead.

Costas Delores: Thanks for taking our question and congrats on the strong quarter. One question from us on Octavia.

Speaker Change: Acknowledging that hemogenics in hemophilia B was approved six months before Octavia in the US and that the hemophilia B population is four to five times smaller than

Speaker Change #129: In the first half of 2024 based on our estimations. Thank you.

Speaker Change: How are you benchmarking Octavian Revenues vs. Eugenics which generated about $15-$30 million in the first half of 2024 based on our estimations? Thank you.

Brian Mueller: Yes. Thanks for the question, Kostas. I'll jump in on that one, Brian, since I've been tracking the HEMGENIX launch along the way. There's different circumstances there between the two launches. Of course, there's differences as well, but we're not getting into launch comparisons, or benchmarking HEMGENIX, more of a qualitative estimate. Again, we are going through similar contracting in access challenges. So, there is a comparison to be made, but we don't do that quantitative level.

Speaker Change #130: Since I have been. Tracking the <unk>. Launch along the way. There's different circumstances there between the two launches of course, there's differences as well but. We're not getting into. Launch comparisons are benchmarking. More of a qualitative assessment again, we are going through similar contracting. Access challenges. So so there is a comparison to be made but we don't we don't do that quantitative level.

Speaker Change #132: Tracking the <unk>. Launch along the way. There's different circumstances there between the two launches of course, there's differences as well but. We're not getting into. Launch comparisons are benchmarking. More of a qualitative assessment again, we are going through similar contracting. Access challenges. So so there is a comparison to be made but we don't we don't do that quantitative level.

Speaker Change #133: Launch along the way. There's different circumstances there between the two launches of course, there's differences as well but. We're not getting into. Launch comparisons are benchmarking. More of a qualitative assessment again, we are going through similar contracting. Access challenges. So so there is a comparison to be made but we don't we don't do that quantitative level.

Speaker Change #134: There's different circumstances there between the two launches of course, there's differences as well but. We're not getting into. Launch comparisons are benchmarking. More of a qualitative assessment again, we are going through similar contracting. Access challenges. So so there is a comparison to be made but we don't we don't do that quantitative level.

Operator: Yeah, thanks for the question, Kostas. I'll jump in on that one since I've been tracking the hemogenics launch along the way. You know, there are different circumstances between the two launches. Of course, there are differences as well, but we're not getting into launch comparisons or benchmarking hemogenics. More of a qualitative assessment. Again, we are going through similar, you know, contracting and access challenges. So there is a comparison to be made, but we don't do that quantitatively.

Speaker Change: Yeah, thanks for the question, Kostas. I'll jump in on that one since I've been tracking the hemogenics launch along the way.

Speaker Change #133: We're not getting into. Launch comparisons are benchmarking. More of a qualitative assessment again, we are going through similar contracting. Access challenges. So so there is a comparison to be made but we don't we don't do that quantitative level.

Speaker Change #133: Launch comparisons are benchmarking. More of a qualitative assessment again, we are going through similar contracting. Access challenges. So so there is a comparison to be made but we don't we don't do that quantitative level.

Speaker Change #133: More of a qualitative assessment again, we are going through similar contracting. Access challenges. So so there is a comparison to be made but we don't we don't do that quantitative level.

Speaker Change: There are different circumstances there between the two launches. Of course, there's differences as well, but we're not getting into, you know...

Speaker Change #133: Access challenges. So so there is a comparison to be made but we don't we don't do that quantitative level.

Speaker Change: Launch comparisons or benchmarking, TeamGeneX, more of a qualitative assessment. Again, we are going through similar, you know, contracting and access challenges. So there is a comparison to be made, but we don't do it at that quantitative level.

Operator: Our next question comes from Mohit Bansal with Wells Fargo. Please go ahead.

Mohit Bansal: Great. Thank you for taking my question. Just want to reconcile some of the VOXZOGO numbers. So, Brian, if I understand correctly, the $20 million number for inventory is all inventory, right? That was one time. And if I assume that to be inventory, you added quite a lot of patients in this first half. I mean, I think the number was about 2,600 patients by the end of the year. Now you are at 3,500, that is 35% increase that is not reflecting in the revenue growth since the last year. So just wanted to understand if there is a pricing dynamic that is in play or geographical mix dynamic that we should know about?

Mohit Bansal: I just wanted to reconcile. Some of the walk through the numbers, though. So Bryan if I understand correctly that 20 million number. Lindsay is all inventory that was one. And if I assume that to be inventory. You added quite a lot of patients in this first half I mean, I think the number was about 2600 patients by the end of the year. No you added 3500 that is 35% increase that is not reflected in the revenue growth since the last year. So just wanted to understand if there is a pricing dynamic that is in play or geographical mix dynamic that we should know about.

Operator: Our next question comes from Mohit Bansal with Wells Fargo. Please go ahead.

Mohit Bansal: Some of the walk through the numbers, though. So Bryan if I understand correctly that 20 million number. Lindsay is all inventory that was one. And if I assume that to be inventory. You added quite a lot of patients in this first half I mean, I think the number was about 2600 patients by the end of the year. No you added 3500 that is 35% increase that is not reflected in the revenue growth since the last year. So just wanted to understand if there is a pricing dynamic that is in play or geographical mix dynamic that we should know about.

Mohit Bansal: So Bryan if I understand correctly that 20 million number. Lindsay is all inventory that was one. And if I assume that to be inventory. You added quite a lot of patients in this first half I mean, I think the number was about 2600 patients by the end of the year. No you added 3500 that is 35% increase that is not reflected in the revenue growth since the last year. So just wanted to understand if there is a pricing dynamic that is in play or geographical mix dynamic that we should know about.

Speaker Change: Our next question comes from Mohit Bansal with Wells Fargo. Please go ahead.

Operator: Great. Thank you for touching on my question.

Lindsay: Lindsay is all inventory that was one. And if I assume that to be inventory. You added quite a lot of patients in this first half I mean, I think the number was about 2600 patients by the end of the year. No you added 3500 that is 35% increase that is not reflected in the revenue growth since the last year. So just wanted to understand if there is a pricing dynamic that is in play or geographical mix dynamic that we should know about.

Mohit Bansal: Thank you for taking my question. Just wanted to reconcile some of the Voxelgo numbers. So, Brian , if I understand correctly, the 20 million number for inventory, it is all inventory, right? That was one time.

Speaker Change #137: And if I assume that to be inventory. You added quite a lot of patients in this first half I mean, I think the number was about 2600 patients by the end of the year. No you added 3500 that is 35% increase that is not reflected in the revenue growth since the last year. So just wanted to understand if there is a pricing dynamic that is in play or geographical mix dynamic that we should know about.

Speaker Change #138: You added quite a lot of patients in this first half I mean, I think the number was about 2600 patients by the end of the year. No you added 3500 that is 35% increase that is not reflected in the revenue growth since the last year. So just wanted to understand if there is a pricing dynamic that is in play or geographical mix dynamic that we should know about.

Mohit Bansal: Just wanted to reconcile some of the Voxelgo numbers. So, Brian, if I understand correctly, the 20 million number for inventory is all inventory, right? That was one time.

Speaker Change #139: No you added 3500 that is 35% increase that is not reflected in the revenue growth since the last year. So just wanted to understand if there is a pricing dynamic that is in play or geographical mix dynamic that we should know about.

Speaker Change: And if I assume that to be inventory...

Speaker Change #100: You added quite a lot of patients in this first half. I mean, I think the number was about 2,600 patients by the end of the year.

Speaker Change #101: Now you are at 3,500. That is a 35% increase. That is not reflecting in the revenue growth since the last year. So I just wanted you to understand if there is a pricing dynamic that is in play or a geographical mixed dynamic that we should know about.

Brian Mueller: Yes. Thanks for the question, Mohit. And there are going to be different dynamics from quarter-to-quarter in terms of the patient growth rates and revenue growth rates. I tried to address that a bit in the prepared remarks, but I might even point you to the last quarter, where there was a significant amount of functions adds, but you didn't see that necessarily a revenue growth. And this time around in the second quarter here because of that order timing that you noted, thanks to increased supply becoming available earlier, that dynamic flipped the other way around. And this time, revenues were ahead of patients a bit and recognized also that in the second half, we could see patient growth rates exceed revenue growth rates in either Q3 or Q4. But I mean, big picture, we're in the third year of this launch. We're still less than 20% penetrated with a long way to go globally. We're on track for VOXZOGO to exceed $1 billion in revenue. Yes, there's going to be differences in quarter-to-quarter timing of patients and orders we think those two will correlate over time. But we're just--we're out there executing quarter-to-quarter. There's going to be timing differences.

Speaker Change #140: There are going to be different. Dynamics from quarter to quarter in terms of the patient growth rates and revenue growth rates I tried to address that a bit in the prepared remarks, but I might even point you to last quarter, where there was a significant amount of patient adds but you didn't see that necessarily in the revenue growth and this time around in the second quarter here. <unk> because of that order timing that you noted thanks to the. Increased supply becoming available earlier. That dynamic flip the other way around in this time revenues were ahead of patients a bit and recognize also that in the second half we could see. Patient growth rates exceed revenue growth rates in either Q3, or Q4, but I mean big picture, we're in the third year. This launch we're still less than 20% penetrated with a long way to go globally. We're on track for <unk> to exceed $1 billion in revenue, yes, theres going to be differences in quarter to quarter timing of patients and orders. We think those two will correlate over time, but. We're just we're out there executing quarter over quarter, there is going to be timing differences.

Speaker Change #141: Dynamics from quarter to quarter in terms of the patient growth rates and revenue growth rates I tried to address that a bit in the prepared remarks, but I might even point you to last quarter, where there was a significant amount of patient adds but you didn't see that necessarily in the revenue growth and this time around in the second quarter here. <unk> because of that order timing that you noted thanks to the. Increased supply becoming available earlier. That dynamic flip the other way around in this time revenues were ahead of patients a bit and recognize also that in the second half we could see. Patient growth rates exceed revenue growth rates in either Q3, or Q4, but I mean big picture, we're in the third year. This launch we're still less than 20% penetrated with a long way to go globally. We're on track for <unk> to exceed $1 billion in revenue, yes, theres going to be differences in quarter to quarter timing of patients and orders. We think those two will correlate over time, but. We're just we're out there executing quarter over quarter, there is going to be timing differences.

Speaker Change #101: Yeah, thanks for the question, Mohit. And, you know, there are going to be different...

Brian Mueller: And if I assume that to be inventory, you added quite a lot of patients in this first half. I mean, I think the number was about 2,600 patients by the end of the year. Now you are at 3,500. That is a 35% increase. That is not reflected in the revenue growth since the last year. So just wanted to understand if there's a pricing dynamic that is in play or a geographical mix dynamic that we should know about.

Speaker Change #102: I'm going to go through the dynamics from quarter to quarter in terms of the patient growth rates and revenue growth rates. I tried to address that a bit in the prepared remarks, but I might even point you to last quarter where there was a significant amount of patient ads, but you didn't see that necessarily in the revenue growth. And this time around in the second quarter here, because of that order timing that you noted, thanks to the

Speaker Change #142: <unk> because of that order timing that you noted thanks to the. Increased supply becoming available earlier. That dynamic flip the other way around in this time revenues were ahead of patients a bit and recognize also that in the second half we could see. Patient growth rates exceed revenue growth rates in either Q3, or Q4, but I mean big picture, we're in the third year. This launch we're still less than 20% penetrated with a long way to go globally. We're on track for <unk> to exceed $1 billion in revenue, yes, theres going to be differences in quarter to quarter timing of patients and orders. We think those two will correlate over time, but. We're just we're out there executing quarter over quarter, there is going to be timing differences.

Speaker Change #142: Increased supply becoming available earlier. That dynamic flip the other way around in this time revenues were ahead of patients a bit and recognize also that in the second half we could see. Patient growth rates exceed revenue growth rates in either Q3, or Q4, but I mean big picture, we're in the third year. This launch we're still less than 20% penetrated with a long way to go globally. We're on track for <unk> to exceed $1 billion in revenue, yes, theres going to be differences in quarter to quarter timing of patients and orders. We think those two will correlate over time, but. We're just we're out there executing quarter over quarter, there is going to be timing differences.

Speaker Change #142: That dynamic flip the other way around in this time revenues were ahead of patients a bit and recognize also that in the second half we could see. Patient growth rates exceed revenue growth rates in either Q3, or Q4, but I mean big picture, we're in the third year. This launch we're still less than 20% penetrated with a long way to go globally. We're on track for <unk> to exceed $1 billion in revenue, yes, theres going to be differences in quarter to quarter timing of patients and orders. We think those two will correlate over time, but. We're just we're out there executing quarter over quarter, there is going to be timing differences.

Speaker Change #102: increased supply becoming available earlier-that that dynamic flipped the other way around and in this time you know revenues were ahead of patience a bit. And recognized also

Speaker Change #142: Patient growth rates exceed revenue growth rates in either Q3, or Q4, but I mean big picture, we're in the third year. This launch we're still less than 20% penetrated with a long way to go globally. We're on track for <unk> to exceed $1 billion in revenue, yes, theres going to be differences in quarter to quarter timing of patients and orders. We think those two will correlate over time, but. We're just we're out there executing quarter over quarter, there is going to be timing differences.

This launch we're still less than 20% penetrated with a long way to go globally. We're on track for <unk> to exceed $1 billion in revenue, yes, theres going to be differences in quarter to quarter timing of patients and orders. We think those two will correlate over time, but. We're just we're out there executing quarter over quarter, there is going to be timing differences.

Speaker Change #102: That in the second half, you know, we could see, you know, patient growth rates exceed revenue growth rates in either Q3 or Q4.

Speaker Change #102: But, I mean, big picture, we're in the third year of this launch, we're still less than 20% penetrated with a long way to go globally.

Speaker Change #102: We're on track for Roxogo to exceed a billion dollars in revenue. Yes, there's going to be differences in quarter-to-quarter timing of patients and orders. We think those two will correlate over time. But, you know, we're out there executing quarter-to-quarter. There's going to be timing differences.

Speaker Change #142: We're just we're out there executing quarter over quarter, there is going to be timing differences.

Brian Mueller: Yeah, thanks for the question, Mohit. And, you know, there are going to be different dynamics. I want to start with the dynamics from quarter to quarter in terms of patient growth rates and revenue growth rates. I tried to address that a bit in the prepared remarks, but I might even point you to last quarter, when there was a significant amount of patient ads, but you didn't necessarily see that in the revenue growth.

Mohit Bansal: Got it. Thank you.

Operator: Your next question comes from Olivia Brayer with Cantor Fitzgerald. Please go ahead.

Olivia Brayer: Hey, good afternoon, guys. Thank you for the question. As you consider profitability next year and just broader uptake of ROCTAVIAN, how are you thinking about sales contribution and breakdown across the three geographies that you're focused on? I'm just trying to get a sense for whether you expect the U.S. to really make up the majority of ROCTAVIAN sales going forward. Thank you.

Olivia Brayer: Consider profitability next year and just broader. Good how are you thinking about sales contribution and breakdown across the three geographies that you're focused on. I'm just trying to get a sense for whether you expect that you can really make up the majority of marketing and sales going forward. Thank you.

Speaker Change #103: Got it. Thank you.

Olivia Brayer: Good how are you thinking about sales contribution and breakdown across the three geographies that you're focused on. I'm just trying to get a sense for whether you expect that you can really make up the majority of marketing and sales going forward. Thank you.

Speaker Change #104: Your next question comes from Olivia Brayer with Kantor Fitzgerald. Please go ahead.

Brian Mueller: And this time around, in the second quarter here, because of that order timing that you noted, thanks to the increased supply becoming available earlier, that dynamic flipped the other way around. But, I mean, big picture, we're in the third year of this launch. We're still less than 20% penetrated, with a long way to go globally. But we're on track for Roxogo to exceed a billion dollars in revenue. Yes, there are going to be differences in the quarter to quarter timing of patients and orders.

Olivia Brayer: Hey, good afternoon, guys. Thank you for the question. As you consider profitability next year and just broader uptake of Octavian,

Speaker Change #145: I'm just trying to get a sense for whether you expect that you can really make up the majority of marketing and sales going forward. Thank you.

Olivia Brayer: How are you thinking about sales contribution and breakdown across the three geographies that you're focused on? I'm just trying to get a sense for whether you expect the U.S. to really make up the majority of Octavian sales going forward. Thank you.

Brian Mueller: Thanks Olivia, it's a good question. Noteworthy there just in terms of the U.S. versus ex-U.S. dynamic. The price of ROCTAVIAN revenue per patient on average in the U.S. is going to be higher. So, as they continue to make progress in the U.S. market, those patients are going to contribute more revenue. Again, we're focused on these three markets, both with the restructured cost envelope, as well as the tactics in this dedicated and focused business unit that will be working exclusively on ROCTAVIAN. It's going to allow the rest of the business to focus on the remainder of the portfolio. So, we're viewing this as the three markets together, not getting into further details of each of those individual markets at this time. But important to know that those three markets are the focus area and that will be the contributors to revenue in 2025.

Speaker Change #147: The price of <unk> TV net revenue per patient on average in the U S is going to be higher so. As we continue to make progress in the U S market those patients are going to contribute more to revenue. Again, we're focused on these three markets both with the restructured. Cost envelope as well as the tactics and the dedicated and focused business unit that we will be working exclusively on rock gaming and its going to allow the rest of the business to focus on the remainder of the portfolio. So were viewing this as the three markets together not getting into further details of each of those individual markets. At this time, but important to know that those three markets are the focus area and that will be the contributors to revenue in 2025.

Speaker Change #106: Yeah, thanks, Olivia. It's a good question. Noteworthy there just in terms of the US versus ex-US dynamic.

Speaker Change #147: As we continue to make progress in the U S market those patients are going to contribute more to revenue. Again, we're focused on these three markets both with the restructured. Cost envelope as well as the tactics and the dedicated and focused business unit that we will be working exclusively on rock gaming and its going to allow the rest of the business to focus on the remainder of the portfolio. So were viewing this as the three markets together not getting into further details of each of those individual markets. At this time, but important to know that those three markets are the focus area and that will be the contributors to revenue in 2025.

Speaker Change #147: Again, we're focused on these three markets both with the restructured. Cost envelope as well as the tactics and the dedicated and focused business unit that we will be working exclusively on rock gaming and its going to allow the rest of the business to focus on the remainder of the portfolio. So were viewing this as the three markets together not getting into further details of each of those individual markets. At this time, but important to know that those three markets are the focus area and that will be the contributors to revenue in 2025.

Speaker Change #107: The price of Roctavian, that revenue per patient on average in the U.S. is going to be higher, so as we continue to make progress in the U.S. market, those patients are going to contribute more revenue.

Speaker Change #147: Cost envelope as well as the tactics and the dedicated and focused business unit that we will be working exclusively on rock gaming and its going to allow the rest of the business to focus on the remainder of the portfolio. So were viewing this as the three markets together not getting into further details of each of those individual markets. At this time, but important to know that those three markets are the focus area and that will be the contributors to revenue in 2025.

Speaker Change #107: Again, we're focused on these three markets, both with the restructured cost envelope, as well as the tactics and this dedicated and focused business unit that

Speaker Change #147: So were viewing this as the three markets together not getting into further details of each of those individual markets. At this time, but important to know that those three markets are the focus area and that will be the contributors to revenue in 2025.

Speaker Change #107: We'll be working exclusively on Roktavian. It's going to allow the rest of the business to...

Speaker Change #107: And finally, I just want to say that we are looking at the three markets together, not getting into further details of each of those individual markets at this time, but important to know that those three markets are the focus area and that will be the contributors to revenue in 2025.

Operator: This will be our last question. Our last question comes from Luca Issi with RBC Capital. Please go ahead.

Luca <unk>: Our last question comes from Luca <unk> with RBC capital. Please go ahead.

Luca Issi: Oh, great. Thanks so much for taking my question. Congrats on the progress. Maybe two quick ones about Roc. You obviously prioritize in Italy, Germany and the U.S., but what's the plan for the other geographies where you still have rights? Is there any plan there to find a partner or try to monetize that in any capacity? Any color there, much appreciated. And maybe sticking to [inaudible]. Can you maybe just talk about net pricing in Italy versus the U.S. I believe last quarter, you reported $800,000 for the only patients treated in Italy versus today $7.4 million from three patients in the U.S. and two in Italy, that would imply net pricing of $1.9 million in the U.S. and $800,000 in Italy. Is that right? And you still how should we think about pricing in Germany? Thanks so much.

Operator: Got it. Thank you. Your next question comes from Olivia Brayer with Cantor Fitzgerald. Please go ahead. Hey, good afternoon, guys. Thank you for the question. Do you consider profitability?

Speaker Change #107: This will be our last question. Our last question comes from Luca Issi with RBC Capital. Please go ahead.

Luca Issi: All right, thanks so much for taking my questions, congrats on the progress. Let me do a quick one on Balrog.

Luca Issi: Maybe you're obviously prioritizing Italy, Germany, and the U.S., but what's the plan for the other geographies where you still have rights? Is there any plan there to find a partner or try to monetize that in any capacity? Any call there, much appreciated. And maybe sticking to Valrox, maybe just talk about net pricing.

Speaker Change #149: And then just sticking to the wall rock. So maybe just talk about net pricing, Italy versus the U S. I believe last quarter, you reported $800000. The only patients treated in Italy. Today is $7 four built off of three patients in the U S and two in Italy that would imply net pricing of $1 $9 million in the U S and eight. <unk> hundred thousand dollars in Italy is that right and if so how should we think about pricing in Germany. Thanks, So much.

Speaker Change #109: in Italy versus the U.S. I believe last quarter you reported $800,000 for the only patients treated in Italy.

Speaker Change #150: <unk> hundred thousand dollars in Italy is that right and if so how should we think about pricing in Germany. Thanks, So much.

Speaker Change #110: There's today 7.4 million of them, three patients in the U.S. and two in Italy.

Brian Mueller: Thanks, Luca. It's Brian. I'll take that one. So first of all, in terms of the focus, it's an important question because the key element behind the driver of the $60 million cost structure for next year is focusing entirely on supporting patients in those three markets as well as the long-term overall clinical and regulatory commitments that come with ROCTAVIAN that means that we are not investing in additional markets at this time. As we prove out and continue to gain traction and get confidence in ROCTAVIAN's overall progress over time, we will retain the right to make select investments that are value accretive to the assets over time. But important to note that it is just those three markets commercially at this time. And on pricing, I'll share, again, it's going to be dynamic over time with variations in the global pricing. I will share that the net revenue that we recognized for those five patients across the two regions where we had sales in Q2 was consistent with our expectations and prior communications. The largest gross to net item in the U.S. is 340 rebates at 23% and then traditional planned discounting in Italy. So, I'll stand by prior comments on that. Thanks for the question.

Speaker Change #110: That would imply net pricing of $1.9 million in the U.S. and $800,000 in Italy. Is that right? And if so, how should we think about pricing in Germany? Thanks so much.

Speaker Change #151: It's an important question because the key element behind the driver of the. $60 million cost structure for next year is focus and focusing entirely on supporting patients in those three markets as well as the long term overall clinical and regulatory commitment come with rock TV that means that we are not investing in additional markets at this time. As we prove out and continue to gain traction and get confidence and rock televisions overall progress over time, we will retain the right to make select investments that are value accretive to the assets that over time, but important to note that it is just those three markets commercially at this time and on pricing I'll share again, it's going to be dynamic over. Time with. Variations in the in the global pricing. I will share that the net revenue that we recognized for those five patients across the two regions, where we had sales in Q2. Was consistent with our expectations in prior communications the largest growth to net item in the U S is $3 40, the rebates that 23% and then traditional planned discounting in Italy. So. I'll standby prior comments on that.

Operator: Your next question comes from Olivia Brayer with Cantor Fitzgerald. Please go ahead. Hey, good afternoon, guys. Thank you for the question.

Brian Mueller: Yeah, thanks, Olivia. It's a good question.

$60 million cost structure for next year is focus and focusing entirely on supporting patients in those three markets as well as the long term overall clinical and regulatory commitment come with rock TV that means that we are not investing in additional markets at this time. As we prove out and continue to gain traction and get confidence and rock televisions overall progress over time, we will retain the right to make select investments that are value accretive to the assets that over time, but important to note that it is just those three markets commercially at this time and on pricing I'll share again, it's going to be dynamic over. Time with. Variations in the in the global pricing. I will share that the net revenue that we recognized for those five patients across the two regions, where we had sales in Q2. Was consistent with our expectations in prior communications the largest growth to net item in the U S is $3 40, the rebates that 23% and then traditional planned discounting in Italy. So. I'll standby prior comments on that.

Speaker Change #110: Thanks, Luca. This is Brian . I'll take that one. So first of all, in terms of the focus, the

Speaker Change #111: It's an important question because the key element behind the driver of the $60 million cost structure for next year is focusing entirely on supporting patients in those three markets, as well as the long-term overall clinical and regulatory commitments that come with Rotavian.

Speaker Change #151: As we prove out and continue to gain traction and get confidence and rock televisions overall progress over time, we will retain the right to make select investments that are value accretive to the assets that over time, but important to note that it is just those three markets commercially at this time and on pricing I'll share again, it's going to be dynamic over. Time with. Variations in the in the global pricing. I will share that the net revenue that we recognized for those five patients across the two regions, where we had sales in Q2. Was consistent with our expectations in prior communications the largest growth to net item in the U S is $3 40, the rebates that 23% and then traditional planned discounting in Italy. So. I'll standby prior comments on that.

Speaker Change #111: That means that we are not investing in additional markets at this time.

Brian Mueller: Noteworthy there, just in terms of the U.S. versus ex-U.S. dynamic, you know, the price of Roctavian net revenue per patient on average in the U.S. is going to be higher. So, as we continue to make progress in the U.S. market, those patients are going to contribute more revenue. Again, we're focused on these three markets, both with the restructured cost envelope, as well as the tactics and this dedicated and focused business unit that will be working exclusively on Roctavian.

Speaker Change #111: As we prove out and continue to gain traction and get confidence in Roctavian's overall progress over time.

Brian Mueller: It's going to allow the rest of the business to focus on the remainder of the portfolio. So we're viewing this as the three markets together, not getting into further details of each of those individual markets at this time, but it's important to know that those three markets are the focus areas and that will be the contributors to revenue in 2021.

Speaker Change #151: Time with. Variations in the in the global pricing. I will share that the net revenue that we recognized for those five patients across the two regions, where we had sales in Q2. Was consistent with our expectations in prior communications the largest growth to net item in the U S is $3 40, the rebates that 23% and then traditional planned discounting in Italy. So. I'll standby prior comments on that.

Speaker Change #111: We'll retain the right to make select investments that are value accretive to the asset over time, but important to note that it is just those three markets commercially at this time.

Speaker Change #151: Variations in the in the global pricing. I will share that the net revenue that we recognized for those five patients across the two regions, where we had sales in Q2. Was consistent with our expectations in prior communications the largest growth to net item in the U S is $3 40, the rebates that 23% and then traditional planned discounting in Italy. So. I'll standby prior comments on that.

Operator: This will be our last question, and it comes from Luca Issi with RBC Capital. Please go ahead.

Speaker Change #151: I will share that the net revenue that we recognized for those five patients across the two regions, where we had sales in Q2. Was consistent with our expectations in prior communications the largest growth to net item in the U S is $3 40, the rebates that 23% and then traditional planned discounting in Italy. So. I'll standby prior comments on that.

Luca Issi: All right, thanks so much for taking my questions. Congratulations on the progress. Let me do a quick one on Balrog.

Speaker Change #112: And on pricing, I'll share, again, it's going to be dynamic over time with, you know, variations in the global pricing. I will share that the net revenue that we recognized for those five patients

Speaker Change #151: Was consistent with our expectations in prior communications the largest growth to net item in the U S is $3 40, the rebates that 23% and then traditional planned discounting in Italy. So. I'll standby prior comments on that.

Luca Issi: And maybe sticking to Valrox, maybe just talk about net pricing in Italy versus the U.S. I believe last quarter you reported $800,000 for the only patients treated in Italy, versus $7.4 million for three patients in the U.S. and two in Italy. That would imply a net price of $1.9 million in the U.S. and $800,000 in Italy. Is that right? And if so, how should we think about pricing in Germany?

Speaker Change #111: across the two regions where we had sales in Q2.

Brian Mueller: Thanks, Luca. This is Brian. I'll take it from here.

Brian Mueller: Thanks, Luca. This is Brian.

Speaker Change #111: was consistent with our expectations in prior communications. The largest gross to net item in the US is 340B rebates at 23% and then traditional planned discounting in Italy.

Speaker Change #151: I'll standby prior comments on that.

Speaker Change #152: Thanks for the question.

Luca Issi: Got it, thanks.

Operator: I will now turn the call back to CEO Alexander Hardy for closing remarks.

Speaker Change #111: I'll stand by prior comments on that.

Alexander Hardy: Thank you very much, everybody, and thank you for joining our call for all your questions. We're very proud of the quarter and the strong growth, both in revenue and profitability that Biomarin has achieved this quarter and very excited about the outlook and raising our guidance. on both the top and the bottom line. And we're also very excited about the progress we've made so far this year with the priority prices we set out in January. A reminder of those priorities: One was to accelerate and maximize the VOXZOGO opportunities. You can see with these strong results. VOXZOGO is growing very, very well. In just its first indication and we're less than 20% penetrated in that, and we have a path forward with five additional indications, and we made progress so far this year in those, both in terms of regulatory feedback and progress in the development programs. Secondly, to establish the ROCTAVIAN opportunity. And today, with this decision, we have clarity on a path forward in addition of ROCTAVIAN within the portfolio. We're excited by the signs we're seeing of progress, but we're also confident in our strategy, reducing the expenditure down to $60 million in 2025 and confident in our ability for this product to be profitable in that time frame.

Speaker Change #113: Thanks to the coach.

Speaker Change #113: Got it, thanks.

Alexander Hardy: We're very proud of the quarter. The strong growth. And revenue and profitability that biomarin. <unk> this quarter. We're excited about the outlook and raising our guidance on both the top and bottom line. Also very excited about the progress we've made so far this year with the priorities we set out in January. A reminder, those priorities one was to accelerate and maximize the box hydro opportunities you can see with these strong results. Thanks, AGA is growing very very well in just its first indication and we're less than 20% penetrated in that and we have a path forward. Five additional indications and we made progress so far this year in those both in terms of regulatory feedback on progressing the development programs. Secondly to establish a octavian opportunity and today with this decision we have clarity on our path forward position abrupt Hadrian within the portfolio. We're excited by the signs were seeing of progress, but we're also confident in our strategy. Using the expenditure down to $60 million in 2025 confirmed our ability for this product to be profitable in that timeframe.

Speaker Change #113: I will now turn the call back to CEO Alexander Hardy for closing remarks.

Speaker Change #155: The strong growth. And revenue and profitability that biomarin. <unk> this quarter. We're excited about the outlook and raising our guidance on both the top and bottom line. Also very excited about the progress we've made so far this year with the priorities we set out in January. A reminder, those priorities one was to accelerate and maximize the box hydro opportunities you can see with these strong results. Thanks, AGA is growing very very well in just its first indication and we're less than 20% penetrated in that and we have a path forward. Five additional indications and we made progress so far this year in those both in terms of regulatory feedback on progressing the development programs. Secondly to establish a octavian opportunity and today with this decision we have clarity on our path forward position abrupt Hadrian within the portfolio. We're excited by the signs were seeing of progress, but we're also confident in our strategy. Using the expenditure down to $60 million in 2025 confirmed our ability for this product to be profitable in that timeframe.

Speaker Change #156: And revenue and profitability that biomarin. <unk> this quarter. We're excited about the outlook and raising our guidance on both the top and bottom line. Also very excited about the progress we've made so far this year with the priorities we set out in January. A reminder, those priorities one was to accelerate and maximize the box hydro opportunities you can see with these strong results. Thanks, AGA is growing very very well in just its first indication and we're less than 20% penetrated in that and we have a path forward. Five additional indications and we made progress so far this year in those both in terms of regulatory feedback on progressing the development programs. Secondly to establish a octavian opportunity and today with this decision we have clarity on our path forward position abrupt Hadrian within the portfolio. We're excited by the signs were seeing of progress, but we're also confident in our strategy. Using the expenditure down to $60 million in 2025 confirmed our ability for this product to be profitable in that timeframe.

Alexander Hardy: Thank you very much, everybody, and thank you for joining our call for all your questions.

Speaker Change #156: <unk> this quarter. We're excited about the outlook and raising our guidance on both the top and bottom line. Also very excited about the progress we've made so far this year with the priorities we set out in January. A reminder, those priorities one was to accelerate and maximize the box hydro opportunities you can see with these strong results. Thanks, AGA is growing very very well in just its first indication and we're less than 20% penetrated in that and we have a path forward. Five additional indications and we made progress so far this year in those both in terms of regulatory feedback on progressing the development programs. Secondly to establish a octavian opportunity and today with this decision we have clarity on our path forward position abrupt Hadrian within the portfolio. We're excited by the signs were seeing of progress, but we're also confident in our strategy. Using the expenditure down to $60 million in 2025 confirmed our ability for this product to be profitable in that timeframe.

Speaker Change #156: We're excited about the outlook and raising our guidance on both the top and bottom line. Also very excited about the progress we've made so far this year with the priorities we set out in January. A reminder, those priorities one was to accelerate and maximize the box hydro opportunities you can see with these strong results. Thanks, AGA is growing very very well in just its first indication and we're less than 20% penetrated in that and we have a path forward. Five additional indications and we made progress so far this year in those both in terms of regulatory feedback on progressing the development programs. Secondly to establish a octavian opportunity and today with this decision we have clarity on our path forward position abrupt Hadrian within the portfolio. We're excited by the signs were seeing of progress, but we're also confident in our strategy. Using the expenditure down to $60 million in 2025 confirmed our ability for this product to be profitable in that timeframe.

Alexander Hardy: We're very proud of the quarter and the strong growth.

Speaker Change #156: Also very excited about the progress we've made so far this year with the priorities we set out in January. A reminder, those priorities one was to accelerate and maximize the box hydro opportunities you can see with these strong results. Thanks, AGA is growing very very well in just its first indication and we're less than 20% penetrated in that and we have a path forward. Five additional indications and we made progress so far this year in those both in terms of regulatory feedback on progressing the development programs. Secondly to establish a octavian opportunity and today with this decision we have clarity on our path forward position abrupt Hadrian within the portfolio. We're excited by the signs were seeing of progress, but we're also confident in our strategy. Using the expenditure down to $60 million in 2025 confirmed our ability for this product to be profitable in that timeframe.

Alexander Hardy: Both in revenue and profitability that Biomarin has achieved this quarter and very excited about the outlook and raising our guidance on both the top and the bottom line.

Speaker Change #156: A reminder, those priorities one was to accelerate and maximize the box hydro opportunities you can see with these strong results. Thanks, AGA is growing very very well in just its first indication and we're less than 20% penetrated in that and we have a path forward. Five additional indications and we made progress so far this year in those both in terms of regulatory feedback on progressing the development programs. Secondly to establish a octavian opportunity and today with this decision we have clarity on our path forward position abrupt Hadrian within the portfolio. We're excited by the signs were seeing of progress, but we're also confident in our strategy. Using the expenditure down to $60 million in 2025 confirmed our ability for this product to be profitable in that timeframe.

Alexander Hardy: We're also very excited about the progress we've made so far this year with the priorities we set out in January .

Speaker Change #157: Thanks, AGA is growing very very well in just its first indication and we're less than 20% penetrated in that and we have a path forward. Five additional indications and we made progress so far this year in those both in terms of regulatory feedback on progressing the development programs. Secondly to establish a octavian opportunity and today with this decision we have clarity on our path forward position abrupt Hadrian within the portfolio. We're excited by the signs were seeing of progress, but we're also confident in our strategy. Using the expenditure down to $60 million in 2025 confirmed our ability for this product to be profitable in that timeframe.

Alexander Hardy: Reminder of those priorities. One was to accelerate and maximize the Boxergo opportunity. As you can see with these strong results, Boxergo is growing very, very well in just its first indication.

Speaker Change #157: Five additional indications and we made progress so far this year in those both in terms of regulatory feedback on progressing the development programs. Secondly to establish a octavian opportunity and today with this decision we have clarity on our path forward position abrupt Hadrian within the portfolio. We're excited by the signs were seeing of progress, but we're also confident in our strategy. Using the expenditure down to $60 million in 2025 confirmed our ability for this product to be profitable in that timeframe.

Alexander Hardy: and we're less than 20% penetrated in that and we have a path forward with five additional indications and we made progress so far this year in those both in terms of regulatory feedback and progress in the development programs.

Speaker Change #158: Secondly to establish a octavian opportunity and today with this decision we have clarity on our path forward position abrupt Hadrian within the portfolio. We're excited by the signs were seeing of progress, but we're also confident in our strategy. Using the expenditure down to $60 million in 2025 confirmed our ability for this product to be profitable in that timeframe.

Brian Mueller: I'll take that one. So first of all, in terms of focus, it's an important question, because the key element behind the driver of the $60 million cost structure for next year is focusing entirely on supporting patients in those three markets, as well as the long-term overall clinical and regulatory commitments that come with It means that we are not investing in additional markets at this time. As we prove out and continue to gain traction and gain confidence in Roktavian's overall progress over time, we'll retain the right to make select investments that are value accretive to the asset over time.

Speaker Change #158: We're excited by the signs were seeing of progress, but we're also confident in our strategy. Using the expenditure down to $60 million in 2025 confirmed our ability for this product to be profitable in that timeframe.

Alexander Hardy: Secondly, to establish the Roktavian opportunity, and today with this decision, we have clarity on a path forward, a position of Roktavian within the portfolio. We're excited by the signs we're seeing of progress, but we're also confident in our strategy.

Speaker Change #158: Using the expenditure down to $60 million in 2025 confirmed our ability for this product to be profitable in that timeframe.

Alexander Hardy: Thirdly, as you can see so far this year, we prioritized our R&D efforts. We focus on three programs that we're most excited about, and we've made consequential decisions around stopping programs that don't meet the high bar for innovation and value creation for shareholders that we have at Biomarin. And fourthly, we're really excited and proud of the progress we made on increasing profitability faster than revenue. You can see that with our results this quarter and our increase in guidance. So, we're looking forward to seeing you all at Investor Day to share our outlook for the future Biomarin. It's a very, very exciting perspective we're going to share. We have a lot of confidence in that corporate strategy. And we're looking forward to seeing you all in New York, either live or online and seeing our plans for Biomarin. And with that, thank you very much for joining us, and wish you a good rest of your day.

Alexander Hardy: Reducing the expenditure down to $60 million in 2025 and confident in our ability for this product to be profitable in that time frame.

Speaker Change #158: Made consequential decisions around stopping programs. The high bar for innovation and value creation for shareholders that we have at Biomarin. Fortunately, we are really excited and proud of the progress rebate on increasing profitability faster than revenue you can see that with our results this quarter. And our increase in guidance. So we're looking forward to seeing you all at Investor day to share. For the future Biomarin is a very very exciting. With respect to if we're going to share and we have a lot of confidence in that corporate strategy. <unk>. We're looking forward to seeing you all in New York, either live online and seeing our plans for Biomarin and with that thank you very much for joining us and wish you a good rest of your day.

Speaker Change #158: The high bar for innovation and value creation for shareholders that we have at Biomarin. Fortunately, we are really excited and proud of the progress rebate on increasing profitability faster than revenue you can see that with our results this quarter. And our increase in guidance. So we're looking forward to seeing you all at Investor day to share. For the future Biomarin is a very very exciting. With respect to if we're going to share and we have a lot of confidence in that corporate strategy. <unk>. We're looking forward to seeing you all in New York, either live online and seeing our plans for Biomarin and with that thank you very much for joining us and wish you a good rest of your day.

Alexander Hardy: Thirdly, as you can see so far this year, we prioritized our R&D efforts.

Alexander Hardy: We focus on three programs that we're most excited about. We've made consequential decisions around stopping programs that don't meet the high bar for innovation.

Speaker Change #158: Fortunately, we are really excited and proud of the progress rebate on increasing profitability faster than revenue you can see that with our results this quarter. And our increase in guidance. So we're looking forward to seeing you all at Investor day to share. For the future Biomarin is a very very exciting. With respect to if we're going to share and we have a lot of confidence in that corporate strategy. <unk>. We're looking forward to seeing you all in New York, either live online and seeing our plans for Biomarin and with that thank you very much for joining us and wish you a good rest of your day.

Alexander Hardy: and Value Creation for Shareholders that we have at Biomarin. And fourthly, we're really excited and proud of the progress we've made on increasing profitability faster than revenue. You can see that with our results this quarter and our increasing guidance.

Speaker Change #158: And our increase in guidance. So we're looking forward to seeing you all at Investor day to share. For the future Biomarin is a very very exciting. With respect to if we're going to share and we have a lot of confidence in that corporate strategy. <unk>. We're looking forward to seeing you all in New York, either live online and seeing our plans for Biomarin and with that thank you very much for joining us and wish you a good rest of your day.

Speaker Change #158: So we're looking forward to seeing you all at Investor day to share. For the future Biomarin is a very very exciting. With respect to if we're going to share and we have a lot of confidence in that corporate strategy. <unk>. We're looking forward to seeing you all in New York, either live online and seeing our plans for Biomarin and with that thank you very much for joining us and wish you a good rest of your day.

Speaker Change #158: For the future Biomarin is a very very exciting. With respect to if we're going to share and we have a lot of confidence in that corporate strategy. <unk>. We're looking forward to seeing you all in New York, either live online and seeing our plans for Biomarin and with that thank you very much for joining us and wish you a good rest of your day.

Speaker Change #158: With respect to if we're going to share and we have a lot of confidence in that corporate strategy. <unk>. We're looking forward to seeing you all in New York, either live online and seeing our plans for Biomarin and with that thank you very much for joining us and wish you a good rest of your day.

Speaker Change #158: <unk>. We're looking forward to seeing you all in New York, either live online and seeing our plans for Biomarin and with that thank you very much for joining us and wish you a good rest of your day.

Alexander Hardy: So we're looking forward to seeing you all at INVEST today to share our outlook for the future with Biomarin. It's a very, very exciting time.

Speaker Change #158: We're looking forward to seeing you all in New York, either live online and seeing our plans for Biomarin and with that thank you very much for joining us and wish you a good rest of your day.

Alexander Hardy: perspective we're going to share. We have a lot of confidence in that corporate strategy. And we're looking forward to seeing you all in New York, either live, online, and seeing our plans for Biomarin. And with that, thank you very much for joining us. I wish you a good rest of your day.

Operator: This concludes today's conference. You may now disconnect.

Speaker Change #114: This concludes today's conference. You may now disconnect.

Brian Mueller: But important to note that it is just those three markets commercially at this time. And on pricing, I'll share again, it's going to be dynamic over time with, you know, variations in global pricing. I will share that the net revenue that we recognized for those five patients, across the two regions where we had sales in Q2, was consistent with our expectations in prior communications. The largest gross-to-net item in the U.S. is 340B rebates at 23 percent. And then, you know, traditional planned discounting in Italy. I'll stand by my prior comments on that.

Speaker Change #114: ,

Q2 2024 Biomarin Pharmaceutical Inc Earnings Call

Request a DemoDemo

BMRN

Biomarin Pharmaceutical

Earnings