Q2 2026 Biomarin Pharmaceutical Inc Earnings Call

August 6, 2026

Operator 2: Good afternoon, welcome everyone to the BioMarin Pharmaceutical Q2 2026 conference call. Today's conference 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 would like to ask a question during this time, simply press the star key followed by the number one on your telephone keypad. If you would like to withdraw your question, press star one again. At this time, I would like to turn the conference over to Traci McCarty, Head of Investor Relations. Please-

Operator: Good afternoon, welcome everyone to the BioMarin Pharmaceutical Q2 2026 conference call. Today's conference 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 would like to ask a question during this time, simply press the star key followed by the number one on your telephone keypad. If you would like to withdraw your question, press star one again. At this time, I would like to turn the conference over to Traci McCarty, Head of Investor Relations. Please.

Speaker #1: session. background noise. If you would like to ask a question during this After the speaker's remarks, keypad. If you would like to withdraw your question, press star 1 again.

Speaker #1: At this time, I would like to turn the conference over to Traci McCarty, Head of Investor Relations. Please.

Speaker #2: Thank you, operator, and thank you all for joining us today. 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.

Traci McCarty: Thank you, operator, and thank you all for joining us today. 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, 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.

Speaker #2: 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.

Speaker #2: And those factors are 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.

Speaker #2: These non-GAAP measures should not be considered in isolation from, as substitutes for, or superior to, financial measures prepared in accordance with US GAAP. And you can find the related reconciliations to US GAAP in the earnings release.

Traci McCarty: 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. 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. Please note that our commentary on today's call will focus on non-GAAP financial measures unless otherwise indicated. Moving to slide three and introducing BioMarin's management team joining today's call. Alexander Hardy, Chief Executive Officer, Cristin Hubbard, Chief Commercial Officer, Greg Friberg, Chief R&D Officer, and Brian Mueller, Chief Financial Officer. I will now turn the call over to Alexander to provide our quarterly highlights. Alexander?

Speaker #2: And earnings presentation. Both of which are available in the Investor Relations section of our website. Please note that our commentary on today's call will focus on non-GAAP financial measures, unless otherwise indicated.

Speaker #2: Moving to slide 3, I’d like to introduce BioMarin's management team joining today’s call: Alexander Hardy, Chief Executive Officer; Cristin Hubbard, Chief Commercial Officer; Greg Friberg, Chief R&D Officer; and Brian Mueller, Chief Financial Officer.

Speaker #2: I will now turn the call over to Alexander to provide our quarterly highlights. Alexander.

Speaker #3: Thank you, Traci, and thank you all for joining us today. Starting on slide 5, BioMarin delivered a standout Q2, combining strong growth to nearly $1 billion in revenue with the successful close and integration of Amicus, while delivering on milestones that strengthen our leadership in genetic conditions.

Alexander Hardy: Thank you, Traci, and thank you all for joining us today. Starting on slide five, BioMarin delivered a standout second quarter, combining strong growth to nearly $1 billion in revenue, with the successful close and integration of Amicus, while delivering on milestones that strengthen our leadership in genetic conditions. Our strong performance demonstrates both the value creation of our portfolio and the continued execution of our commercial organization executing at scale, integrating meaningfully accretive assets, and continuing to innovate, enabling us to bring important medicines to people living with rare diseases as we enter an exciting new phase of growth. Second quarter highlights start with 20% year-over-year total revenue growth, accelerated by a more diversified portfolio, setting up an even stronger H2 of 2026 to be fueled by full Q3 and Q4 Galafold and Pombiliti and Opfolda contributions, and sustained demand across our other products.

Speaker #3: Our strong performance demonstrates both the value creation of our portfolio and the continued execution of our commercial organization—executing at scale, integrating meaningfully accretive assets, and continuing to innovate.

Speaker #3: Enabling us to bring important medicines to people living with rare diseases, as we enter an exciting new phase of growth. Q2 highlights start with 20% year-over-year total revenue growth.

Speaker #3: Accelerated by a more diversified portfolio, and setting up an even stronger second half of 2026 to be fueled by full third and fourth quarter gallifold and pombility and upfolder contributions.

Speaker #3: And sustained demand across our other products. Turning to Voxogo, double-digit revenue growth in both the US and international markets, led us to increase Voxogo's full-year guidance, putting it on a path to become BIOMARIN's first $1 billion product.

Alexander Hardy: Turning to VOXZOGO, double-digit revenue growth in both the U.S. and international markets led us to increase VOXZOGO's full year guidance, putting it on a path to become BioMarin's first $1 billion product. In its Q1 with a U.S. competitor, the continued revenue growth demonstrates our ability to defend VOXZOGO's leadership position. Building on this momentum, we are pleased to share that we have submitted our sNDA for the approval of VOXZOGO to treat hypochondroplasia based on strong pivotal data shared during the quarter. We will provide an update on the submission as part of our Q3 results. Turning to Amicus, as anticipated, when we announced the acquisition, this deal demonstrates that BioMarin can leverage our scale and capabilities to rapidly integrate high-growth assets to maximize value creation. Today, we provide estimated peak revenue for Galafold of $1.4 billion and for Pombiliti and Opfolda of $1.2 billion.

Speaker #3: In its first quarter, with a US competitor, the continued revenue growth demonstrates our ability to defend Voxogo's leadership position. Building on this momentum, we are pleased to share that we have submitted our SNDA for the approval of Voxogo to treat hypochondroplasia based on strong pivotal data shared during the quarter.

Speaker #3: We will provide an update on the submission as part of our third quarter results. Turning to Amicus, as anticipated, when we announced the acquisition, this deal demonstrates that BIOMARIN can leverage our scale and capabilities to rapidly integrate high-growth assets to maximize value creation.

Speaker #3: Today, we provide estimated peak revenue for gallifold of $1.4 billion and for pombility and upfolder of $1.2 billion. Together, these innovative therapies combined with significant cost synergies expected to reach approximately $220 million annual run rate in 2028 are anticipated to drive substantial EPS accretion and operating cash flow.

Alexander Hardy: Together, these innovative therapies, combined with significant cost synergies expected to reach approximately $220 million annual run rate in 2028, are anticipated to drive substantial EPS accretion and operating cash flow. Most importantly, we look forward to bringing Galafold and Pombiliti and Opfolda to more patients with Fabry and Pompe disease worldwide. Briefly on our pipeline, which continues to build momentum. We recently added BMN 820, formerly DMX-200, an exciting new late-stage pipeline opportunity resulting from the Amicus acquisition that Greg will expand upon in a moment. I am also impressed by the speed at which we submitted the sNDA for VOXZOGO for the treatment of hypochondroplasia. The speed of our submission reflects the benefits of our investments in AI capabilities and sets the new standard for how BioMarin will execute going forward. Together, these results tell a clear story.

Speaker #3: Most importantly, we look forward to bringing gallifold and pombility and upfolder to more patients with Fabre and Pompe disease worldwide. Briefly on our pipeline, which continues to build momentum.

Speaker #3: We recently added BMN 820, formerly DMX 200, an exciting new late-stage pipeline opportunity resulting from the Amicus acquisition that Greg will expand upon in a moment.

Speaker #3: I'm also impressed by the speed at which we submitted the SNDA for Voxogo for the treatment of hypochondroplasia. The speed of our submission reflects the benefits of our investments in AI capabilities and sets a new standard for how BioMarin will execute going forward.

Speaker #3: Together, these results tell a clear story. BIOMARIN is executing at scale, raising guidance, outperforming on the Amicus integration, with substantial combined peak revenue potential ahead.

Alexander Hardy: BioMarin is executing at scale, raising guidance, outperforming on the Amicus integration with substantial combined peak revenue potential ahead, and advancing pivotal pipeline data towards VOXZOGO's next indication. As we enter the H2 of 2026, BioMarin is stronger, more diversified, and better positioned than ever to lead in rare disease and deliver for patients worldwide. Now on slide six, the addition of Amicus transforms BioMarin's growth trajectory through the mid-2030s with a combined peak revenue potential of $2.6 billion from Galafold and Pombiliti and Opfolda, and significant cost synergies layering in along the way. We expect meaningful non-GAAP EPS accretion, expanding operating margins, and stronger operating cash flow, powering our next phase of growth. Moving now to slide seven and starting with Galafold, a growing product in a growing market.

Speaker #3: An advancing pivotal pipeline data towards Voxogo's next indication. As we enter the second half of 2026, BIOMARIN is stronger, more diversified, and better positioned than ever to lead in rare disease.

Speaker #3: And deliver for patients worldwide. Now on slide 6, the addition of Amicus, transforms BIOMARIN's growth trajectory through the mid-2030s. With a combined peak revenue potential of $2.6 billion from gallifold and pombility and upfolder, and significant cost synergies layering in along the way we expect meaningful non-GAAP EPS accretion, expanding operating margins, and stronger operating cash flow.

Speaker #3: Powering our next phase of growth. Moving now to slide 7, and starting with gallifold. A growing product in a growing market. From a $522 million base in 2025, we project peak revenue of $1.4 billion by the mid-2030s, supported by two complementary growth drivers.

Alexander Hardy: From a $522 million base in 2025, we project peak revenue of $1.4 billion by the mid-2030s, supported by two complementary growth drivers. First, we see significant opportunity to expand diagnosis and treatment. Leveraging BioMarin's proven diagnostic capabilities, our goal is to more than double the number of US patients treated with Galafold. We plan to do this through scaling AI-enabled patient identification initiatives, expanded genetic testing, newborn screening, and family cascade screening, helping more amenable patients access treatment earlier. Second, we see meaningful opportunity to expand market penetration globally. With Galafold already established in 40 countries, we intend to deepen penetration within existing markets while selectively expanding into new geographies, leveraging BioMarin's global commercial infrastructure to accelerate access and broaden reach. Together, these drivers are expected to support approximately 10% CAGR from 2027 to 2032.

Speaker #3: First, we see significant opportunity to expand diagnosis and treatment. Leveraging BIOMARIN's proven diagnostic capabilities. Our goal is to more than double the number of US patients treated with gallifold.

Speaker #3: We plan to do this through scaling AI-enabled patient identification initiatives, expanded genetic testing, newborn screening, and family cascade screening, helping more amenable patients access treatment earlier.

Speaker #3: Second, we see meaningful opportunity to expand market penetration globally. With gallifold already established in 40 countries, we intend to deepen penetration within existing markets, while selectively expanding into new geographies.

Speaker #3: Leveraging BIOMARIN's global commercial infrastructure, to accelerate access and broaden reach. Together, these drivers are expected to support approximately 10% CAGR from 2027 to 2032.

Speaker #3: Turning to slide 8, pombility and upfolder, is at an earlier stage in its commercial journey compared to gallifold. We estimate $1.2 billion in peak revenue by the mid to late 2030s, growing at a greater than or equal to 20% CAGR from 2027 to 2032.

Alexander Hardy: Turning to slide eight, Pombiliti and Opfolda is at an earlier stage in its commercial journey compared to Galafold. We estimate $1.2 billion in peak revenue by the mid to late 2030s, growing at a greater than or equal to 20% CAGR from 2027 to 2032. We expect growth to be driven primarily by increased patient switching and global market expansion, complemented by continued improvements in diagnosis and treatment rates. We believe switching will be supported by growing awareness amongst healthcare providers and patients of the expanded body of real-world evidence demonstrating the benefits of Pombiliti and Opfolda on disease outcomes. At the same time, we plan to leverage our diagnostic capabilities to identify and support treatment of additional eligible patients across our global footprint. These growth drivers are expected to be further strengthened by planned expansion into more than 20 additional markets over time. Turning to slide nine.

Speaker #3: We expect growth to be driven primarily by increased patient switching and global market expansion. Complemented by continued improvements in diagnosis and treatment rates. We believe switching will be supported by growing awareness amongst healthcare providers and patients, of the expanded body of real-world evidence demonstrating the benefits of pombility and upfolder on disease outcomes.

Speaker #3: At the same time, we plan to leverage our diagnostic capabilities to identify and support treatment of additional eligible patients across our global footprint. These growth drivers are expected to be further strengthened by planned expansion into more than 20 additional markets over time.

Speaker #3: Turning to slide 9, we are pleased with the significant cost synergies identified, which we expect to contribute to substantial EPS accretion beginning next year.

Alexander Hardy: We are pleased with the significant cost synergies identified, which we expect to contribute to substantial EPS accretion beginning next year. We anticipate approximately $220 million of synergies to be fully realized in 2028, representing a roughly 50% reduction from Amicus's 2025 non-GAAP operating expenses of $432 million. These synergies are weighted towards G&A, which makes up more than 70% of the total, with the remainder coming primarily from R&D. As planned, we retained Amicus's commercialization team to ensure patient continuity and supplement our global capabilities. These synergies, combined with peak revenue, align with the value creation we anticipated when we announced the deal last year and demonstrate our ability to successfully integrate large accretive assets that strengthen our growth profile. Turning to slide 10.

Speaker #3: We anticipate approximately $220 million of synergies to be fully realized in 2028, representing a roughly 50% reduction from Amicus's 2025 non-GAAP operating expenses of $432 million.

Speaker #3: These synergies are weighted towards GNA, which makes up more than 70% of the total, with the remainder coming primarily from R&D. As planned, we retained Amicus's commercialization team to ensure patient continuity, and supplement our global capabilities.

Speaker #3: These synergies combined with peak revenue align with the value creation we anticipated when we announced the deal last year, and demonstrate our ability to successfully integrate large accretive assets that strengthen our growth profile.

Speaker #3: Turning to slide 10, by accelerating our financial profile with the addition of accretive assets that benefit from our proven global expertise serving patients with genetic conditions, the Amicus acquisition sets the stage for our next phase of growth.

Alexander Hardy: By accelerating our financial profile with the addition of accretive assets that benefit from our proven global expertise serving patients with genetic conditions, the Amicus acquisition sets the stage for our next phase of growth. With peak targets of $1.4 billion for Galafold and $1.2 billion for Pombiliti and Opfolda, these revenues, combined with $220 million in anticipated annual cost synergies fully realized in 2028, support substantial expected non-GAAP diluted EPS accretion beginning in 2027 and a significant increase in operating cash flow. Galafold and Pombiliti and Opfolda combined are expected to reach over 60% non-GAAP operating margin by 2030. At the same time, we believe our rapid integration and growth plans will enable us to deleverage approximately one year sooner than initially communicated. This quarter reinforces what sets BioMarin apart.

Speaker #3: With peak targets of $1.4 billion for gallifold and $1.2 billion for pombility and upfolder, these revenues combined with $220 million in anticipated annual cost synergies fully realized in 2028, support substantial expected non-GAAP diluted EPS accretion beginning in 2027 and a significant increase in operating cash flow.

Speaker #3: Gallifold and pombility and upfolder combined are expected to reach over 60% non-GAAP operating margin by 2030. At the same time, we believe our rapid integration and growth plans will enable us to deleverage approximately one year sooner than initially communicated.

Speaker #3: This caught a reinforces what sets BIOMARIN apart. We are the leading rare disease company operating at scale, with a proven integration capability to maximize the value of high-growth assets.

Alexander Hardy: We are the leading rare disease company operating at scale, with a proven integration capability to maximize the value of high-growth assets. We look forward to updating you on our progress scaling Galafold and Pombiliti and Opfolda as we enter the next exciting phase of BioMarin's growth. I will now turn the call over to Cristin for the commercial update. Cristin.

Speaker #3: We look forward to updating on our progress, scaling gallifold and pombility and upfolder, as we enter the next exciting phase of BIOMARIN's growth. I will now turn the call over to Cristin for the commercial update.

Speaker #3: Cristin.

Speaker #2: Thank you, Alexander. The second quarter demonstrated the strength and growing diversity of our commercial portfolio. Now turning to slide 12, gallifold and pombility and upfolder are off to a strong start, as we move quickly to integrate following the April close.

Cristin Hubbard: Thank you, Alexander. The Q2 demonstrated the strength and growing diversity of our commercial portfolio. Now turning to slide 12. Galafold and Pombiliti and Opfolda are off to a strong start as we moved quickly to integrate following the April close. On a pro forma basis, Q2 revenue for Galafold grew approximately 10% year over year, and Pombiliti and Opfolda grew over 65%. Galafold delivered broad-based patient growth across both established and newer markets. This was driven largely by increased diagnosis and patient identification, including continued success with family cascade screening and expanding newborn screening programs, alongside ongoing gains in reimbursed access. For Pombiliti and Opfolda, we continue to add patients both in the US and in more recently launched geographies, supported by its differentiated clinical profile as we help more physicians better identify disease progression on prior therapies.

Speaker #2: On the pro forma basis, second quarter revenue for gallifold grew approximately 10% year over year, and pombility and upfolder grew over 65%. Gallifold delivered broad-based patient growth across both established and newer markets, this was driven largely by increased diagnosis and patient identification, including continued success with family cascade screening, and expanding newborn screening programs, alongside ongoing gains in reimbursed access.

Speaker #2: For pombility and upfolder, we continue to add patients, both in the United States and in more recently launched geographies, supported by its differentiated clinical profile, as we help more physicians better identify disease progression on prior therapies.

Speaker #2: Importantly, both brands maintained commercial momentum while we rapidly integrated, a testament to the focus our combined team has kept on patients and execution. These are the levers Alexander described, and the results to date reinforce our confidence in the long-term opportunity for both medicines.

Cristin Hubbard: Importantly, both brands maintained commercial momentum while we rapidly integrated, a testament to the focus our combined team has kept on patients and execution. These are the levers Alexander Hardy described, and the results to date reinforce our confidence in the long-term opportunity for both medicines. Turning to slide 13 and the broader Metabolic Conditions business unit, formerly known as Enzyme Therapies. With the addition of the Amicus medicines, we have changed the name of the business into the Metabolic Conditions business unit to better capture the breadth of our portfolio. Total Metabolic Conditions revenue was $695 million and grew 25% year over year, inclusive of Galafold and Pombiliti and Opfolda, and the number of patients on therapy grew across every one of our marketed Metabolic Conditions brands, both year over year and sequentially.

Speaker #2: Turning to slide 13 and the broader metabolic conditions business unit, formerly known as enzyme therapies, with the addition of the Amicus medicines, we have changed the name of the business into the Metabolic Conditions Business Unit to better capture the breadth of our portfolio.

Speaker #2: Total metabolic conditions revenue was $695 million and grew 25% year over year, inclusive of gallifold and pombility and upfolder, and the number of patients on therapy grew across every one of our marketed metabolic conditions brands, both year over year and sequentially.

Speaker #2: LNZ revenue grew 27% year over year on continued patient demand, while also benefiting from order timing in the US during the quarter. We were pleased to have recently received European approval to broaden the Palanziq label to adolescents ages 12 and older with PKU.

Cristin Hubbard: PALYNZIQ revenue grew 27% year over year on continued patient demand, while also benefiting from order timing in the US during the quarter. We were pleased to have recently received European approval to broaden the PALYNZIQ label to adolescents ages 12 and older with PKU. In the US, we have had a strong start in the adolescent age group, and the team is energized to have the opportunity to serve this population more broadly. Across the rest of the portfolio, revenue in any given quarter reflects the timing of large orders. In Q2, order timing was a headwind for VIMIZIM following a strong Q1, while it was a slight tailwind for NAGLAZYME ahead of an expected lighter Q3. Because of these dynamic shifts between quarters, our full-year metabolic conditions guidance remains the best indicator of expected underlying performance.

Speaker #2: In the US, we have had a strong start in the adolescent age group, and the team is energized to have the opportunity to serve this population more broadly.

Speaker #2: Across the rest of the portfolio, revenue in any given quarter reflects the timing of large orders. In the second quarter, order timing was a headwind for Vimazem following a strong first quarter, while it was a slight tailwind for Naglyzyme ahead of an expected lighter third quarter.

Speaker #2: Because of these dynamic shifts between quarters, our full-year metabolic conditions guidance remains the best indicator of expected underlying performance. Beneath that quarterly timing, the consistent signal is that patient demand continues to grow across our portfolio, both year over year and sequentially.

Cristin Hubbard: Beneath that quarterly timing, the consistent signal is that patient demand continues to grow across our portfolio both year over year and sequentially. Now turning to slide 14. VOXZOGO delivered 14% year-over-year revenue growth in Q2, driven by double-digit growth in the US and OUS markets. The number of children treated with VOXZOGO grew more than 20% year over year globally, and approximately three-quarters of VOXZOGO revenue was generated outside of the US. Notably, even in Q1 facing a US competitor, the number of children in the US treated with VOXZOGO increased year over year. We also saw continued traction in the under-two age group, which represented more than half of the new US patient starts in the quarter, reinforcing our position as the only approved treatment for children two and younger.

Speaker #2: Now turning to slide 14, Voxzogo delivered 14% year-over-year revenue growth in Q2, driven by double-digit growth in the U.S. and OUS markets.

Speaker #2: The number of children treated with Voxogo grew more than 20% year over year globally, and approximately three quarters of Voxogo revenue was generated outside of the US.

Speaker #2: Notably, even in the first quarter facing a US competitor, the number of children in the US treated with Voxogo increased year over year. We also saw continued traction in the under-2 age group, which represented more than half of the new US patient starts in the quarter, reinforcing our position as the only approved treatment for children 2 and younger.

Speaker #2: As expected, we did see switching to the competitor product since it was approved in February of this year. Since the competitor's launch, approximately 90% of the US children treated with Voxogo remained on therapy, as of the end of July, based on information available to us.

Cristin Hubbard: As expected, we did see switching to the competitor product since it was approved in February 2024. Since the competitor's launch, approximately 90% of the US children treated with VOXZOGO remained on therapy as of the end of July, based on information available to us. That reflects the continued confidence physicians and families place in VOXZOGO's evidence base. Internationally, momentum remains strong across both established and newer markets, and we expect both patient additions and order timing to drive higher total VOXZOGO revenue in H2 2026 compared to H1. We are confident in VOXZOGO's durability, built on a growing evidence base and the experience of the thousands of children treated to date and their caregivers. That durability is further reinforced by our exclusive ability to treat patients from birth worldwide.

Speaker #2: That reflects the continued confidence physicians and families place in Voxogo's evidence base. Internationally, momentum remains strong across both established and newer markets, and we expect both patient additions and order timing to drive higher total Voxogo revenue in the second half of 2026, compared to the first half.

Speaker #2: We're confident in Voxogo's durability, built on a growing evidence base, and the experience of the thousands of children treated to date and their caregivers.

Speaker #2: That durability is further reinforced by our exclusive ability to treat patients from birth worldwide. Our ambition is to remain the leader in skeletal conditions through competitor launches in the near term, supported by Voxogo's anticipated launch for hypochondroplasia in 2027, and the potential of BMN333 should data be supportive.

Cristin Hubbard: Our ambition is to remain the leader in skeletal conditions through competitor launches in the near term, supported by VOXZOGO's anticipated launch for hypochondroplasia in 2027 and the potential of BMN 333 should data be supportive. Stepping back, VOXZOGO is on track to become BioMarin's first $1 billion product. Galafold and Pombiliti and Opfolda are on the path to join it, each carrying peak revenue potential well above $1 billion, and VIMIZIM is expected to also reach the $1 billion mark over time. Together, they are a clear sign that we can take innovative, genetically targeted medicines and expand their reach worldwide. With that, I'll turn it over to Greg.

Speaker #2: Stepping back, Voxogo was on track to become BIOMARIN's first $1 billion product. Gallifold and pombility and upfolder are on the path to join it, each carrying peak revenue potential well above $1 billion and Vimazem is expected to also reach the $1 billion mark over time.

Speaker #2: Together, they are a clear sign that we can take innovative, genetically targeted medicines and expand their reach worldwide. With that, I'll turn it over to Greg.

Speaker #3: Thank you, Cristin. The second quarter was a productive period for our pipeline, with meaningful progress across our slide 16, you can see that we have had a lot of positive news over the last few months.

Greg Friberg: Thank you, Cristin. The second quarter was a productive period for our pipeline with meaningful progress across our portfolio. Turning to slide 16, you can see that we have had a lot of positive news over the last few months. I am particularly pleased to highlight our very recent submission of the supplemental sNDA for VOXZOGO for the treatment of hypochondroplasia. By implementing parallel work processing aided by technological advancements, we were able to shrink the time from database lock to filing down to just 79 days, easily within the top quartile for modern industry benchmarks. The full phase III data will be presented at ESPE as a late-breaking oral presentation in September. As Cristin noted, we're also very pleased that adolescents in both the US and Europe will now have access to PALYNZIQ following the label expansion in both regions earlier this year.

Speaker #3: I am particularly pleased to highlight our very recent submission of the supplemental NDA for Voxogo for the treatment of hypochondroplasia. By implementing parallel work processing, aided by technological advancements, we were able to shrink the time from database lock to filing down to just 79 days, easily within the top quartile for modern industry benchmarks.

Speaker #3: The full phase three data will be presented at SP as a late-breaking oral presentation in September. As Cristin noted, we're also very pleased that adolescence in both the US and Europe will now have access to Palanziq, following the label expansion in both regions earlier this year.

Speaker #3: Moving now to slide 17 and BMN820, formerly known as DMX200, a late-stage addition to our pipeline resulting from the Amicus acquisition. BMN820 is a first-in-class oral CCR2 inhibitor, which it accomplishes through blockade of receptor heterodimerization.

Greg Friberg: Moving now to slide 17 and BMN 820, formerly known as DMX-200, a late-stage addition to our pipeline resulting from the Amicus acquisition. BMN 820 is a first-in-class oral CCR2 inhibitor, which it accomplishes through blockade of receptor heterodimerization. It is currently in phase III development for focal segmental glomerulosclerosis or FSGS. This is an asset for which we hold exclusive US commercialization rights and are partnered with Dimerix, who remains responsible for operationalizing the phase III study. If the data are supportive, this could provide a new mechanism of action for the treatment of FSGS. FSGS is a progressive kidney disease that leads to proteinuria and declining kidney function over time. There are an estimated 30,000 addressable patients in the United States. Only one therapy is approved as of today, and its label is somewhat narrow, excluding patients with nephrotic syndrome.

Speaker #3: It is currently in phase 3 development for focal segmental glomerulosclerosis, or FSGS. This is an asset for which we hold exclusive U.S. commercialization rights, and we are partnered with Dimerix, who remains responsible for operationalizing the phase 3 study.

Speaker #3: If the data are supportive, this could provide a new mechanism of action for the treatment of FSGS. FSGS is a progressive kidney disease that leads to proteinuria and declining kidney function over time.

Speaker #3: There are an estimated 30,000 addressable patients in the United States. Only one therapy is approved as of today, and its label is somewhat narrow, excluding patients with nephrotic syndrome.

Speaker #3: Durable stabilization of kidney function remains a significant unmet need. BMN820 targets an orthogonal mechanism to the vascular targeting agents with the potential to treat a broad FSGS population.

Greg Friberg: Durable stabilization of kidney function remains a significant unmet need. BMN 820 targets an orthogonal mechanism to the vascular targeting agents with the potential to treat a broad FSGS population. It has shown a favorable safety and tolerability profile to date. The FDA has agreed that proteinuria is an appropriate endpoint for approval in our phase III ACTION 3 trial, and we expect phase III data in 2028. If the data are supportive, BMN 820 represents an attractive new pipeline asset with upside in a large area of unmet need. With that, I will turn the call over to Brian. Brian?

Speaker #3: It has shown a favorable safety and tolerability profile to date. The FDA has agreed that proteinuria is an appropriate endpoint for approval in our Phase 3 ACTION-3 trial, and we expect Phase 3 data in 2028.

Speaker #3: If the data are supportive, BMN820 represents an attractive new pipeline asset with upside in a large area of unmet need. With that, I will turn the call over to Brian.

Speaker #3: Brian?

Speaker #4: Thank you, Greg. Please refer to today's press release for detailed second quarter 2026 results. Including reconciliations of gap to non-gap financial measures, which will also be available in our upcoming Form 10-Q.

Brian Mueller: Thank you, Greg. Please refer to today's press release for detailed Q2 2026 results, including reconciliations of GAAP to non-GAAP financial measures, which will also be available in our upcoming Form 10-Q. Turning to slide 19. We were pleased that Q2 revenue reached nearly $1 billion, representing 20% top-line growth year over year. Q2 non-GAAP operating margin was 36.4%, with non-GAAP diluted earnings per share of $1.20

Speaker #4: Turning to slide 19, we were pleased that second quarter revenue reached nearly $1 billion, representing 20% top-line growth year over year. Second quarter non-gap operating margin was 36.4%, with non-gap diluted earnings per share of $1.20.

Speaker #4: Non-gap R&D and SG&A expenses each increased year over year. Reflecting the operating expenses of the acquired Amicus business, together with continued investment in our pipeline and commercial execution.

Brian Mueller: Non-GAAP R&D and SG&A expenses each increased year-over-year, reflecting the operating expenses of the acquired Amicus business, together with continued investment in our pipeline and commercial execution. On a GAAP basis, Q2 SG&A results also included approximately $84 million of transaction and integration-related charges associated with the acquisition. These charges are excluded from our non-GAAP results. Below the operating income line, interest expense increased year-over-year due to the acquisition debt financing, and interest income decreased as we liquidated investments to fund the acquisition. These items, along with the higher operating expenses, contributed to the year-over-year decrease in non-GAAP diluted earnings per share. I want to spend a moment on our interest expense and interest income to make sure your expectations are aligned with ours.

Speaker #4: On a gap basis, second quarter SG&A results also included approximately $84 million of transaction and integration-related charges associated with the acquisition. These charges are excluded from our non-gap results.

Speaker #4: Below the operating income line, interest expense increased year over year due to the acquisition debt financing, and interest income decreased as we liquidated investments to fund the acquisition.

Speaker #4: These items, along with the higher operating expenses, contributed to the year-over-year decrease in non-gap diluted earnings per share. I want to spend a moment on our interest expense and interest income to make sure your expectations are aligned with ours.

Speaker #4: Based on current interest rates, interest expense associated with the acquisition debt financing is estimated at approximately $200 million on an annualized basis, or approximately $50 million per quarter.

Brian Mueller: Based on current interest rates, interest expense associated with the acquisition debt financing is estimated at approximately $200 million on an annualized basis, or approximately $50 million per quarter, with the term loans and senior notes scheduled to mature after 2030. Importantly, this interest expense is included in our non-GAAP results and therefore reduces non-GAAP diluted earnings per share. In addition, due to lower cash and investment balances following the close of the acquisition, we expect interest income to decrease year-over-year in the near term. Turning to slide 20 and our updated full-year 2026 guidance. On the strength of our H1 performance and our expectations for the balance of the year, we are raising our full-year total revenues, VOXZOGO revenue, and non-GAAP diluted earnings per share guidance.

Speaker #4: With the term loans and senior notes scheduled to mature after 2030. Importantly, this interest expense is included in our non-GAAP results and therefore reduces non-GAAP diluted earnings per share.

Speaker #4: In addition, due to lower cash and investment balances, following the close of the acquisition, we expect interest income to decrease year over year in the near term.

Speaker #4: Turning to slide 20 and our updated full year 2026 guidance, on the strength of our first half performance, and our expectations for the balance of the year, we are raising our full year total revenues Voxogo revenue and non-gap diluted earnings per share guidance.

Speaker #4: As you can see, our guidance updates today reflect double-digit growth from the midpoint, and our strong trajectory leading into the second half of 2026.

Brian Mueller: As you can see, our guidance updates today reflect double-digit growth from the midpoint and our strong trajectory leading into the H2 of 2026. Briefly on phasing. We expect Q3 revenue to be slightly higher than the Q2, reflecting a full quarter of Amicus revenue contributions and continued patient growth across our brands. Similar to prior years, we expect the Q4 to be our strongest quarter of the year, with a significant step-up versus Q3 and representing well over 50% of our H2 revenue outlook, primarily due to ordering dynamics in select markets. On non-GAAP diluted earnings per share, the Q3 will reflect a full quarter of Amicus operating expenses, while benefits from cost synergies are expected to become more meaningful in the Q4.

Speaker #4: Briefly on phasing, we expect third quarter revenue to be slightly higher than the second quarter, reflecting a full quarter of Amicus revenue contributions and continued patient growth across our brands.

Speaker #4: Similar to prior years, we expect the fourth quarter to be our strongest quarter of the year, with a significant step-up versus Q3 and representing well over 50% of our second half revenue outlook.

Speaker #4: Primarily due to ordering dynamics in select markets. On non-gap diluted earnings per share, the third quarter will reflect a full quarter of Amicus operating expenses, while the benefits from cost synergies are expected to become more meaningful in the fourth quarter.

Speaker #4: Combined with the anticipated revenue phasing and realization of synergies, we expect third quarter non-GAAP earnings per share to be slightly higher than Q2, and fourth quarter non-GAAP earnings per share to be significantly higher, representing the highest quarterly earnings per share of the year.

Brian Mueller: Combined with the anticipated revenue phasing and realization of synergies, we expect Q3 non-GAAP earnings per share to be slightly higher than Q2 and Q4 non-GAAP earnings per share to be significantly higher, representing the highest quarterly earnings per share of the year. In summary, Q2 reflected strong execution, disciplined investment, and continued progress integrating Amicus. While some integration activities will continue into next year, the integration is well underway and on track, with the majority of enabling decisions made and operating plans in place. We are impressed by and appreciative of the focus and efforts of both our BioMarin colleagues and all of our Amicus colleagues since the close of the acquisition.

Speaker #4: In summary, the second quarter reflected strong execution, disciplined investment, and continued progress integrating Amicus. While some integration activities will continue into next year, the integration is well underway and on track, with the majority of enabling decisions made and operating plans in place.

Speaker #4: We are impressed by and appreciative of the focus and efforts of both our BIOMARIN colleagues and all of our Amicus colleagues since the close of the acquisition.

Speaker #4: We are looking forward to the second half of this year where we remain focused on delivering our updated 2026 outlook while building towards the longer-term revenue and earnings potential that Alexander outlined in his remarks.

Brian Mueller: We are looking forward to H2 of this year, where we remain focused on delivering our updated 2026 outlook while building towards the longer-term revenue and earnings potential that Alexander outlined in his remarks. Thank you for your attention. We will now open the call to your questions. Operator?

Speaker #4: Thank you for your attention. We will now open the call to your questions. Operator?

Speaker #2: Thank you. We will now begin the question-and-answer session. 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.

Operator 2: Thank you. We will now begin the question and answer session. 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 join the queue. If you would like to withdraw your question, simply press star one again. We will go to our first question from Chris Raymond at Raymond James.

Operator: Thank you. We will now begin the question and answer session. 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 join the queue. If you would like to withdraw your question, simply press star one again. We will go to our first question from Chris Raymond at Raymond James.

Speaker #2: If you would like to star your question, simply press star 1 again. We'll go to our first question from Chris Raymond at Raymond James.

Speaker #3: Yeah, thanks for taking my question. I got two, actually. First, on Amicus, you guys gave a lot of metrics here on synergies with this integration.

Chris Raymond: Thanks for taking my question. I got two, actually. First, on Amicus. You guys gave a lot of metrics here on synergies with this integration. Just looking at 2028 synergies, they look really impressive. I was just wondering if maybe you could give a few waypoints as we get through 2027 and tell us the specific steps you are taking to get to these numbers, and maybe what is behind the delivery of the two and a half leverage a year early. Then I have a VOXZOGO question. Thanks.

Speaker #3: Just looking at 2028 synergies, they look really impressive. But just wondering if maybe you could maybe give a few waypoints as we get through 2027 and tell us the specific steps you're taking to get to these numbers.

Speaker #3: And maybe what's behind the delivery of the 2.5 leverage a year early. And then I have a Voxogo question. Thanks.

Speaker #4: Hi, Chris. It's Brian. I'll take that. Thanks. So yeah, just to start, we know we communicated a lot of metrics today. We do believe that this Amicus integration framework demonstrates the comprehensive value creation from this acquisition.

Brian Mueller: Hi, Chris. It's Brian. I'll take that. Thanks. Yeah, just to start, we know we communicated a lot of metrics today. We do believe that this Amicus integration framework demonstrates the comprehensive value creation from this acquisition. We quantified synergies at this approximately 50% level. That's going to drive significant accretion powered by the revenue growth as well. This not only validates our hypothesis at the time of the transaction, but exceeded our expectations at the time. I'd also like to emphasize that these decisions are made. We are now executing on an end-to-end integration plan to bring the Amicus business onto the BioMarin platform. As I noted there in the prepared remarks, it took a lot of work on both sides, and it's going very well.

Speaker #4: We quantified synergies at this approximately 50% level. That's going to drive significant accretion powered by the revenue growth as well. This not only validates our hypothesis at the time of the transaction, but exceeded our expectations at the time.

Speaker #4: And I'd also like to emphasize that these decisions are made. We are now executing on an end-to-end integration plan to bring the Amicus business onto the BioMarin platform.

Speaker #4: And as I noted there in the prepared remarks, it took a lot of work on both sides, and it's going very well. This accretion also translates to cash flow.

Brian Mueller: This accretion also translates to cash flow. As you noted, we pulled forward our leverage target by approximately one year. Altogether, we're not only delivering on the potential of this transaction, but exceeding it. Specific to your question, in terms of waypoints, we shared that we expected the transaction to be modestly dilutive in calendar 2026. I'll share that that's still the case, but it's honestly close to breakeven. Still expecting it to be accretive in the first calendar year. I would point you to the substantial accretion that begins next year, and then 2028 being the first full year. Because we are still integrating next year, 2028 will be the first full year where we're realizing all of the benefits from the synergies and integration.

Speaker #4: As you noted, we pulled forward our leverage target by approximately one year. Altogether, we're not only delivering on the potential of this transaction, but exceeding it.

Speaker #4: And specific to your question, in terms of waypoints, we shared that we expected the transaction to be modestly diluted in calendar '26. I'll share that that's still the case, but it's honestly close to break even.

Speaker #4: Still expecting it to be accretive in the first calendar year, but I would point you to the substantial accretion that begins next year and then 2028 being the first full year.

Speaker #4: Because we are still integrating next year, 2028 will be the first full year where we're realizing all of the benefits from the synergies and integration.

Speaker #4: And in terms of a waypoint, I might guide you to say that next year we're expecting half to slightly more than half of the synergies to be realized.

Greg Friberg: In terms of a waypoint, I might guide you to saying that next year we're expecting half to slightly more than half of the synergies to be realized.

Speaker #3: Great. Thanks. And maybe on Voxogo, just hearing Cristin's commentary around 90% of Voxogo patients remaining on therapy, I think you mentioned post the UV well launch.

Chris Raymond: Great. Thanks. Maybe on VOXZOGO, just hearing Cristin's commentary around 90% of VOXZOGO patients remaining on therapy, I think you mentioned post the U.S. launch. Ascendis gave us some numbers today. I think they said, 170 patients are enrolled to start therapy, and I think that's two-thirds of those are actually paid. Maybe just doing some math on the switchers and based on your commentary, can you maybe give us a sense of the dynamic in terms of your competition for new patients?

Speaker #3: Ascendus gave us some numbers today. I think they said 170 patients are enrolled to start therapy, and I think two-thirds of those are actually paid.

Speaker #3: Maybe just doing some math on the switchers—based on your commentary, can you give us a sense of the dynamic in terms of your competition for new patients?

Speaker #4: Yeah, thanks very much for your question, Chris. This is Alexander. Yeah, I think this is really important to sort of unpack the numbers and make sure there's no misunderstandings here with the various data points communicated by both companies.

Alexander Hardy: Thanks very much for your question, Chris. This is Alexander. I think this is really important to sort of unpack the numbers and make sure there is no misunderstandings here with the various data points communicated by both companies. I mean, clearly their number was based on a total patient enrollment number that includes naive patients, VOXZOGO discontinuations, whenever those may have happened, and of course, switches from VOXZOGO. Of course, the latter one, the switch rate, is of course the one that is relevant for us. So according to our data, and we have good visibility, as you would expect, in the United States, in terms of patient numbers and ongoing treatment. We have seen approximately 10% of patients switch. That translates to less than 100 patient switches in the approximately six months since they have been approved.

Speaker #4: I mean, clearly their number was based on a total patient enrollment number. That includes naïve patients. Voxogo discontinuations were never—those may have happened.

Speaker #4: And of course, switches from Voxzogo. And of course, the latter one, the switch rate, is the one that's relevant for us. So, according to our data—and we have good visibility, as you would expect, in the United States in terms of patient numbers and ongoing treatment—we've seen approximately 10% of patients switch.

Speaker #4: That translates to less than 100 patients switches. In the approximately six months since they were being approved, that translates, of course, to a very small impact on our almost 4 billion dollars in revenue this year.

Alexander Hardy: That translates, of course, to a very small impact on our almost $4 billion in revenue this year. Taking all things into account, based on the strong growth that we are seeing and projecting, with 20% of increase in patients globally on VOXZOGO in the quarter, we feel comfortable increasing VOXZOGO revenue guidance for the year for over $1 billion. I think zooming out as well, if I could comment, I think bigger picture, their update on the launch and the pace reflects how hard the US market is in achondroplasia. When you have geographically dispersed patients, you have low visit frequency, you have care split between general pediatricians and specialists, and this all impacts the opportunity and the pace of switches and starts. Of course, as you know, Chris, new patient starts are dominated by the zero to two patient populations.

Speaker #4: So taking all things into account, based on the strong growth that we are seeing and projecting, is 20% of increase in patients globally on Voxogo in the quarter.

Speaker #4: We feel comfortable increasing Voxogo revenue guidance for the year for over 1 billion dollars. I think zooming out as well, if I could comment, I think bigger picture, their update on the launch and the pace reflects how hard the US market is in chondroplasia.

Speaker #4: When you have geographically dispersed patients, you have low visit frequency, you have care split between general pediatricians and specialists. And this all impacts the opportunity and the pace of switches and starts.

Speaker #4: And of course, as you know, Chris, new patients start at the dominated by the 0 to 2 patient populations. The guidelines say diagnose and treat as early as possible after birth.

Alexander Hardy: The guidelines say diagnose and treat as early as possible after birth. As you know, we remain the only product with the less than two indication, and we expect to remain so for a good amount of time. I hope that helps, gives you a little bit of perspective on the data and what we are seeing and what we are looking forward to in the remainder of the year.

Speaker #4: And as you know, that remain we remain the only product with the less than 2 indication and we are expect to remain so for a good amount of time.

Speaker #4: So I hope that helps, gives you a little bit of perspective on the data and what we're seeing and what we're looking forward to in the remainder of the year.

Speaker #3: Yep. Thank you.

Chris Raymond: Yep. Thank you.

Speaker #1: We'll go next to Corey Kazimov at Evercore ISI.

Operator 2: We'll go next to Cory Kasimov at Evercore ISI.

Operator: We'll go next to Cory Kasimov at Evercore ISI.

Speaker #5: Hey, good afternoon, guys, and thanks for taking my question. So I also want to ask something on the heels of the competitive update this morning.

Cory Kasimov: Hey, good afternoon, guys, and thanks for taking my question. I also want to ask something on the heels of the competitive update this morning, and I'm wondering how you think about the combination of a weekly CNP analog plus growth hormone eventually slotting into the treatment algorithm. Is there anything that's stopping you from, or physicians from using VOXZOGO and/or BMN 333 in the future with growth hormone? Thank you.

Speaker #5: And I'm wondering how you think about the combination of weekly CMP analog plus growth hormone eventually slotting into the treatment algorithm. And is there anything that's stopping you from or physicians from using Voxogo and/or BMN333 in the future with growth hormone?

Speaker #5: Thank you.

Speaker #4: Thanks, Corey. This is Greg Friberg. I think I'll tackle that one. Looking at that data, I think first and foremost, we have to recognize that the coach study is a small study.

Greg Friberg: Thanks, Cory. This is Greg Friberg. I think I'll tackle that one. Looking at that data, I think first and foremost, we have to recognize that the COACH study is a small study. I think it's about 21 patients, single arm, split into two cohorts. We have to be careful in over-interpreting it, particularly when we slice the data at six-month intervals. Now that being said, I think the question with the growth hormone combination today is the same as it has been from the start. We know growth hormone alone can cause increases in AGV, but they're temporary, and actually, they don't result in major increases in achondroplasia and increases in final adult height. The question remains, what are we learning from the dataset?

Speaker #4: I think it's about 21 patients, single-arm, split into two cohorts. So we have to be careful in overinterpreting it, particularly when we slice the data at six-month intervals.

Speaker #4: Now, that being said, I think the question with the growth hormone combination today is the same as it has been from the start. We know growth hormone alone can cause increases in AGV, but they're temporary.

Speaker #4: And actually, they don't result in major increases in acondroplasia and increases in final adult height. So the question remains, what are we learning from the data set?

Speaker #4: With this data point, I would say just the eyeball test tells us that it looks like the effects of growth hormone adding on to CMP appear to be waning.

Greg Friberg: With this data point, I would say just the eyeball test tells us that it looks like the effects of growth hormone adding on to CNP appear to be waning. I don't know why that would be in the naive patients more than the add-on to people who are already on CNP. It's a small dataset.

Speaker #4: I don't know why that would be in the naive patients more than the add-on to people who are already on CMP. It's a small data set.

Speaker #4: But the question then becomes, is this the beginning of a longer-term trend? It's an unanswerable question. I think the question that our endocrinologist, care most about, which there are sophisticated group, they've worked with growth hormone for a long time, is will this ultimately contribute to the health and wellness and by extension, the final adult height of patients?

Greg Friberg: The question then becomes, is this the beginning of a longer-term trend? It's an unanswerable question. I think the question that our endocrinologists care most about, which they're a sophisticated group, they've worked with growth hormone for a long time, is will this ultimately contribute to the health and wellness and, by extension, the final adult height of patients? The concern always is that growth hormone may close growth plates early, and that is not something that is in a short study of 18-month duration, something that you can really get a read on. So I think today it's incremental data. We're certainly seeing that the growth spurts might be declining. You see the slopes increases, and that's as compared to the ACHIEVE. I'm sorry, the APPROACH study, where you don't see that kind of a shift out at two years.

Speaker #4: The concern always is that growth hormone may close growth plates early, and that is not something that is in a short study of 18-month duration, something that you can really get a read on.

Speaker #4: So I think today it's incremental data. We're certainly seeing that the growth spurts might be declining. You see the slopes increases. And that's as compared to the achieves I'm sorry, the approach study, where you don't see that kind of a shift out at two years.

Speaker #4: I was happy to see that they're following up on the two-year data there as well. And again, I think there are unanswered questions that time will very closely.

Cristin Hubbard: I was happy to see that they're following up on the two-year data there as well. Again, I think there are unanswered questions that time will tell. Of course, we're watching this very closely. We're going to follow the data. We're going to make evidence-based decisions. From a biologic standpoint, there is nothing unique about TransCon CNP when it comes to taking a CNP agent and combining it with growth hormone. It's too early to tell, and we're looking forward to seeing more data out in the order of three years plus.

Speaker #4: We're going to follow the data. We're going to make evidence-based decisions and from a biologic standpoint, there is nothing unique about transcon CNP when it comes to taking a CNP agent and combining it with growth hormone.

Speaker #4: It's too early to tell, and we're looking forward to seeing more data out in the order of three years plus.

Speaker #5: Very helpful. Thanks, Greg.

Cory Kasimov: Very helpful. Thanks, Greg.

Speaker #1: We'll take our next question from Jess Pye at JP Morgan.

Operator 2: We'll take our next question from Jess Meyer at JP Morgan.

Operator: We'll take our next question from Jess Meyer at JP Morgan.

Speaker #6: Hey, guys, good afternoon. Thanks for taking my question. I was curious if you could speak to whether those synergies associated with the Amicus deal will fall to the bottom line, or whether you expect those to be reinvested in the business.

Jess Meyer: Hey, guys. Good afternoon. Thanks for taking my question. I was curious if you could speak to whether those synergies associated with the Amicus deal will fall to the bottom line or whether you expect those to be reinvested in the business.

Speaker #4: Hey, Jess. It's Brian. Thanks. Great question. We do expect those synergies to drop to the bottom line. However, to your point, there is also reinvestment.

Brian Mueller: Hey, Jess. It's Brian. Thanks. Great question. We do expect those synergies to drop to the bottom line. However, to your point, there is also reinvestment. Part of the strategy is to accelerate the growth potential of Galafold and Pombiliti and Opfolda, which will require some incremental investment. I'll share with you that compared to the synergy numbers we shared today, it is a modest portion of that. More importantly, and this is why we spoke to synergies on a growth basis, any of that reinvestment sits within the existing structure of our P&L. It's part of normal Metabolic Conditions, sales and marketing going forward. We don't consider it an offset to the synergies itself, which will live on. Again, if you chose to calculate net synergies with the investments, it's very modest.

Speaker #4: So part of the strategy is to accelerate the growth potential of GalaFold and Pobility and Abfolda, which will require some incremental investment. I'll share with you that compared to the synergy numbers we shared today, it is a modest portion of that.

Speaker #4: But more importantly, and this is why we spoke to Synergies on a growth basis, any of that reinvestment fits within the existing structure of our P&L.

Speaker #4: It's part of normal metabolic condition, sales and marketing going forward. So we don't consider it an offset to the synergies itself, which will live on.

Speaker #4: And again, if you chose to calculate in that synergies with the investments, it's very modest.

Speaker #6: Thank you.

Jess Meyer: Thank you.

Speaker #1: Our next question comes from Salvine Richter at Goldman Sachs.

Operator 2: Our next question comes from Salveen Richter at Goldman Sachs.

Operator: Our next question comes from Salveen Richter at Goldman Sachs.

Speaker #2: Thanks for taking our question. This is Tommy on for Salvine. Curious if you could provide more detail. You spoke to the mechanisms behind driving increased diagnosis and switching for Poability, Abfolda, and for Galafold.

[Analyst] (Goldman Sachs): Thanks for taking our question. This is Tommy on for Salveen. Curious if you could provide more detail. You spoke to the mechanisms behind driving increased diagnosis and switching for Pombiliti, Opfolda and for Galafold. A follow-up, what is your appetite for future BD and what stage or type if so? Thank you.

Speaker #2: How do these efforts differ in or strategy differ in the US versus ex-US? And a follow-up, what is your appetite for future BD and what stage or type if so?

Speaker #2: Thank you.

Speaker #6: Yeah, I'll take that first part of the question, Tommy. Thank you so much. And I hope you could hear it in the prepared remarks, but I'll say it again.

Cristin Hubbard: Yeah. I'll take that first part of the question, Tommy. Thank you so much. I hope you could hear it in the prepared remarks, but I'll say it again. We are absolutely delighted about what we have, both the opportunity and quite frankly, the responsibility to do for both the Fabry and Pompe communities. The more we've been able to dig into it since the close, we've really unearthed what I think are some meaningful levers that we can pull to drive the growth and therefore target the peak revenues of $1.4 billion for Galafold and $1.2 billion for Pompe, excuse me, for Pomup in the future. Now, looking specifically, I know you asked the question differentially across the US and ex-US. In large part, the overall lever or the levers are very similar.

Speaker #6: We are absolutely delighted about what we have both the opportunity and, quite frankly, the responsibility to do for both the Febre and Pompei communities.

Speaker #6: And the more we've been able to dig into it since the close, we've really unearthed, I think, are some meaningful levers that we can pull to drive the growth and therefore target the peak revenues of 1.4 billion for GalaFold and 1.2 billion for Pompei excuse me, for Pomp in the future.

Speaker #6: Now, looking specifically—and I know you asked the question kind of differently across the US and ex-US—in large part, the overall lever or the levers are very similar.

Speaker #6: While they make it executed at the country level slightly differently, the areas that we're going to really put our investment into are quite similar.

Cristin Hubbard: While they may get executed at the country level slightly differently, the areas that we're going to really put our investment into are quite similar, and they fit very well within the BioMarin set of capabilities that we've built over the decades that we've been doing this. For Galafold, this really is going to be about diagnosis. We recognize that in the metabolic population or in the Fabry community at large, there really is still very limited diagnosis, especially for those with late onset and or the female patients. We plan to really target those communities trying to drive broader diagnosis and importantly, really starting to close the gap between diagnosis and treatment so that we can show that physicians that treating earlier and even in milder sets or milder conditions is really important.

Speaker #6: And they fit very well within the biomarine set of capabilities that we've built over the decades that we've been doing this. For GalaFold, this really is going to be about diagnosis.

Speaker #6: We recognize that in the medical population, or in the Fanbre community at large, there really is still very limited diagnosis, especially for those with late onset and/or the female patients.

Speaker #6: So we plan to really target those communities trying to drive broader diagnosis and, importantly, really starting to close the gap between kind of diagnosis and treatment so that we can show that kind of physicians that treating earlier and even in milder sets or milder conditions is really important.

Speaker #6: On the Pomp side, this really is about accelerating switches. And this is true, again, in both the US as well our focus is going to be on the waning or the clinically declining patients that are on a current therapy, where we believe that we can really kind of continue to show what disease progression could look like.

Cristin Hubbard: On the Pomup side, this really is about accelerating switches, and this is true, again, in both the US as well as outside the US. Here our focus is going to be on the waning or the clinically declining patients that are on a current therapy where we believe that we can really continue to show what disease progression could look like, and if patients aren't meeting those targets, how to ensure that they're advocating for treatment. Those are the areas we're really going to be focused. I know that the next question. Oh, Greg, do you have something to add? Yeah. I would just add, Tommy, from the medical affairs standpoint, particularly for Fabry, where we know, I would say generously, maybe only 40% of the patients are actually diagnosed with the condition. Just to give you some granularity there.

Speaker #6: And if patients aren't meeting those targets, how to ensure that they're advocating for treatment. So those are the areas we're really going to be focused.

Speaker #6: And I know that the next question oh, Greg, do you have something to add?

Speaker #5: Yeah, I would just add, Tommy, from the medical affairs standpoint, particularly for Febre, where we know I would say generously, maybe only 40% of the patients are actually diagnosed with a condition.

Speaker #5: Just to give you some granularity there, electronic health record work to, again, shorten the time between diagnosis and, again, symptom when the symptoms arrive.

Greg Friberg: Electronic health record work to, again, shorten the time between diagnosis, and again, when the symptoms arrive. Family cascade testing and reclassification of variants. These are things that we've done previously in other settings. There are also some great work that our former Amicus colleagues had begun, and we have an opportunity to scale that a bit larger. Just to give you a data point, there's over almost 50 different diagnostic activities and programs going on around the globe right now. It is a very local phenomenon. We think that we can put more firepower

Speaker #5: Family cascade testing and reclassification of variants, these are things that we've done previously in other settings. There are also some great work that our former amicus colleagues had begun.

Speaker #5: And we have the opportunity to scale that a bit larger. Just to give you a data point, there's over almost 50 different diagnostic activities and programs going on around the globe right now.

Speaker #5: And so it is a very local phenomenon, and we think that we can put more firepower and technology behind some of those assets.

Greg Friberg: Technology behind some of those assets.

Speaker #6: These are things that we can start right away in the countries where GalaFold and Pomp are already commercialized, but what's also really important to note is that we plan on geographically expanding these products.

Cristin Hubbard: These are things that we can start right away in the countries where Galafold and Pombiliti and Opfolda are already commercialized. What's also really important to note is that we plan on geographically expanding these products. In over 10 countries relative to where we are today with Galafold and more than 20 countries for Pombiliti and Opfolda, which will also help to drive that growth. Over to you, Alexander.

Speaker #6: So in over 10 countries, relative to where we are today with GalaFold and more than 20 countries for Pobility and Abfolda, which will also help to drive that growth.

Speaker #6: Over to you, Alexander.

Speaker #5: Thanks, Tommy. Yes, this is Alexander Holland to your BD question. So as you've heard from Kristin and from Greg, the integration and acceleration of GalaFold and Pobility and Abfolda strengthen our growth outlook for biomarine.

Alexander Hardy: Thanks, Tommy. Yes, this is Alexander. I'll answer your BD question. As you heard from Cristin and from Greg, the integration and acceleration of Galafold and Pombiliti and Opfolda strengthen our growth outlook for BioMarin. With our now more diversified and growing commercial portfolio, our focus is now more shifting to expanding our clinical stage pipeline. Of course, we're going to continue doing research collaborations as we've always done. I think you would've seen the announcement recently of the collaboration with agorum. As we de-lever, you can expect us to do deals to expand out our clinical stage programs over the next 12 to 18 months.

Speaker #5: So with our now more diversified and growing commercial portfolio, our focus is now more shifting to expanding our clinical stage pipeline. Of course, we're going to continue doing research collaborations as we've always done I think you just saw you would have seen the announcement recently of the collaboration with Lorum.

Speaker #5: But as we deliver, you can expect us to do deals to expand out our clinical stage programs over the next 12 to 18 months.

Speaker #1: We'll move to our next question from Phil Nadeau au at TD Cowan.

Operator 2: We'll move to our next question from Phil Nadeau at TD Cowen.

Operator: We'll move to our next question from Phil Nadeau at TD Cowen.

Speaker #7: Good afternoon. Thanks for taking our questions. Two from us. First, on the upcoming ITC case, we expect a decision by the end of August.

Phil Nadeau: Good afternoon. Thanks for taking our questions. Two from us. First, on the upcoming ITC case, we expect a decision by the end of August. We're curious to get your most recent thoughts on that case, and in particular, any thoughts you have on the possibility of a settlement. Second, just to follow up on the diagnosis points that you just made. I think 20 years ago, we heard from Genzyme that they thought they were going to penetrate more quickly the late-onset in female patient populations. You've talked about what you can do, but why haven't those patients been diagnosed so far? It does seem like others have had efforts. Where those efforts fallen short? Thanks.

Speaker #7: We're curious to get your most recent thoughts on that case and, in particular, any thoughts you have on the possibility of a settlement. And then second, just to follow up on the diagnosis points that you just made, I think 20 years ago we heard from Gen Z that they thought they were going to penetrate more quickly the late onset and female patient populations.

Speaker #7: So, you've talked about what you can do, but why haven't those patients been diagnosed so far? It does seem like others have made efforts.

Speaker #7: Where are those efforts falling short? Thanks.

Speaker #8: Thanks. Thanks for your question, Phil. This is Alexander. I'll take the opportunity just to clarify some of the timelines and the facts around the ITC case.

Alexander Hardy: Thanks for your question, Phil. This is Alexander. I'll take the opportunity just to clarify some of the timelines and the facts around the ITC case. As you can probably expect, I'm not going to get into our legal strategy or speculate on the outcomes. On the 21 August of this month, the administrative law judge will deliver their initial determination. Within weeks following that initial determination, the commission decides whether they're going to review that initial determination. The final decision is expected on the 21 December of this year. That's either affirming or reversing all or a portion of that initial determination. Either party has 60 days to lobby the president. He has a presidential review period, which goes through 21 February of next year.

Speaker #8: But as you can probably expect, I'm not going to get into our legal strategy or speculate on outcomes. So on the 21st, of this month, the 21st of August, the administered law judge will deliver their initial determination.

Speaker #8: Within weeks following that initial determination, the commission decides whether they're going to review that initial determination. The final decision is expected on the 21st of December of this year.

Speaker #8: At that time, affirming or reversing all or a portion of that initial determination. And then either party has 60 days to lobby the president. He has a presidential review period, which goes through February 21st of next year.

Speaker #8: But I would just highlight that if an exclusion order is determined in that decision by the commission, then it's effective during that period. So that's sort of what you can expect from a timeline perspective.

Alexander Hardy: I would just highlight that if an exclusion order is determined in that decision by the commission, then it's effective during that period. That's sort of what you can expect from a timeline perspective. We're awaiting that date of the 21st, which is coming shortly. I'd also highlight that upon the completion of the ITC process, we would expect to enforce our patent in federal district court, where, of course, monetary damages are available.

Speaker #8: And we're waiting that data the 21st, which is coming shortly. I'd also highlight that upon the completion of the ITC process, we would expect to enforce our patent in federal district court where, of course, monetary damages are available.

Speaker #4: And Phil, I'm going to tax this is Greg Freberg. I'm going to tackle your second question on the diagnostic points, if that's all right.

Greg Friberg: Phil, this is Greg Friberg. I'm going to tackle your second question on the diagnostic points, if that's all right. With regard to why there hasn't been more progress in the field, I think it's a pretty simple answer in that this is a very elusive disease. It's one where patients can have a very heterogeneous presentation, something like seven different organ systems that can be affected, presenting in a variety of clinics. True with all rare diseases, it's this elusiveness of diagnosis, seven to 10 years to actually make their way to knowing what's causing their symptoms. Fabry is really a case study in that. That's why we're trying to focus on where we think we can have the most impact. Again, we highlighted a couple of them, but obviously, this isn't just about educating community physicians to be on the lookout.

Speaker #4: With regard to why there hasn't been more progress in the field, I think it's a pretty simple answer in that this is a very elusive disease.

Speaker #4: It's one where patients can have a very heterogeneous presentation, something like seven different organ systems that can be affected, presenting in a variety of

Speaker #1: Of clinics and , you know , true with all rare diseases . It's this elusiveness of diagnosis . The , you know , 7 to 10 years to actually make their way to knowing what's causing their symptoms .

Speaker #1: And Fabry is really a case study in that . That's why we're trying to focus on where we think we can have the most impact .

Speaker #1: And again , we highlighted a couple of them . But obviously , this isn't just about educating community physicians to be on the lookout , using technology , using electronic health records , using testing and training approaches to try to identify flags earlier .

Greg Friberg: Using technology, using electronic health records, using testing and training approaches to try to identify flags earlier. Again, it's not just about patient finding, it's about shortening that time to diagnosis. I would say that on top of that, one of the very fruitful endeavors that we've been involved with in other genetic conditions is family cascade testing. If you find one person in the family who is affected by this, again, you do the boots on the ground work to find the other patients that could be affected. Finding them early is the name of the game with Fabry, but it's been elusive up till now, and I think it reflects the wiliness and unfortunately the heterogeneity of this disease.

Speaker #1: Again , it's not just about patient finding , it's about shortening that time to diagnosis . I would say that on top of that , you know , one of the very fruitful endeavors that we've been involved with in other genetic conditions is family cascade testing .

Speaker #1: If you find one person in the family who is affected by this , again , you do the boots on the ground work to find the other patients .

Speaker #1: That could be affected , finding them early is the name of the game with Fabry , but it's been elusive up until now , and I think it reflects the the wiliness and unfortunately , the heterogeneity of this disease .

Speaker #2: That's very helpful . Thank you

Phil Nadeau: That's very helpful. Thank you.

Speaker #3: Next we'll go to Ellie Merle at Barclays

Operator 2: Next, we'll go to Ellie Merrill at Barclays.

Operator: Next, we'll go to Ellie Merrill at Barclays.

Ellie Merrill: Hey, guys. Thanks so much for taking the question. I guess of those 10% of patients in the US who switch from VOXZOGO by the end of July, I guess what trends or characteristics are you noticing in those patients versus, say, patients that are more likely to stay on VOXZOGO? I guess what degree of switching in the US is baked into the guidance for this year? A second part of a question on VOXZOGO. You mentioned that over 50% of US new starts were ages two and under. Maybe just how should we think about, I guess, the annual incidence of new starts in under age two, or maybe just the size of the US incident market for that age group, and how you're thinking about this as a growth contributor going forward? Thanks.

Eliana Merle: Hey, guys. Thanks so much for taking the question. I guess of those 10% of patients in the US who switch from VOXZOGO by the end of July, I guess what trends or characteristics are you noticing in those patients versus, say, patients that are more likely to stay on VOXZOGO? I guess what degree of switching in the US is baked into the guidance for this year? A second part of a question on VOXZOGO. You mentioned that over 50% of US new starts were ages two and under. Maybe just how should we think about, I guess, the annual incidence of new starts in under age two, or maybe just the size of the US incident market for that age group, and how you're thinking about this as a growth contributor going forward? Thanks.

Speaker #4: You guys , thanks so much for taking the question . So I guess of those 10% of patients in the US who switched from Voxzogo by the end of July , I guess what trends or characteristics are you noticing in those patients versus , say , patients that are more likely to stay on voxzogo ?

Speaker #4: And I guess what degree of switching in the US is baked into the guidance for this year ? And then the second part of a question on you mentioned that over 50% of US new starts were at ages two and under .

Speaker #4: Maybe just how should we think about , I guess , the annual incidence of new starts in under age two , or maybe just the size of the US incident market for that age group and how you're thinking about this as a growth contributor going forward .

Speaker #4: Thanks

Speaker #5: Yeah . Thank you for the . Thank you very much for the question . So one of the first question around the 10% that have switched , I mean , primarily what we're hearing is , is injection fatigue or wanting to try a weekly therapy .

Cristin Hubbard: Yeah. Thank you very much for the question. Onto the first question around the 10% that have switched. Primarily, what we're hearing is injection fatigue or wanting to try a weekly therapy. What I think is more important is looking at the 90% who we retained. What we're finding there is that really it is about not only the surround sound services we have around these patients and their families, namely with our clinical coordinators, how we're in there talking to the family and really building out that trusted relationship that is so important in this community. Also importantly, reminding them of the evidence base that we have, the safety, the efficacy, and something that quite frankly, no competitor can catch up to. That is something that we find is continued to be very compelling, and I expect that to be true in the future.

Speaker #5: But what I think is more important is looking at the 90% who we retain and , and what we're finding there is that really , it is about not only the surround sound services we have around these patients and their families , namely with our clinical coordinators , how we're in there , talking to the family and really building out that trusted relationship that is so important in this community , but also , importantly , reminding them of the evidence base that we have , the safety , the efficacy , and something that , quite frankly , no competitor can catch up to .

Speaker #5: That is something that we find is continued to be very compelling . And I expect that to be true in the future . Now , with regard to the the incidence of the 0 to 2 population , we estimate that there's about 150 births in the US , a year for with infants with achondroplasia .

Cristin Hubbard: With regard to the incidence of the zero to two population, we estimate that there's about 150 births in the US a year with infants with achondroplasia. Our intention is to very much target treatment as early as possible. As you know, the consensus guidelines certainly state that this is the most efficacious and beneficial for them to be treated early. Also what we're finding is that by targeting new specialties such as maternal fetal medicine, and really getting out early to help them understand possibly when in utero or even right at birth, the attributes of treating early. That's what we're out there doing, and we find that to be quite successful.

Speaker #5: And so our intention is to very much target treatment as early as possible . As you know , the consensus guidelines certainly state that this is the most efficacious and beneficial for them to be treated early .

Speaker #5: But also what we're finding is that by targeting new specialties such as maternal , maternal , fetal medicine and really getting out early to help them understand possibly when in utero or even right at birth , the attributes of treating early .

Speaker #5: That's what we're out there doing . And we find that to be quite successful

Speaker #6: Thanks , Kristen Hi , Ellie , it's Brian . I'll take your guidance question . Absolutely . We appreciate the interest in our switch assumptions , especially given the competitor update today and our competition metric as well As noted in February , when we initially gave guidance , we do expect switching and we shared what we're observing today .

Brian Mueller: Thanks, Cristin. Hi, Ellie. It's Brian. I'll take your guidance question. Absolutely. We appreciate the interest in our switch assumptions, especially given the competitor update today and our competition metric as well. As noted, in February when we initially gave guidance, we do expect switching and we shared what we're observing today. Our guidance did include a switch assumption. We're not going to quantify that at this time, nor comment on expectations at this time. Thank you.

Speaker #6: Our guidance did include a switch assumption. But we're not going to quantify that at this time, nor comment on expectations at this time.

Speaker #6: Thank you

Speaker #3: Next, we'll move to Mohit Bansal at Wells Fargo.

Operator 2: Next, we'll move to Mohit Bansal at Wells Fargo.

Operator: Next, we'll move to Mohit Bansal at Wells Fargo.

Speaker #7: Great . Thank you very much for taking my questions . So , so , Kristen , the the 10% patients who have switched based on market research , your market research , where do you think , you expect this to settle in the US market ?

Mohit Bansal: Great. Thank you very much for taking my questions. Cristin, regarding the 10% patients who have switched, based on your market research, where do you expect this to settle in the US market? The related question is, how different or similar is ex-US market in terms of how entrenched you are versus how challenging it could be for the competitor to come in and take share from you? Thank you.

Speaker #7: And the related question is how different or similar is Ex-u.s. market in terms of in terms of your how entrenched you are versus how challenging it could be for the competitor to , to , to come in and take share from you .

Speaker #7: Thank you .

Speaker #5: And if I could , maybe could you maybe that second question , just so I make sure I answered it , I didn't quite understand the question in the second one .

Cristin Hubbard: Mohit, could you maybe that second question, just so I make sure I answer it. I didn't quite understand the question in the second one.

Speaker #7: So the question is like how xx's market is similar or different versus the US market in terms of setup and structure , where it could be a challenging or easy for a competitor to take share versus the US market .

Mohit Bansal: The question is how ex-US market is similar or different versus the US market in terms of setup and structure, where it could be challenging or easy for a competitor to take share versus the US market. Just trying to understand the structure of the US versus ex-US market for achondroplasia there. Thank you.

Speaker #7: So just , just trying to understand the structure of the US versus market for a contemplation there . Thank you .

Speaker #5: Very good . Okay . Thank you very much for , for , for the question . Now , of course , to the first question as to when do we expect , you know , what do we expect going forward ?

Cristin Hubbard: Very good. Okay. Thank you very much for the question. Now, of course, to the first question as to when do we expect, what do we expect going forward, as Brian shared, we're not necessarily going to share our expectations because the truth of the matter is we need to continue to monitor this and closely watch if this levels out, if this is a bolus or if this is steady state. This is something that we need to very much monitor at this stage in time. I do think that what you would find most likely is that the segment that is most apt to switch, first and foremost, as I'd said, are those that either have injection fatigue, are looking for the convenience of a product that VOXZOGO has a very similar efficacy profile to it. However, they might want a weekly shot.

Speaker #5: As Brian shared , we're not necessarily going to share our expectations because the truth of the matter is we need to to continue to monitor this and closely watch if this levels out , if this is , you know , if this is a bolus or if this is if this is steady state , this is something that we need to very much monitor at this and at this stage in time .

Speaker #5: I do think that what you would find most likely is that the segment that is most apt to switch , first and foremost , as I'd said , are those that either have injection fatigue , are looking for the convenience of a product that has a very , you know , that that has a very similar efficacy profile to it .

Speaker #5: However , they might want a weekly shot . So those are kind of what we're seeing out there . But I wouldn't be able to comment at this juncture in terms of how this is going to go in the future .

Cristin Hubbard: Those are kind of what we're seeing out there, I wouldn't be able to comment at this juncture in terms of how this is going to go in the future. Now, looking at it relative to the ex-US, I would say the biggest difference, and I know we've talked about this before in the US, is you have a much more segmented market, much more geographically dispersed. You have more specialties involved. We see that certainly as, I guess, a component in the competitive dynamics here. What we expect ex-US, we have not seen, there's not been any approvals or any product in other countries at this point in time. What we expect ex-US might, well, I'm not going to speak to it necessarily, I don't think that the dynamics are going to be wholly different ex-US.

Speaker #5: Now , looking at it relative to the ex-US , I would say the biggest difference , and I know we've talked about this before in the US , is you have a much more segmented market , much more geographically dispersed .

Speaker #5: You have more specialties involved . And we see that certainly as , I guess , a component in the competitive dynamics here , what we expect ex us , we don't we have not seen there's not been any approvals or any product in other countries at this point in time .

Speaker #5: But what we expect ex us might , you know , well , I'm not going to speak to it necessarily , but I don't think that the dynamics are going to be wholly different ex us .

Speaker #5: But that is something , again , that we will have to to remain vigilant on . And continue to see

Cristin Hubbard: That is something, again, that we will have to remain vigilant on and continue to see.

Speaker #3: Thank you . Moving next , we'll go to Akish Tewari at Jefferies .

Mohit Bansal: Thank you.

Operator 2: Moving next, we'll go to Akash Tewari at Jefferies.

Operator: Moving next, we'll go to Akash Tewari at Jefferies.

Speaker #8: Hi , this is Phoebe on for Akash . Thank you for taking our question . Another one on Voxzogo . Can you talk about what market work you've done so far for Hypochondroplasia and whether you expect any bolus at initial approval ?

[Analyst] (Jefferies): Hi, this is Phoebe on for Akash. Thank you for taking our question. Another one on VOXZOGO. Whether you expect any bolus at initial approval? If you've already identified a certain number of hypochondroplasia patients. Thank you.

Phoebe Tan: Hi, this is Phoebe on for Akash. Thank you for taking our question. Another one on VOXZOGO. Whether you expect any bolus at initial approval? If you've already identified a certain number of hypochondroplasia patients. Thank you.

Speaker #8: And if you've already identified a certain number of Hypochondroplasia patients , thank you .

Speaker #5: Yeah . Thank you very much for the question . I suppose Greg and I might might want to take this on together . I think in terms of the hypochondroplasia market , you know , what we've said very clearly is that we expect a a global total addressable , addressable patient population of around 14,000 .

Cristin Hubbard: Yeah. Thank you very much for the question. I suppose Greg and I might want to take this on together. I think in terms of the hypochondroplasia market, what we've said very clearly is that we expect a global total addressable patient population of around 14,000. The work now is really getting in there in the countries and identifying those patients as early as possible so that by the time, assuming we're able to get a regulatory approval, by the time we get there, we can launch immediately and cover as many of those patients who are amenable to treatment as possible. We've already talked a little bit about some of the global initiatives we've been working on to improve diagnosis. We talked about some of the targeted genetic reclassification work we're doing, as well as a lot of the physician and caregiver awareness that we're doing.

Speaker #5: But the work now is really getting in there in the countries . And identifying those patients as early as possible , so that by the time assuming we're able to get a regulatory approval by the time we get there , we can launch immediately and cover as many of those patients who are amenable to treatment as possible .

Speaker #5: And so we've already talked a little bit about some of the global initiatives we've been working on to include , excuse me , to improve diagnosis .

Speaker #5: We've talked about some of the targeted genetic reclassification work we're doing , as well as a lot of the physician and caregiver awareness that we're doing .

Speaker #5: Most recently, we've launched tactics that are really around having multiple kinds of digital and media campaigns, really primarily targeted in the US.

Cristin Hubbard: Most recently, we've launched tactics that are really around having multiple kind of digital and media campaigns, really primarily targeted in the US, and that's about shaping the marketplace. What we want to do is make sure that we're including HCP-directed disease education content, as well as caregiver and patient awareness programming. That really, again, is about making sure that we're getting as many patients diagnosed as possible, and then importantly, shortening that path from the time that they are diagnosed to the time that they're willing to treat. Over to you, Greg.

Speaker #5: And that's about shaping the marketplace and what we want to do is make sure that we're including HCP directed disease education content , as well as caregiver and patient awareness programming that really , again , is about making sure that we're getting as many patients diagnosed as possible .

Speaker #5: And then, importantly, shortening that path from the time that they are diagnosed to the time that they're willing to treat. Over to you, Greg.

Speaker #1: Yeah . Thanks . And just to go back as well , we only turn the card over two months ago . We're really pleased by the data that we saw in phase three .

Greg Friberg: Yeah. Thanks. Just to go back as well, we only turned the card over 2 months ago. We're really pleased by the data that we saw in phase III. Again, the AGV exceeded our expectations. We hit statistical significance on the height variables as well as arm span, and we're looking forward to presenting the subsets and additional safety data, and so forth, at ESPE in September. I would say with regard to patient finding, you can rest assured that we are working hard to bring what we think could be a potentially safe and effective therapy to hypochondroplasia patients. We're doing testing work. Again, we have metrics looking at not only testing rates, but testing yield. We're certainly preparing, again, to know what we think, again, the age of diagnosis is and so forth, and that's work that's ongoing right now.

Speaker #1: Again , the AGV exceeded our expectations . We hit statistical significance on the height variables as well as arm span . And we're looking forward to presenting the subsets and additional safety data .

Speaker #1: And so forth . At SPX in September , I would say with regard to patient finding , you can be rest assured that we are working hard to bring what we think could be a potentially safe and effective therapy to hypochondroplasia patients .

Speaker #1: We're doing testing work again . We have metrics looking at not only testing rates , but testing yield . We're certainly preparing , you know , again , to know .

Speaker #1: What we think . Again , the age of diagnosis is and so forth . And that's work that's ongoing right now . We see that age going downwards , which again , is a good sign that the classic challenge here that these patients aren't making their way to the right specialist is something that we've intervened with .

Greg Friberg: We see that age going downwards, which again, is a good sign that the classic challenge here that these patients aren't making their way to the right specialist is something that we've intervened with. At another time, I'd be happy to talk about other implementation science work we're doing. Again, to try to prepare the field in a pre-approval appropriate way, to again, make sure that the science is following and that we'll be able to, again, reach the most number of patients as possible.

Speaker #1: At another time , I'd be happy to talk about other implementation science work . We're doing again to try to prepare the field in , you know , in a pre pre approval appropriate way to again , make sure that that the science is following and that we'll be able to again , reach the most number of patients as possible

Speaker #3: Well , go next to Paul Matisse at Stifel

Operator 2: We'll go next to Paul Matteis at Stifel.

Operator: We'll go next to Paul Matteis at Stifel.

Speaker #9: On , on BD . What's next in terms of the scope of the types of things that Biomarin is looking at ? And Alexander , when you take a step back now and look at the revenue base and the profitability profile , you have , what's the optimal number of , I guess , phase one , two , three assets in a pipeline of biomarin size ?

Paul Matteis: On BD, what's next in terms of the scope of the types of things that BioMarin's looking at? Alexander, when you take a step back now and look at the revenue base and the profitability profile you have, what's the optimal number of, I guess, phase I, II, III assets in a pipeline of BioMarin size? Thank you.

Speaker #9: Thank you

Speaker #10: Thanks very much for the question . So , you know , we're looking obviously at , you know , we see see ourselves as a as a leader in the space of genetic conditions .

Alexander Hardy: Thanks very much for the question, Paul. We're looking obviously at. We see ourselves as a leader in the space of genetic conditions. We have strong business units, as you heard now. We call it Metabolic Conditions, Skeletal Conditions. Those are areas we're looking to supplement the nine products we have in those spaces, the nine products in our portfolio. But we're also interested in genetic conditions where it's a good fit with our capability, our expertise in genetics, for example, our regulatory expertise, our manufacturing expertise, our commercialization. Without getting specific at this point about what those additional therapy areas within the umbrella of genetic conditions, I think you probably have a sense of the sorts of types of diseases which really leverage that capability. And I think when you look at the Amicus acquisition, whilst it dropped just perfectly into that Metabolic Conditions business unit.

Speaker #10: We have strong business units , as you heard . Now , we we call it metabolic conditions , cells , conditions . So , you know , those are areas we're looking to , to , to supplement the , the many products have in those in those spaces that .

Speaker #10: Nine products in our portfolio , but we're also interested in genetic conditions where it's a good fit with our capability , our expertise in genetics , for example , our regulatory expertise , our manufacturing expertise , our commercialization .

Speaker #10: So without getting specific at this point about what those additional therapy areas within the umbrella of genetic conditions , I think you probably have a sense of sorts of the sorts of types of diseases which really leverage that capability .

Speaker #10: And I think when you look at the the amicus acquisition , it dropped just perfectly into that metabolic conditions business unit . You can see it's really the capabilities that we have that , that we can leverage that allows us to , to , to really say that , you know , the peak sales potential of these products is , is greater than they were before .

Greg Friberg: You can see it's really the capabilities that we have, that we can leverage, that allows us to really say that the peak sales potential of these products is greater than they were before. We think there's a really great opportunity for us to do that with other programs, but bringing them in the clinical stages. Expect more progress in the next 12 to 18 months. We're looking for a nice steady flow of products at all stages of development. We were excited, and I think it's worth just highlighting. We're excited to announce this quarter, we actually put more of a focus and really dug in on the DMX-200 asset, which we call now BMN 820. And we're really excited with that asset as we dug into it to have a phase III asset in the renal space.

Speaker #10: So we think there's a really great opportunity for us to do that with other programs, but bringing them in, in the clinical stages.

Speaker #10: So expect more progress in the next 12 to 18 months. We're looking for, you know, a nice, steady flow of products at all stages of development.

Speaker #10: We were excited . And I think , you know , it's worth just highlighting . We're excited to announce this this quarter . We , we actually put more of a focus and really dug in on the DMX 200 asset , which we call now DM 820 .

Speaker #10: And we're really excited to , to with that asset . As we've dug into it , to have a phase three asset in , in the renal space .

Speaker #10: So this hopefully gives you a sense of kind of what we're thinking about from a BD standpoint in a general perspective , but , you know , we're excited about the growth prospects and , and the opportunity for cash flow generation and the optionality .

Greg Friberg: This hopefully gives you a sense of kind of what we're thinking about from a BD standpoint, in a general perspective. But we're excited about the growth prospects and the opportunity for cash flow generation and the optionality this gives us to further strengthen our pipeline.

Speaker #10: This gives us to , to further strengthen our pipeline

Speaker #3: We'll take our next question from Sean Layman at Morgan Stanley .

Operator 2: We'll take our next question from Sean Laaman at Morgan Stanley.

Operator: We'll take our next question from Sean Laaman at Morgan Stanley.

Speaker #11: Good afternoon , Alexandra and Team . Hope everyone's well . And thanks for taking my questions . I guess if you look at the .

Sean Laaman: Good afternoon, Alexander and team. Hope everyone's well, thanks for taking my questions. I guess if you look at the $2.6 billion in POMP and Galafold guidance for mid-2035, how much of that is like market acceleration versus what BioMarin's adding to the pie. Since you've been able to get the business under your hood, what have you learned down that front that gives you good confidence that you might not have known before? Then if I can slip one in on BMN 333, how would you characterize the rate of enrollment in that study, and when might we see the next signpost? Thank you.

Speaker #11: Is it $2.6 billion in pomp and Galafold guidance , for mid 2035 ? Like how much of that is like market acceleration versus what Biomarin's adding to the pie and , you know , since you've been able to , you know , get the , the business under your hood , what have you learned down that front that gives you good confidence that you might not have known before ?

Sean Laaman: like market acceleration versus what BioMarin's adding to the pie. Since you've been able to get the business under your hood, what have you learned down that front that gives you good confidence that you might not have known before? Then if I can slip one in on BMN 333, how would you characterize the rate of enrollment in that study, and when might we see the next signpost? Thank you.

Speaker #11: And then if I can slip one in on BM three , three , three , you know , how would you characterize the rate of enrollment in that study ?

Speaker #11: And when might we see the next signpost ? Thank you

Speaker #5: Yeah . So thanks for the question , Sean , around kind of how much of the of the contribution there is related to , to perhaps what we can do differently ?

Cristin Hubbard: Yeah. Thanks for the question, Sean, around how much of the contribution there is related to perhaps what we can do differently. I'd say that when you look at the contribution in the build, we really did do a bottoms-up looking country by country about what we could do. We weren't playing around with the prevalence numbers or changing anything about the disease characteristics per se. This really was about when we put this onto the BioMarin platform, what could we do differently, and how does that look country by country? I would say the biggest contributor, as you would expect on the Galafold side, was opening up the diagnosis. Certainly, treatment rate plays a role there, but really the biggest contributor there was around opening up the diagnosis rates.

Speaker #5: I'd say that when , when you look at the contribution in the build and we really did do a bottoms up looking kind of country by country about what we could do .

Speaker #5: So we weren't playing around with the prevalence numbers or changing anything about the disease characteristics per se . This really was about when we put this onto the Biomarin platform , what could we do differently ?

Speaker #5: And how does that look ? Country by country ? So I would say the biggest , you know , kind of contributor as , as you would expect on the Galafold side was opening up the diagnosis .

Speaker #5: Certainly treatment rate plays a plays a role there , but really the biggest contributor there was around opening up the diagnosis rates on the permeability and opfolda that really was the biggest contributor was definitely how , you know , around switches and how quickly we could switch rate to move on that .

Cristin Hubbard: On the Pombiliti and Opfolda, that really was the biggest contributor was definitely around switches and how quickly we could get the switch rate to move on that. The question becomes how are we able to do this? As I'd mentioned, we looked at this country by country and really do feel confident about how we can click these into either existing countries where they're already opened up in those markets or importantly, have already set in motion what is going to be the regulatory as well as the reimbursement pathway moving into those specific countries. I'd say that that's the biggest thing. Again, this wasn't about changing prevalence numbers or tweaking with the funnel in that way. This really was about building on our own capabilities.

Speaker #5: And so the question becomes , how are we able to do this ? And so as I'd mentioned , we looked at this country by country and really do feel confident about how we can click these into either existing countries where they're already opened up in those markets , or importantly , have already set in motion what is going to be the regulatory as well as the reimbursement pathway moving into into those specific countries .

Speaker #5: So I'd say that that is the that's the biggest thing . And again , this wasn't about changing prevalence numbers or tweaking with the funnel in that way .

Speaker #5: This really was about building on our own capabilities .

Speaker #1: Yeah . And thanks , Sean , for the interest in three , three , three . We of course , are incredibly excited .

Greg Friberg: Yeah. Thanks, Sean, for the interest in BMN 333. We, of course, are incredibly excited. We have active enrollment going on in multiple time zones, multiple countries around the world. Again, we're looking for naive patients. We're entering the steep part of the enrollment curve. I don't expect that we'll give an update until we're completed enrollment in those 40 patients for the phase II portion. We want to just reiterate that again, our expectation is in 2027. We're going to answer this question. I know that there's been debate out there of whether or not, again, the free CNP will translate into more AGV. We have a strong conviction that it is an absolutely valid hypothesis. Happy to drill into that with others in more detail.

Speaker #1: We have active enrollment going on in multiple time zones , multiple countries around the world . Again , we're looking for naive patients .

Speaker #1: We're entering the steep part of the enrollment curve . I don't expect that we'll give an update until we're completed . Enrollment in those 40 patients for the phase two portion .

Speaker #1: But we just reiterate that , again , our expectation is in 2027 , we're going to answer this question . I know that there's been debate out there of whether or not , again , the free CNP will translate into more AGV .

Speaker #1: We have a strong conviction that it is an absolutely valid hypothesis. Happy to drill into that with others in more detail. Now is the time, building on the Phase 1 data that we saw.

Greg Friberg: Now is the time, building on the phase I data that we saw when we know we can increase exposure of free CNP. We know that pulls into pharmacodynamics in the plasma cyclic GMP. Now is the question to look at growth. That'll be a question that we answer in the next calendar year.

Speaker #1: When we know we can increase exposure of free CNP, we know that pulls into pharmacodynamics in the plasma cyclic GMP. Now is the question to look at growth.

Speaker #1: And that'll be a question that we answer in the next calendar year.

Speaker #11: Wonderful .

Alexander Hardy: Wonderful.

Sean Laaman: Wonderful.

Speaker #3: We'll go next to Alex Hammond at Wolfe Research.

Operator 2: We'll go next to Alex at Wolfe Research.

Operator: We'll go next to Alex at Wolfe Research.

Speaker #12: Thanks for taking the question . Just two from us . So first on the guidance bump on box , does that have less to do with about switching or more about growth ?

Alex Hammond: Thanks for taking the question. Just two from us. First on the guidance bump on VOXZOGO, does that have less to do with about switching or more about growth? Is that growth more US or OUS? On BMN 333 as well, how does the ASPEN study's operational seamless design give you levers to pull the timeline forward from the phase III perspective? Thank you.

Speaker #12: And is that growth more us or us ? And then on BM3 , three , three as well . How does the Aspen studies operational .

Speaker #12: Seamless design give you levers to pull the timelines forward from the phase three perspective . Thank you

Speaker #6: Hey , Alex . Thanks . This is Brian . I appreciate the question on the VOCs guidance raise . First and foremost , pleased with the strong Q2 and our confidence in the outlook for the second half of the year to be able to raise the guidance and get to the blockbuster status at the bottom end of the guidance .

Brian Mueller: Hey, Alex. Thanks. This is Brian. Appreciate the question on the VOXZOGO guidance raise. First and foremost, pleased with the strong performance in Q2 and our confidence in the outlook for H2 of the year to be able to raise the guidance and get VOXZOGO to the blockbuster status at the bottom end of the guidance. I'll note that you'll remember previously one of the variables that I pointed out at the beginning of the year when we guided was a couple of international price negotiations that were in process. I'll share that one of those closed successfully with a good outcome, and the other had some initial setbacks, but we are continuing with the process. There was some upside there to some of the contingency that was in the range. That was a bit behind it.

Speaker #6: I'll note that you'll remember, previously, one of the variables that I pointed out at the beginning of the year, when we guided, was a couple of international price negotiations that were in process.

Speaker #6: I'll share that . One of those closed successfully with a good outcome . And the other had some initial setbacks . But we are continuing with the process .

Speaker #6: But there was some upside there to to some of the contingency that was in the range . So so that was a bit behind it .

Speaker #6: And then the rest of it was growth and performance . And I'll just say that it was both us and global adding new patients , growing revenue confidence in 2026 .

Greg Friberg: The rest of it was growth and performance. I'll just say that it was both US and global, adding new patients, growing revenue, confidence in 2026. Thanks. Yeah. Thank you for the question again on 333. The operationally seamless phase II/III design really provides most of its benefit through recruitment acceleration and site startup. Not every country can start at the same time. They have different requirements with regard to regulatory approvals and so forth. This allows us to, under the umbrella of one protocol, work with the same IRBs, work with the same sites, have a parking lot of patients identified, and really, I think the most impressive benefits will come with the phase III recruitment. The phase II, again, is up and going, and we are off to the races.

Speaker #6: Thanks .

Speaker #1: Yeah . And thank you for the question again on three , three , three , the operationally seamless phase two three design really gets most of or provides most of its benefit through recruitment , acceleration , and site start up .

Speaker #1: Not every country can start at the same time . They have different requirements with regard to regulatory approvals and so forth . This allows us to , under the under the umbrella of one protocol , work with the same IRBs , work with the same sites , have a parking lot of patience identified .

Speaker #1: And really, I think the most impressive benefits will come with the Phase 3 recruitment. The Phase 2, again, is up and going, and we are off to the races.

Speaker #3: And this concludes our Q&A session . I will now turn the conference back over to Biomarin CEO Alexander Hardy for closing remarks .

Operator 2: This concludes our Q&A session. I will now turn the conference back over to BioMarin's CEO, Alexander Hardy, for closing remarks.

Operator: This concludes our Q&A session. I will now turn the conference back over to BioMarin's CEO, Alexander Hardy, for closing remarks.

Speaker #10: Thank you , operator , and thank you all for joining us today . Standout quarter across the business 20% top line growth , rapid , close and integration of amicus advancing pivotal data Award .

Alexander Hardy: Thank you, operator, and thank you all for joining us today. A standout quarter across the business. 20% top-line growth, the rapid close and integration of Amicus, advancing pivotal data toward VOXZOGO's second indication, hypochondroplasia. Strong demand for our innovative products led us to increase guidance today, including full-year total revenues. VOXZOGO now at the low end of $1 billion non-GAAP earnings per share. As we enter H2 of 2026, BioMarin is stronger, more diversified, better positioned than ever to lead in rare disease, to deliver for patients worldwide. Thank you for your continued support. Look forward to speaking to you soon.

Speaker #10: Voxzogo second indication Hypochondroplasia strong demand for our innovative products as to . Increase guidance today , moving full year total revenues of Togo .

Speaker #10: Now with a low end of $1 billion non-GAAP earnings per share. As we enter the second half of 2026, BioMarin is stronger, more diversified, and better positioned than ever to lead in rare disease and to deliver for patients worldwide. Thank you for your continued support.

Speaker #10: And look forward to speaking to you soon

Operator 2: This concludes today's conference call. Thank you for your participation. You may now disconnect.

Operator: This concludes today's conference call. Thank you for your participation. You may now disconnect.

Q2 2026 Biomarin Pharmaceutical Inc Earnings Call

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