Q3 2023 Biomarin Pharmaceutical Inc Earnings Call

November 1, 2023

Operator: Hello, and welcome to the BioMarin Pharmaceutical Q3 2023 conference call. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press star one on your telephone keypad. If you would like to withdraw your question again, press star one. We'll now turn the conference over to Traci McCarty, Head of Investor Relations. Please go ahead.

Hello, and welcome to the Biomarin Pharmaceuticals third quarter 2023 conference call. 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 star one on your telephone keypad.

Speaker 1: Hello and welcome to the BioMarin Pharmaceuticals Third Quarter 2023 Conference Call. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press star 1 on your telephone keypad. If you would like to withdraw your question, again press star 1. I'll turn the conference over to Tracy McCarty, Head of Investor Relations. Please go ahead.

I'd like to withdraw your question again press Star one well now turn the conference over to Traci Mccarty head of Investor Relations. Please go ahead.

Speaker 2: Thank you, JL, and thank you all for joining us today. To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of Biomorin Pharmaceutical Inc., including expectations regarding Biomorin's financial performance, commercial products, and potential future products in different areas of therapeutic research and development.

Traci McCarty: Thank you, JL, 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. On the call today from BioMarin management are JJ Bienaimé, Chairman and Chief Executive Officer; Jeff Ajer, Executive Vice President, Chief Commercial Officer; Hank Fuchs, President, Worldwide Research and Development; Greg Guyer, Executive Vice President, Chief Technical Officer; and Brian Mueller, Executive Vice President and Chief Financial Officer.

Thank you James 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 <unk> financial performance commercial products and potential future products in different areas of therapeutic research and development.

Speaker 2: Results may differ materially depending on the progress of Biomarin's product programs, actions of regulatory authority, availability of capital, future actions in the pharmaceutical market, and developments by competitors. And those factors detail in Biomarin's filings with the Securities and Exchange Commission, such as 10Q, 10K, and 8K reports.

Results may differ materially depending on the progress of <unk> product programs actions.

The regulatory authorities availability of capital future actions in the pharmaceutical market and developments by competitors and those factors detailed in Biomarin filings with the Securities and Exchange Commission, such as 10-Q, 10-K and 8-K reports.

Speaker 2: On the call today from Bimer and Management, our JJVNMA, Chairman and Chief Executive Officer, Jeff A.J. Executive Vice President, Chief Commercial Officer, Tank Fuchs, President, Worldwide Research and Development, Greg Geier, Executive Vice President, Chief Technical Officer, and Brian Mueller, Executive Vice President, and Chief Financial Officer. We do plan to end this call promptly at 2.30 PM Pacific time, and we thank you for your understanding. I will now turn the call over to Bimer and Chairman and CEO , JJVNMA.

On the call today from Biomarin management are JJ, if your enemy Chairman and Chief Executive Officer, Jeff <unk> Executive Vice President Chief Commercial Officer, Hank Fuchs, President worldwide Research and development, Greg Guyer Executive Vice President Chief Technical Officer, and Brian Mueller Executive Vice President and Chief Financial Officer.

Traci McCarty: We do plan to end this call promptly at 2:30PM Pacific Time, and we thank you for your understanding. I will now turn the call over to BioMarin's Chairman and CEO, Jean-Jacques Bienaimé.

We do plan to end the call promptly at 230 P. M Pacific time, and we thank you for your understanding I will now turn the call over to Byron's, Chairman and CEO J J P enemy.

Jean-Jacques Bienaimé: Thank you, Traci. Good afternoon. Good morning, everybody. As you will have seen this afternoon, we announced that I have decided after over 18 years with BioMarin, to retire as the Chairman and Chief Executive Officer, effective 1 December 2023. And as many of you are aware, I recently actually turned 70. I cannot believe it myself. So I will not... it will not be a surprise to you that succession planning has been underway with the board over the past several years. This announcement and the appointment of Alexander Hardy to succeed me as CEO follows a thorough multi-year planning process. Together with the board, we conducted a comprehensive search over the past year, and we are thrilled to have Alexander step into the role.

Thank you Traci good afternoon, and good morning, everybody as you will have seen this afternoon, we announced that.

Speaker 3: Thank you, Tracy. Good afternoon. Good morning, everybody. As you will have seen this afternoon, we announced that the.

Speaker 3: I have decided after over 18 years with Balmarine to retire as the chairman and chief executive officer effective December 1st.

I have decided not to over 18 years with Biomarin to retire as the chairman and Chief Executive Officer effective <unk>.

December 1st of this year.

Speaker 3: And as video you are aware, I recently actually turned 70. I cannot believe it myself. So I will not be surprised to you that sufficient planning has been underway with the board over the past seven years.

And as many of you are aware.

Recently actually turned 70 I cannot do it myself.

So I will not even have to have.

Surprise to you that sufficient planning has been underway with our board over the past.

Yes.

Speaker 3: This announcement and the appointment of Alexander Hardy to succeed me as CEO follows a thorough multi-year planning process.

These announcements and the appointment of Alexander Hardy to succeed me as CEO follows a thorough multiyear planning process.

Together with the board, we conducted a comprehensive search over the past year.

Speaker 3: Together with the board, we conducted a comprehensive search over the past year, and we are thrilled to have Alexander step into the role.

And we are thrilled to have that zehnder stepped into the role.

Speaker 3: As part of this transition, I would also step away from my position, the position that's chairman of the board of directors. However, I would continue to serve on the board until the 2024 annual meeting of step stock holders. And I will say as a consultant to the company for the remainder of 2024.

Jean-Jacques Bienaimé: As part of this transition, I will also step away from my position as Chairman of the Board of Directors. However, I will continue to serve on the board until the 2024 annual meeting of stockholders, and I will stay as a consultant to the company for the remainder of 2024. I look forward to working with Alexander and the board during this period to ensure a smooth transition. I am pleased that our leading independent director, Richard A. Meier, will be our new chair of the board. When I joined the company in May 2005, we had just one marketed product and recorded about $26 million in annual revenues. Since then, our team has developed and commercialized seven additional medicines, pioneered groundbreaking firsts, and transformed the lives of countless of patients.

As part of this transition that would also stepping away from my position the position as chairman of the board of.

Directors however.

That will continue to serve on the board until the 'twenty 'twenty four annual meeting of stockholders.

And I would say as a consultant to the company for the remainder of 2024.

I look forward to working with Alexander on the board during this period to ensure a smooth transition.

Speaker 3: I look forward to working with Alexander on the board during this period to ensure a smooth transition.

Speaker 3: I am pleased that our leading independent director, Richard Meyer, with a Randy will be our new chair of the board. When I joined the company in May.

I am pleased that our lead independent director, Richard Meier, with Randy will be our new chair of the board.

When I joined the company in May 2005.

Speaker 3: We had just one marketed product and recorded about $26 million in annual revenue.

Yeah, just one marketed product and recorded about $26 million.

Okay.

Speaker 3: Since then, our team has developed and commercialized seven additional medicines.

Since then our team has developed and commercialized seven additional medicine.

Speaker 3: Pioneer ground breaking first and transform the lives of countless patients.

Our new year to groundbreaking first and transform the lives of countless patients.

Jean-Jacques Bienaimé: BioMarin is now a profitable, fully integrated industry leader and one of the most successful biotech company, with annual revenues anticipated to be more than $2.4 billion this year, and also expected to grow to close to $3 billion next year. BioMarin is extremely well-positioned for substantial future growth and profitability. Our market cap has grown from around $300 million when I started as CEO, to more than $15 billion today. Our stock price went from $6 when I joined as CEO 18 years ago, to over $83 today at the close. So we have generated returns greater than the XBI index over the past 1 year, 3 years, 5 years, and 18 years.

What I'm wondering is now profitable fully integrated industry leader and one of the most successful biotech company with annual revenues anticipated to be more than $2 $4 billion. This year and also expect it to grow to close to $3 billion next year.

Speaker 3: Our marine is now a profitable, fully integrated industry leader and one of the most successful biotech company with annual revenues anticipated to be more than 2.4 billion dollars this year. And also expected to grow to close the $3 billion next year.

Speaker 3: BioMarine is extremely well positioned for substantial future growth and profitability.

I am AURELIA is extremely well positioned for substantial future growth and profitability.

Speaker 3: Our market cap has grown from around $300 million when I started as CEO to more than $15 billion today.

Our market cap has grown from Iran.

$800 million when I started as CEO to more than $15 billion today.

Our stock price went from $6 when I joined US you 18 years ago to over $83 to the other close.

Speaker 3: Our stock price went from $6 when I joined at C-18 years ago to over $83 to the other close.

Speaker 3: So we have generated returns greater than the XBI index over the past one year, three years, five years, and eight years.

So we have generated returns greater than DXP index over the past one year three years five years.

Yes.

Speaker 3: Bauerin is now a profitable 40-degree industry leader with annual revenues of more than $2 billion and extremely well-positioned for substantial future growth and profitability.

Jean-Jacques Bienaimé: BioMarin is now a profitable, fully integrated industry leader with annual revenues of more than $2 billion and extremely well-positioned for substantial future growth and profitability. I, you know, we have expanded from operating only in the United States when I started with the company, with, at that time, 300 employees, to operating already in almost 80 countries around the world and more than 3,000 employees. As the company is poised for its next phase of growth with the recent introduction of VOXZOGO and ROCTAVIAN, this is the ideal time to hand over the reins to the next leader. In the search, the board focused on finding a successor with experience driving commercial growth and operational excellence, while at the same time bringing a deep understanding of the science and a strong background in product development.

Although it is now a profitable fully integrated industry theater with annual revenues of more than $2 billion, and you're extremely well positioned for substantial future growth and profitability.

Yeah.

Yeah, you know.

Speaker 3: We have expanded from operating only in the United States.

We have expanded our.

Operating only.

In the United States.

Speaker 3: when I started with a company with now, at that time 300 employees to sitting already seen in almost 80 countries around the world and more than 3000 employees.

When I started with the company.

Now that sounds 300, I'm pleased to sitting already seen in almost 80 countries around the world and more than 3000 employees.

Speaker 3: The company is supposed for its next phase of growth with a recent introduction of Voxogo and Rock-Davidian. This is the ideal time to hand over the reins to the next leader.

As the company exposed through its next phase of growth with the recent introduction of box Sogo. Andrew gave you and this is the ideal time to hand over the range to the next leader.

Speaker 3: In the search, the board focused on finding a successor with experience driving commercial growth and operational excellence, while at the same time bringing a deep understanding of the science and a strong background in product development.

In the search the board is focused on finding a successor with experience driving commercial growth and operational excellence.

While at the same time, bringing a deep understanding of the science and is strong.

In product development.

Jean-Jacques Bienaimé: We are confident that Alexander perfectly meets those criteria. As CEO of Genentech, Alexander led the commercialization of 10 new products in therapeutic areas, including cancer and hemophilia. Serving as CEO of this industry-leading, innovative and impactful company over the past 18 years has been the highlight of my career. We have built an incredible organization with great potential to be further unlocked by Alexander and the team. While I will miss working with our talented and inspiring employees day in and day out, I am confident that the company is well positioned for future success. So to those on the call today, thank you for your engagement and support over the years. I still also want to emphasize that I will still be involved with the company and, I will still be very involved with biotech in general, as you will find out in the future.

Speaker 3: We are confident that Alexander perfectly meets the loose criteria, a CEO of Genentex, and Alexander led the commercialization of 10 new products in teoputics areas, including cancer and hemophilia.

We are confident that Alexander perfectly meets those criteria.

CEO of genetics, Alexander let Leslie.

That's a commercialization of 10, new products in COPD areas, including cancer on hemophilia.

Speaker 3: Having a CEO of this industry in innovative and impactful company over the past 18 years has been the highlight of my career. We have built an incredible organization with great potential to be further unlocked by Alexander and the T.

Joking as CEO of this industry, leading innovative and impactful company.

Over the past 18 years has been the highlight of my career we.

We had deals an incredible organization with great potential to be further unlocked.

Hi, Alexander entity.

Speaker 3: But I will miss working with our talented and inspiring employees Dean and they out. I'm confident that the company is well positioned for

But I will miss working with our talented and inspiring employees day in and day I'll tell you I'm confident that.

But the company is well positioned for future success.

Speaker 3: So to do it on the call today, thank you for your engagement and support.

Did you lose on the call today and thank you for your engagement and support.

Speaker 3: over the years. I still also want to emphasize that I will still be involved with the company and I will still be very involved with biotech in general as you will find out in the future.

Over the years I still also want to emphasize that I will still be involved with the company.

And I will still be very involved with biotech in general as you will find out in the future.

Yeah.

Speaker 3: So now let's turn back to the quality results. We are very pleased with the results in the third quarter.

Jean-Jacques Bienaimé: So, now let's turn back to the quarterly results. We were very pleased with the results in the third quarter, recording 15% total revenue growth compared to the third quarter last year, and continued strong growth in earnings per share faster than revenue. These results were driven primarily by VOXZOGO, which remains on track to become our first blockbuster product. We saw strong demand and uptake in the quarter, and we expect this trend to continue. In the third quarter, more than 300 additional children began treatment with VOXZOGO since the end of Q2, and additional strong growth is expected in Q4. As a result, today, we increased full year 2023 VOXZOGO guidance to between $435 million and $455 million.

So now let's turn back to the quarterly results. We are very pleased with the results in the third quarter.

Speaker 3: According 15% total revenue growth compared to the third quarter last year and continued strong growth in earnings per share passed on the revenue.

Regarding 15% total revenue growth compared to the third quarter last year and continued strong growth in earnings per share faster than revenue.

Speaker 3: These results were driven primarily by Voxogo, which remains on track to become our first blockbuster butters. With those strong demand that have taken in the quarter and we expect this to end to continue.

These results were driven primarily by Ewok, Sogou, which remains on track to become our first blockbuster products, we saw strong demand and uptake in the quarter and we expect this trend to continue.

Speaker 3: In the third quarter, more than 300 additional children become treatment with Voxlogo since the end of Q2. An additional strong growth is expected in Q4. As a result, today we increase full year 2020, Voxlogo riders to between 435, 135, 455.

In the third quarter more than 300 additional children become began treatment with oxoco since the end of Q2.

And additional strong growth is expected in Q4.

As a result today, we increased full year 2000, twenty's feedbox over there either.

So between 47 535 million and 455 million.

With recent U S and European approvals of OXXO goes eight expansions all agents in the U S and for children Ages four months in Northern Europe.

Jean-Jacques Bienaimé: With recent US and European approvals, VOXZOGO's age expansions, all ages in the US and for children ages 4 months and older in Europe, over 1,000 additional children now have the opportunity to benefit from a longer VOXZOGO treatment window. We are also pleased to share that based on increased field finish manufacturing commitments, VOXZOGO supply is planned to increase from 23 levels to Q1 and Q2 in 2024, and is expected to be fully unconstrained by mid-year 2024. Turning now to ROCTAVIAN, where we have made tremendous progress building and connecting the network that will facilitate patient treatment. We're laying the groundwork for patient access to ROCTAVIAN. It has taken time, but has been an essential step to ensuring access and reimbursement.

Speaker 3: With recent US and European approvals, Voxogos age expansions, all ages in the US, and for children ages four months and older in Europe , over 1,000 additional children now have the opportunity to benefit from a longer Voxogos treatment window.

Over 1000 additional trailers children now have the opportunity to benefit from a longer walk sogo treatment window.

Speaker 3: We are also pleased to share that based on increased hill finish manufacturing commitments.

We are also pleased to share that based on increased COPD manufacturing commitments.

Speaker 3: Voxog was supplied, it's planned to increase from 23 levels to the first and second quarter and 2024 and is expected to be fully unconstrained by mid-year 2024.

So the supply is planned to increase from 23 levels.

The first and second quarter in 2024 and is expected to be fully and constrained by mid year 2024.

Speaker 3: Building down to Roquevian, where we have made tremendous progress building and connecting the network that will facilitate patient treatment, we'll be laying the groundwork for our patient access to Roquevian. It has taken time but has been an essential step to ensuring access and reimbursement.

Turning now to <unk>, where we have made tremendous progress building and connecting the network.

We will facilitate patient treatment.

Well be laying the groundwork for our patient access to Royal Caribbean.

I was taken.

Time, but has been an essential step to ensuring access and reimbursement.

Speaker 3: Well, over 100 people have been tested for rock-tab-ion, K85 eligibility globally.

Jean-Jacques Bienaimé: Well, over 100 people have been tested for ROCTAVIAN AAV5 eligibility globally, which is the first step that a patient needs to do to determine if he can be treated with ROCTAVIAN. The seronegativity rate on the samples that were tested to date is approximately 70%. So 70% of the patients tested are eligible for ROCTAVIAN. They still need to have a liver scan, but most of the ineligibility comes from the AAV5 positivity. And we expect the testing momentum to continue as awareness and access increases. While it has taken more time than anticipated to get global healthcare system wired to facilitate ROCTAVIAN treatment, we remain encouraged by the high level of interest and demand from patients, physicians, patient advocacy groups, Hemophilia Treatment Centers, and payers.

Well over 100 people they'll be tested for Roque gave you an 85 eligibility globally.

Speaker 3: which is the first step that a patient needs to do to determine if he can be treated as a Floritavian. The seronegativity rate on the samples that were tested today is approximately 70%.

Which is the first step that a patient needs to do to determine if he can be treated with TVN.

The seronegative if he raised on the samples.

We have tested to date is approximately 70% to 70% of a patient tests that are eligible.

Speaker 3: 70% of the patient tested are eligible for Roquevio, and they still need to have to leave a scan, but most of the eligible outcomes from the AB5 positivity.

For Rajeev, you and they still need to have but he was scan, but most of the either you build he comes from the 85 positivity.

Speaker 3: And we expect the testing momentum to continue as awareness and access increases.

And we expect the destiny momentum continue as awareness and access increases.

Speaker 3: Well, it has taken more time than an easy pay to give global healthcare system work to facilitate routine treatment will remain encouraged by the high level of interest and demand from patients, physicians, patient advocacy groups, chemotherapy treatment centers, and Bayou.

While it has taken more time than anticipated to get global health care system wired to facilitate rode even treatment. We remain encouraged by the high level of interest and demand from patients.

You shouldn't advocacy groups hemophilia treatment centers and payers.

Speaker 3: In Europe , we have strong progress in the quarter and expect to have final German and Italian prices for Octavion published by year and in the US, significant momentum connecting the network to city treatment is occurring.

Jean-Jacques Bienaimé: In Europe, we have strong progress in the quarter and expect to have final German and Italian prices for ROCTAVIAN published by year-end. In the US, significant momentum connecting the network to facilitate treatment is occurring. Jeff will provide more detail on launch progress and our updated expectations for ROCTAVIAN. Turning to BioMarin's record year-to-date revenues of nearly $1.8 billion in the first nine months of the year, it represents 14% year-over-year growth, even with lower than expected contributions from ROCTAVIAN. These results underscore the strength of our business and the achievement of double-digit revenue growth, and our profitability objectives as set out at the start of the year. The business is poised for strong growth in 2024 and beyond.

In Europe, Yes, we have a strong progress in the quarter and expect to have final German and Italian prices for Octavian.

By year end.

And in the U S significant momentum connecting the network to take treatment it is occurring.

Speaker 3: Yeah, we provide more detail on launch progress and our updated expectations for Octavio.

Yes, if you could provide more detail on loss progress and our updated expectations for David.

Turning to borrow means record year to date revenues of nearly $1 $8 billion in the first nine months of the year.

Speaker 3: According to Bahamian's record, here today revenues of really nearly $1.8 billion in the first nine months of the year.

Speaker 3: represents 14% year-over-year growth, even with lower than expected contributions.

This represents 14% year over year growth.

Even with lower than expected contributions from Rob Dave Yes.

Speaker 3: His results underscore the strength of our business and the achievement of double digit revenue growth and our profitability objectives as it out as the start of the year. The business is voice for strong roles in 24 and beyond.

These results underscore the strength of our business and the achievement of a double digit revenue growth.

And our profitability objectives as set out at the start of the year.

The business is poised for strong growth in 'twenty four and beyond.

Jean-Jacques Bienaimé: With VOXZOGO continuing to exceed expectations, sorry, and ROCTAVIAN access and reimbursement in place for meaningful uptake next year, we believe BioMarin revenues are set up to approach $3 billion in 2024. In summary, we have built an industry-leading company. The strategy to create a sustainably profitable and growing business that provides patients with innovative and essential medicine is coming to fruition. The future looks very bright for BioMarin, and I want to thank my colleagues for the tremendous collaboration and what we have built together. So thank you for continued support. I will now turn the call over to Jeff Ajer to discuss the commercial business update. Jeff?

With October continuing to exceed expectations.

Speaker 3: Sorry, and we're cave in access. We are inverse being placed for a meaningful update next year. We believe our revenues are set up to approach $3 billion in 2024. In summary, we are built.

Sorry, Andrew gave an access and.

On reimbursement in place for a meaningful uptick next year. We believe biomarin revenues are set up to approach $3 billion in 2024.

In summary, we have built an industry leading company.

Speaker 3: So I did need to create a sustainably profitable and growing business that provides patients with innovative and essential medicine is coming to fruition.

The strategy to create a sustainably profitable and growing business that provides patients with innovative and essential medicine is coming to fruition.

Speaker 3: This future looks very bright for Baham Ren. And I want to thank my colleagues for the tremendous collaboration and for what we have built together.

The future looks very bright for Biomarin and I want to thank my colleagues for their tremendous collaboration and what we have built together.

Speaker 3: So thank you for continuing to support. I will knock down the call over to Jeff. Age you're to discuss the commercial business of this. Jeff, thank you JJ in so many ways.

So thank you for continued support and I will now turn the call over to.

The jet figure to discuss the commercial business update Jeff. Thank you J J in so many ways.

Jeff Ajer: Thank you, JJ, in so many ways. I'm very pleased with our commercial performance in Q3, resulting in $581 million in total revenues and representing 15% growth year-over-year, including KUVAN, and 20% growth, excluding KUVAN. Turning to VOXZOGO, as JJ mentioned, today, we updated full-year guidance to between $435 and $455 million, representing approximately 165% year-over-year growth at the midpoint. Appreciating that the run rate for Q4 and full-year 2023 may seem conservative based on VOXZOGO revenues of $123 million in Q3, we note the impact from supply constraints discussed last quarter continue to limit growth through the end of this year.

Speaker 4: I'm very pleased with our commercial performance in the third quarter, resulting in $581.00 in total revenues, and representing 15% growth year-over-year, including Cuvan, and 20% growth excluding Cuvan.

I'm very pleased with our commercial performance in the third quarter, resulting in $581 million in total revenues and representing 15% growth year over year, including Kuban and 20% growth excluding Kuban.

Speaker 4: Turning to Vox Sago, as JJ mentioned, today we updated full year guidance to between $435 and $455 million, representing approximately 165% your over your growth at the midpoint.

Turning to box Sogo as J J mentioned today, we updated full year guidance to between 135 and $455 million, representing approximately 165% year over year growth at the midpoint.

Speaker 4: Appreciating that the run rate for the fourth quarter in all year 2023 may seem conservative based on boxfogo revenues of 123 million dollars in the third quarter. We note the impact from supply constraints last quarter continued limit growth through the end of the year

Adding that the run rate for the fourth quarter and full year 2023 may seem conservative based on box I'll go revenues of $123 million in the third quarter. We note the impact from supply constraints discussed last quarter continue to limit growth through the end of this year.

Speaker 4: While we continue to closely manage new patient growth and limit inventory stocking, we expect to fulfill smaller orders from various regions in Q4 while at the same time adding hundreds of new patients in the fourth quarter. As a reminder, new patient starts with Vaxhogo in the fourth quarter will represent only a fraction of a full year revenue patient in 2023.

Jeff Ajer: While we continue to closely manage new patient growth and limit inventory stocking, we expect to fulfill smaller orders from various regions in Q4, while at the same time adding hundreds of new patients in the fourth quarter. As a reminder, new patient starts with VOXZOGO in the fourth quarter will represent only a fraction of a full year revenue patient in 2023. Importantly, we expect approximately 2,600 patients to be receiving VOXZOGO treatment by year-end 2023. VOXZOGO supply is planned to increase from 2023 levels through Q1 and Q2 of 2024 and is expected to be fully unconstrained by mid-year next year. These commitments will ensure that we have ample supply to exceed full year 2024 consensus, currently at $624 million, and support growth beyond 2024.

While we continue to closely manage new patient growth and limit inventory stocking, we expect the fulfill smaller orders from various regions in Q4.

While at the same time, adding hundreds of new patients in the fourth quarter.

As a reminder, new patient starts with box over on the fourth quarter.

Present, only a fraction of our full year revenue patient in 2023.

Speaker 4: importantly we expect approximately 2600 patients to be receiving botzogo treatment by urine 2023

Importantly, we expect approximately 2600 patients to be receiving box sogo treatment by year end 2023.

Speaker 4: Boxego Supply has planned increase from 2023 levels through the first and second quarters of 2024 and is expected to be fully unconstrained by mid-year next year.

So the supply is planned to increase from 2023 levels through the first and second quarters of 'twenty 'twenty four and is expected to be fully unconstrained by mid year next year.

Speaker 4: These commitments will ensure that we have ample supply to exceed full year 2020-24 consensus. Currently at $624 million and support growth beyond 2020.

These commitments will ensure that we have ample supply to exceed full year 2024 consensus currently at $624 million and support growth beyond 2024.

Jeff Ajer: At the end of Q3, more than 2,300 children with achondroplasia in 38 markets were being treated with VOXZOGO. Uptake to date represents 12% penetration of indicated patients in BioMarin's commercial footprint, highlighting the significant growth potential remaining. With the opportunity to now treat up to an additional 1,000 children following the recent age expansion approvals in the US and Europe, we look forward to the possibility of the youngest children in these regions benefiting from VOXZOGO therapy. Turning now to ROCTAVIAN, we are pleased to share that the second commercial patient was treated today in Germany. As JJ highlighted, we continue to make progress connecting the network that will facilitate treatment. 2023 has turned out to be the year of building that network to ensure and support meaningful patient uptake in 2024.

At the end of the third quarter more than 2300 children with achondroplasia in 38 markets were being treated with box sogo.

Speaker 4: At the end of the third quarter, more than 2300 children with ACounder Plasia in 38 markets were being treated with BoxFogo. Uptake to date represents 12% penetration of the indicated patients in Biomurin's commercial footprint, highlighting the significant growth potential remaining.

Uptake to date represents 12% penetration of indicated patients in biomarin commercial footprint, highlighting the significant growth potential remaining.

Speaker 4: With the opportunity to now treat up to an additional 1,000 children following the recent age expansion approvals in the US and Europe , we look forward to the possibility of the youngest children in these regions benefiting from Baxogothera.

With the opportunity and now treat up to an additional 1000 children. Following the recent age expansion approvals in the U S and Europe, we look forward to the possibility of the youngest children in these regions benefiting from box I'll go therapy.

Speaker 4: Turning now to Raktavian, we are pleased to share that the second commercial patient was treated today in Germany.

Turning now Directv and we are pleased to share that the second commercial basin was treated today in Germany.

Speaker 4: As JJ highlighted, we continue to make progress connecting the network that will facilitate treatment.

As JJ highlighted we continue to make progress connecting the network that will facilitate treatment.

Speaker 4: 2023 has turned out to be the year of building that network to ensure and support meaningful patient uptake in 2024.

<unk> thousand 23, and it's turned out to be the year of building that network to ensure and support meaningful patient uptake in 2024.

Speaker 4: Starting with Europe , we have tentative agreement with the German National Association of Statutory Health Insurance Funds or GKV on the final German prize for rock caveans, which we expect to be formalized and published by the end of the year.

Jeff Ajer: Starting with Europe, we have tentative agreement with the German National Association of Statutory Health Insurance Funds, or GKV, on the final German price for ROCTAVIAN, which we expect to be formalized and published by the end of the year. As we have said previously, we believe this important step, given the unique treatment and reimbursement profile of ROCTAVIAN, will open up the access funnel to treatment in Germany. We are pleased to share that in Germany, 60 people are known to be eligible based on CDX testing for AAV5 antibodies. The vast majority of those are in follow-up for next steps. As expected, the seronegativity rate of about 70% is in line with our study results and consistent with expectations. We are also pleased to report that there are now 10 sites in Germany testing for eligibility.

Starting with Europe, we have tentative agreement with the German National Association of statutory health insurance funds or GTA V. On the final German price for <unk>, which we expect to be formalized and published by the end of the year.

Speaker 4: As we have said previously, we believe this important step, given the unique treatment and reimbursement profile of Rock Avian will open up the access funnel to treatment in Germany.

As we have said previously we believe this important step given the unique treatment and reimbursement profile of Octavian will open up the access bundle the treatment in Germany.

We are pleased to share that in Germany, 60 people are known to be eligible based on C. Dx testing for AAV five antibodies.

Speaker 4: We are pleased to share that in Germany, 60 people are known to be eligible based on CDX testing for AAV-5 antibodies.

Speaker 4: The vast majority of those are in follow-up for next step.

The vast majority of those are in follow up for next steps as.

Speaker 4: As expected, the seronegativity rate of about 70% is in line with our study results and consistent with expectation.

As expected the Cerro negativity rate of about 70% is in line with our study results and consistent with expectations.

Speaker 4: We are also pleased to report that there are now ten sites in Germany testing for eligibility.

We are also pleased to report that there are now 10 sites in Germany testing for eligibility.

Speaker 4: With a large full of eligible patients and the final German price expected to be published soon, we are optimistic about patient uptake rates in 2024.

Jeff Ajer: With a large pool of eligible patients and the final German price expected to be published soon, we are optimistic about patient uptake rates in 2024. Important to note is that the first patient treated resulted in net revenue of approximately $800,000. Gross to net revenue adjustments going forward under the GKV agreement will be different than for the first patient reported, but our overall expectations are to maintain similar net revenue per patient in Germany, noting the impact of weight-based dosing and variable weight of patients. In Italy, negotiations with the Italian Medicines Agency are going well, and we expect to have a final price published by year-end. Italy represents then our second major European market that has agreement on price and reimbursement, opening the door to ROCTAVIAN access likely in early 2024.

With a large pool of eligible patients and the final German prices expected to be published soon we are optimistic about patient uptake rates in 2024.

Speaker 4: Born to note is that the first patient treated resulted in net revenue of approximately $800,000.

Important to note is that the first patient treated resulted in net revenue of approximately $800000.

Speaker 4: Gross to net revenue adjustments going forward under the GKB agreement will be different than for the first patient reported. But our overall expectations are to maintain similar net revenue per patient in Germany, noting the impact of weight-based dosing and variable weight of patients.

Gross to net revenue adjustments going forward under the <unk> agreement will be different than for the first patient reported but our overall expectations are to maintain similar net revenue per patient in Germany, noting the impact of weight based dosing and variable weight of patients.

Speaker 4: Italy negotiations with the Italian medicine agency are going well, and we expect to have a final price published by a year end. Italy represents then our second major European market that has agreement on price and reimbursement opening the door to rock, KVN access likely in early 2024. We continue to work with the authorities in France and expect pricing negotiations to extend into 2024.

Italy negotiations with the Italian medicines agency are going well and we expect to have a final price published by year end.

Italy represents then our second major European market that has agreement on price and reimbursement opening the door in Iraq, JV and access likely in early 2024, we continue to work with the authorities in France and expect pricing negotiations to extend into 2024.

Jeff Ajer: We continue to work with the authorities in France and expect pricing negotiations to extend into 2024. It has taken several months in the US following approval, to align stakeholders and to facilitate first patients treated. In general terms, this is a result of that unique profile of one-time reimbursement and treatment with ROCTAVIAN. Navigating hospital administration and finance adds a level of complexity for hemophilia treatment centers or HTCs, leading to the final step of patient treatment with ROCTAVIAN. We expect that these administrative steps taken at the HTC level, while time-consuming, are one-time events and essential to facilitate patient access going forward. In short, the groundwork completed in 2023 is expected to enable simplified access to ROCTAVIAN, drive more and more AAV5 eligibility testing, and giving us confidence in the prospects for 2024.

Speaker 4: It has taken several months in the U.S. following approval to line stakeholders and to facilitate first patients treated. In general terms, this is a result of that unique profile of one-time reimbursement and treatment with rock tivion.

As taken several months in the U S. Following approval through aligns stakeholders into it facilitate first patients treated.

In General terms. This is a result of that unique profile, a onetime reimbursement and treatment with <unk>.

Speaker 4: Navigating hospital administration and finance adds a level of complexity for him affiliate treatment centers or HTC's, leading to the final step the patient treatment with Rod Cabean.

Navigating Hospital administration, and finance adds a level of complexity for hemophilia treatment centers, where htc's, leading to the final step of patient treatment with Roth caveats.

We expect that these administrative steps taken at the HTC level, while time consuming are one time events and the central that facilitate patient access going forward.

Speaker 4: We expect that these administrative steps taken at the HTC level while time consuming are one-time events and essential to facilitate patient access going forward. In short, the groundwork completed in 2023 is expected to enable simplified access to Rock-Tavian. Drive more and more AAV5 eligibility testing and giving us confidence in the prospects for 2024.

Short groundwork completed in 2023 is expected to enable simplified access to rock Davey and drive more and more AAV five eligibility testing and giving us confidence in the prospects for 2024.

Jeff Ajer: However, with less than two months remaining in the year and the holiday season beginning soon, we are lowering full-year ROCTAVIAN 2023 guidance to less than $10 million. Moving now to specific steps completed this quarter, starting with the issuance of coverage policies across payers, both private and public. We are pleased to share that nearly two-thirds of all covered lives in the United States have been issued coverage policies to date, representing approximately 205 million people. Importantly, the vast majority are in line with label or clinical trial criteria and are thus favorable in meeting expectations. Additionally, we are encouraged by the rapid uptake of the outcomes-based warranty, with signed warranties in place representing more than 95 million US lives. We have also received notice that the J-Code for ROCTAVIAN has been assigned, effective 1 January 2024.

Speaker 4: However, with less than two months remaining in the air, and the holiday season beginning soon, we are lowering full year rock-tabian 2023 guidance to less than $10 million.

However, with less than two months remaining in the air and the holiday season. Beginning soon we are lowering full year activity in 2023 guidance the less than $10 million.

Speaker 4: Moving out a specific steps completed this quarter, starting with the issuance of coverage policies across payers, both private and public, we are pleased to share that nearly two thirds of all covered lives in the United States have been issued coverage policies today, representing approximately 205 million people. Importantly, the vast majority are in line with label or clinical trial criteria, and are thus favorable in meeting expectations.

Moving now to specific steps completed this quarter, starting with the issuance of coverage policies across payers, both private and public. We are pleased to share that nearly two thirds of all covered lives in the United States have been issued coverage policies to date, representing approximately 205 million people.

Paul.

Ordinarily the vast majority are in line with label or clinical trial criteria and are thus favorable and meeting expectations.

Speaker 4: Additionally, we are encouraged by the rapid take of the outcomes-based warranty with signed warranties in place representing more than 95 million US dollars.

Additionally, we are encouraged by the rapid uptake of the outcomes based warranty with signed warranties in place representing more than 95 million U S lives.

Speaker 4: We have also received notice that the J-code for Raktavian has been assigned effective January 1, 2024. The permanent J-code is expected to accelerate Pairs prior authorization review process, ultimately speeding time to patient treatment.

We have also received notice that the J code for rock Damien has been assigned effective January one 2020 for.

Jeff Ajer: The permanent J-Code is expected to accelerate payers' prior authorization review process, ultimately speeding time to patient treatment. Taken together, these developments are a strong indication of the value the US health system places in a cost-effective, one-time gene therapy. We stated in our approval in Q2, two earnings call, we expect it could take patients 2 to 5 months to complete the steps necessary before treatment with ROCTAVIAN. The commercial team has been hard at work implementing a number of tactics to prepare physicians, HTCs, and patients for the opportunity to benefit from ROCTAVIAN. With respect to site readiness, HTCs are being prepared and educated on how to administer ROCTAVIAN. We have targeted the largest and most capable HTCs for site readiness by year-end, a number of which have readiness plans before the end of this year.

The permanent J code is expected to accelerate payers prior authorization review process, ultimately speeding time to patient treatment.

Speaker 4: Akin together, these developments are a strong indication of the value of the US health system places in a cost effective one-time gene therapy.

Speaker 4: We stated in our approval in Q2 earnings call, we expect it could take patients two to five months to complete the steps necessary before treatment with Rock Tavius.

Speaker 4: The commercial team has been hard at work, implementing a number of tactics to prepare physicians, HTC's and patients for the opportunity to benefit from Octavian. With respect to site readiness, HTC's are being prepared and educated on how to administer Octavian. We have targeted the largest and most capable HTC's for site readiness by year end, a number of which have readiness plans before the end of this year.

Jeff Ajer: Increasing HCP and patient awareness in the US was also an important focus in the quarter. A number of activities were conducted, including national HCP and patient webinars, and the third annual Global Gene Therapy Scientific Summit, sponsored by BioMarin. We are especially encouraged by the number of local patient advocacy groups which have been willing to host branded patient awareness programs for ROCTAVIAN. Medical eligibility, including CDX testing, is underway, and as a result, the US patient funnel is beginning to take shape, giving us confidence in the prospects for treating patients going forward. In conclusion, we are on track to achieve 2023 full-year total revenue guidance, driven by the strength of our enzyme product revenues and continued high demand for VOXZOGO globally.

Speaker 4: Increasing HCP and patient awareness in the US was also an important focus in the quarter. A number of activities were conducted, including national HCP and patient webinars, and the third annual global gene therapy scientific summit sponsored by Biomorin.

Speaker 4: We are especially encouraged by the number of local patient advocacy groups which have been willing to host branded patient awareness programs for ROTKIVIA.

By the number of local patient advocacy groups, which have been willing to host branded patient awareness programs <unk>.

Speaker 4: Medical eligibility, including CDX testing, is underway. And as a result, the US patient funnel is beginning to take shape, giving us confidence in the prospects for treating patients going forward. In conclusion, we are on track to achieve 2023 full-year total revenue guidance driven by the strength of our inside product revenues and continued high demand for BotsoGo globally.

Medical eligibility, including C. D accessing is underway and as a result, the worst patient bunnell is beginning to take shape, giving us confidence in the prospects for treating patients going forward.

In conclusion, we are on track to achieve 2023 full your total revenue guidance driven by the strength of our inside product revenues and continued high demand for <unk> globally.

Speaker 4: Looking ahead, we've leave the network built to facilitate treatment with Rothkivian combined with our high expectations for Botsego and strong enzyme business will enable by a Miranda approach close to $3 billion in total revenues in 2024.

Jeff Ajer: Looking ahead, we believe the network built to facilitate treatment with ROCTAVIAN, combined with our high expectations for VOXZOGO and strong enzyme business, will enable BioMarin to approach close to $3 billion in total revenues in 2024. Thank you for your attention, and I will now turn the call over to Hank to provide an R&D update. Hank?

Head, we'd leave the network built to facilitate treatment <unk> combined with our high expectations for <unk> and strong enzyme business will enable by Miranda approach close to $3 billion in total revenues in 2024.

Speaker 4: Thank you for your attention and I will now turn the call over to Hank, Reviving R and D Update. Hank? Thanks, Jess. Thank you, JJ. The privilege is to start.

Thank you for your attention and I will now turn the call over to heck, providing R&D update <unk>.

Hank Fuchs: Thanks, Jeff. Thank you, JJ. The privilege is ours. Thank you all for joining us today. We provided a thorough update on our earlier-stage pipeline at R&D Day in September. If you missed it, please find the full presentation on BioMarin's IR website. Starting with VOXZOGO in the quarter and shortly beyond, we received some important updates from global health authorities, resulting in expanded access to children, younger children in both the US, at where it is now approved in all age groups, and in Europe for children four months of age and older. We're gratified that the youngest children now have the possibility of greater potential long-term benefits from treatment with VOXZOGO. I wanna thank not just the usual clinical and regulatory teams at these finish line efforts, but also the extended BioMarin team of safety professionals, biometrics professionals, operations, pharmacology.

Thanks, Jeff.

Changes a privilege skinner's.

Speaker 5: And thank you all for joining us today. We provided a thorough update on our earlier state pipeline at R&D Day in September . So if you missed it, please find the full presentation on BioMorons IR website.

And thank you all for joining US today, we provided a thorough update on our earlier stage pipeline at our D day of September. So if you missed it. Please find the full presentation on Byron's I our website.

Speaker 5: Starting with OxoGo in the quarter and shortly beyond, we received some important updates from global health authorities, resulting in expanded access to children, younger children in both the US and where it has now approved in all age groups, and in Europe for children four months of age and older.

Starting with Oxford, though and a quarter and shortly beyond we received some important updates from global health authorities, resulting in expanded access to children younger children and both the U S. It where it is now approved in all age groups and in Europe for children four months of agent older <unk> that the youngest children now have the possibility of greater potential long.

Speaker 5: We're gratified that the youngest children now have the possibility of greater potential long-term benefits from treatment of Fox OGO. And I want to thank not just the usual clinical and regulatory teams at these finish line efforts.

Term benefits from treatment <unk> ago, and I'm Gonna say, not just the usual clinical and regulatory teams at the finish line efforts, but also the extended <unk> team of safety professionals biometrics professionals operations pharmacology really the whole entire fully integrated team played a strong role in enabling this advancement and access <unk>.

Speaker 5: but also the extended bomber and team of safety professionals, biometrics, professionals, operations, pharmacology, really the whole entire fully integrated team play this strong role in enabling this advancement in access to patients.

Hank Fuchs: Really, the whole entire fully integrated team played a strong role in enabling this advancement and access of patients. So on behalf of the families and children with achondroplasia, thank you all. As mentioned last quarter, we have solidified our plans to begin the pivotal program using VOXZOGO for the treatment of hypochondroplasia, with the natural history arm of the study expected to begin this quarter. The pathogenesis and epidemiology are similar between achondroplasia and hypochondroplasia. While hypochondroplasia presents with a milder phenotype in some patients in terms of short stature, disproportionally in clinical manifestations, we plan to target the population with the most severe form of the disease, and therefore, the highest unmet need, with high deficits beyond 3 standard deviations below their means.

Speaker 5: So on behalf of the families and children with ACONTRA pleasure, thank you all.

<unk> so on behalf of the families and children with a contemplation. Thank you. Paul is mentioned last quarter, we have solidified our plans to begin the pivotal program using Vox ogre for the treatment of type of contemplated up with a natural history arm of the study expected to begin this quarter, the pathogenesis and epidemiology.

Speaker 5: As mentioned last quarter, we have solidified our plans to begin the pivotal program using Vactoga for the treatment of hypochondriplasia with the natural history arm of the study expected to begin this quarter. The pathogenesis and epidemiology are similar between acondriplasia and hypochondriplasia.

Are similar between achondroplasia and hypo contract pleasure, what type of contemplation presents with a minor theater my elder phenotype in some patients in terms of short stature disproportionately in clinical manifestations, we plan to target the population with the most severe form of the disease and therefore, the highest unmet needs with high deficits beyond three standard deviations <unk>.

Speaker 5: Well, hypocontrolasia presents with a minor, milder phenotype in some patients in terms of short stature, disproportionately in clinical manifestations. We plan to target the population with the most severe form of the disease and therefore the highest button that needs with high deficits beyond three standard deviations below that it means. With a line-in from FDA on the single registration and abling, 52-week randomized double blind.

<unk> means with a line from F. D. A on a single registration, enabling 52 week randomized double blind placebo controlled study with a primary outcome of annualized growth velocity, we look forward to initiating the treatment arm of the study in 2024 following the natural history running portion in.

Hank Fuchs: With alignment from FDA on the single registration, enabling 52-week randomized double-blind placebo-controlled study with a primary outcome of annualized growth velocity, we look forward to initiating the treatment arm of the study in 2024, following the natural history run-in portion. In addition, we plan to initiate two new clinical programs next year with VOXZOGO, one in idiopathic or polygenic short stature, and the other in defined genetic short stature conditions. We know that CNP is a pivotal physiologic stimulator of endochondral bone growth on the level of the growth plate. Further, human genetic studies of CNP and not FGFR3, demonstrate that even modest nudges in CNP signaling have a significant impact on final adult height. This supports our confidence that VOXZOGO can deliver on the transformational improvement in growth across many forms of skeletal impairment, while retaining the strong safety profile demonstrated in achondroplasia.

Speaker 5: placebo controlled study with a primary outcome of annualized growth fall out velocity. We look forward to initiating the treatment arm of the study in 2024 following the natural history run in portion.

Operator: Hello and welcome to the Biomarin Pharmaceutical Third Quarter 2023 conference call. All lines have been placed on a 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 star one on your telephone keypad.

Tracy McCarty: If you would like to withdraw your question, again, press star one will now turn the conference over to Tracy McCarty, head of investor relations, please go ahead. Thank you, JL and thank you all for joining us today to remind you this non confidential presentation. It contains forward-looking statements about the business prospects of Biomarin Pharmaceutical Inc, including expectations regarding Biomarin's financial performance, commercial products and potential teacher products in different areas of therapeutic research and development.

Speaker 5: In addition, we plan to initiate two new clinical programs next year with Foxhogo, one in idiopathic or polygenic short stature, and the other in defined genetic short stature conditions. We know that CNP is a pivotal physiologic stimulator of endocundral bone growth on the level of the growth plate.

In addition, we plan to initiate two new clinical programs next year with box ago. One in idiopathic were apologetic short stature and the other in define genetic short stature conditions, we know that C. N P. As a pivotal physiological stimulator of <unk> growth on the level of the growth plate further human genetic studies of C. M. P. S.

Speaker 5: further human genetic studies of CNP and not FGFR-3 demonstrate that even modest nudges and CNP signaling have a significant impact on final adult height.

And not F. G F. R. Three demonstrate that even modest images in C. N P signalling have a significant impact on final adult height.

Tracy McCarty: Results may differ materially depending on the progress of Biomarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market, and developments by competitors and those factors detailed in Biomarin's filing with the securities in exchange. There is a range commission, such as 10Q, 10K, and 8K reports.

Speaker 5: This supports a confidence that Vaxogu can deliver on the transformational improvement in growth across many forms of skeletal impairment, while retaining the strong safety profile demonstrated in acondriplasia.

[noise] supports a confidence that back so you can deliver on the transformational improvement and growth across many forms of skeletal impairment while remaining this law retaining the strong safety profile demonstrated in a contemplation for these reasons, we look forward to starting the clinical programs and these additional statue all indications in 2024. Please take.

Hank Fuchs: For these reasons, we look forward to starting the clinical programs in these additional statural indications in 2024. Please take a look at the full R&D Day presentation for the latest update on our seven portfolio candidates discussed in September, and we look forward to keeping you apprised as they progress through development. And thanks for your support, and I'll turn the call over to Brian to update financial results in the quarter. Brian?

Speaker 5: For these reasons, we look forward to starting the clinical programs and these additional stetural indications in 2024.

Speaker 5: Please take a look at the full R&D Day presentation to the latest update in our seven portfolio candidates discussed in September and we look forward to keeping you apprised as they progress through development.

Look at the full R&D day presentation for the latest updates on our seven portfolio candidates discussed in September and we look forward to keeping you apprised as they progress through development and thanks for your support now I'll turn the call over to Brian to update financial results in the quarter, Brian. Thank you <unk> and thank you J J as well.

Tracy McCarty: On the call today from Biomarin Management, our JJVNMA, Chairman and Chief Executive Officer, Jeff Ager, Executive Vice President, Chief Commercial Officer, Hank Fuchs, President Worldwide Research and Development, Greg Geier, Executive Vice President, Chief Technical Officer, and Brian Mueller, Executive Vice President and Chief Financial Officer. We do plan to end this call promptly at 2.30 PM Pacific Time, and we thank you for your understanding.

Speaker 6: And thanks for your support, and I'll turn the call over to Brian to update financial results in the quarter of Brian . Thank you, Hank, and thank you, JJ, as well.

Brian Mueller: Thank you, Hank, and thank you, JJ, as well. Please refer to today's press release summarizing our financial results for full details on Q3 2023. Since JJ and Jeff spoke to our revenue performance for the quarter and future revenue outlook, I will primarily focus on the remainder of our P&L and other key financial updates this quarter. As usual, all results will be available in our upcoming Form 10-Q, which we expect to file tomorrow. We are pleased with our year-to-date revenue growth of 14% and non-GAAP earnings per share growth of 33%, which well positions us to achieve our top and bottom-line financial objectives in 2023, as indicated by our narrowed guidance provided today on total revenue and earnings per share.

Speaker 6: Please refer to today's press release summarizing our financial results for full details on the third quarter of 2020.

Please refer to today's press release summarizing our financial results for full details on the third quarter of 2023.

Speaker 6: Then JJ and Jeff spoke to our revenue performance for the quarter and future revenue outlook. I will primarily focus on the remainder of our P&L and other key financial updates this quarter.

J J and Jeff spoke to our revenue performance for the quarter and future revenue outlook I will primarily focus on the remainder of our piano and other key financial updates this court.

Jeff Ajer: I will now turn the call over to Biomarin's Chairman and CEO, JJVNMA. Thank you, Tracy. Good afternoon and good morning, everybody.

Speaker 6: As usual, all results will be available in our upcoming form 10Q, which we expect to file tomorrow.

As usual all results will be available in our upcoming Form 10-Q, which we expect to file tomorrow.

Jeff Ajer: As you will have seen this afternoon, we announced that I have decided after over 18 years with Biomarin to retire as the Chairman and Chief Executive Officer, respectively, December 1 of this year. In this video, you are aware I recently actually turned 70. I cannot believe it myself, so I will not be surprised to you that sufficient planning has been underway with the board over the past several years. These announcements and the appointment of Alexander Hardy to succeed me as CEO follows a thorough multi-year planning process.

Speaker 6: We are pleased with our year-to-date revenue growth of 14% and non-GAP earnings per share growth of 33% which well positions us to achieve our top and bottom line financial objectives in 2023. As indicated by our narrow guidance provided today, I'm total revenue and earnings per share.

We are pleased with our year to date revenue growth at 14 per cent and non-GAAP earnings per share growth of 33% with well positions us to achieve our top and bottom line financial objectives in 2023 as indicated by our narrowed guidance provided today in total revenue and earnings per share.

Speaker 6: As underscored in today's guidance updates, we continue to expect double-digit revenue growth, coupled with meaningful P&L leverage with GAP and non-GAP earnings per share growth faster than revenue.

Brian Mueller: As underscored in today's guidance updates, we continue to expect double-digit revenue growth, coupled with meaningful P&L leverage, with GAAP and non-GAAP earnings per share growth faster than revenue. Moving on to Q3 results, BioMarin's $581 million of total revenue in the third quarter of 2023 grew 15% compared to the third quarter of 2022, and 19% on a constant currency basis. VOXZOGO's strong performance and healthy growth in the enzyme products marketed by BioMarin drove this result, with some partial offsets of lower KUVAN revenue due to continued generic competition, and ALDURAZYME timing of order fulfillment to Sanofi.

As underscored in today's guidance updates, we continue to expect double digit revenue growth, coupled with meaningful P&L leverage with gap and non-GAAP earnings per share growth faster than rabbit.

Speaker 6: Moving on to Q3 results, I'm a rents $581 million of total revenue in the third quarter of 2023 through 15% compared to the third quarter of 2022. And 19% on a constant currency base.

Moving onto Q3 results <unk> $581 million, a total revenue in the third quarter of 2023 grew 15% compared to the third quarter of 2022, and 19% on a constant currency basis.

Jeff Ajer: Together with the board, we conducted a comprehensive search over the past year, and we are thrilled to have Alexander step into the role. As part of this transition, I would also step away from my position as Chairman of the board of directors. However, I would continue to serve on the board until the 2024 annual meeting of staff stockholders, and I will stay as a consultant to the company for the remainder of 2024. I look forward to working with Alexander on the board during this period to answer a smooth transition.

Speaker 6: BoxoGo's strong performance and healthy growth in the enzyme products marketed by Biamarin drove this

<unk> strong performance and healthy growth and the enzyme products marketed by Biomarin drove this result, with some partial offset of lower Kuban revenue due to continued generic competition and.

Speaker 6: With some partial offsets of lower coup d'an revenue, due to continued generic competition, and Al Dersheim timing of order fulfillment to Sanofi.

<unk> timing, a order fulfillment to Sanofi.

Brian Mueller: The strength of our portfolio, consisting of eight commercial products, allows us continued patience on the pioneering nature of the ROCTAVIAN launch, specifically the ability to maintain our original full year 2023 total revenue growth expectations, which represents 16% year-over-year growth at the midpoint, and our implied Q4 2023 growth rate at the guidance midpoint of over 20%. We remain confident in the long-term prospects of ROCTAVIAN and its potential to drive growth in 2024. With respect to gross margins, Q3 2023 gross margin of 78.5% is an improvement of 1.6 percentage points as compared to the third quarter of 2022.

Speaker 6: The strength of our portfolio, consisting of eight commercial products, allows us to continue patients on the pioneering nature of the rock-taving enlarge. Specifically, the ability to maintain our original full year, 2023, total revenue growth expectations, which represents 16% of year-over-year growth at the midpoint. And our implied Q4 2023 growth rate at the guidance midpoint of over 20%.

The strength of our portfolio consisting of eight commercial products allows us continued patience and the pioneering nature of the rock caving in March specifically the ability to maintain our original full year 2023, total revenue growth expectations, which represents 16% a year over year growth at the mid.

Jeff Ajer: I am pleased that our leading independent director Richard Meyer, with a Randy, will be our new chair of the board. When I joined the company in May 2005, we had just one marketed product and recorded about $26 million in annual revenues. Since then, our team has developed and commercialized seven additional medicines, pioneered groundbreaking firsts and transformed the lives of countless patients. The armoring is now a profitable, fully integrated industry leader, and one of the most successful biotech company with annual revenues anticipated to be more than $2.4 billion this year, and also expected to grow to close the $3 billion next year, here, Biomarin is extremely well-positioned for substantial future growth and profitability.

Jeff Ajer: Our market cap has grown from around $300 million when I started CEO to more than $15 billion today. Our stock price went from $6 when I joined a CEO 18 years ago to over $83 today at the close. So we have generated returns greater than the XBI index over the past one year, three years, five years, and 18 years. Biomarin is now a profitable 40-degree industry leader with annual revenues of more than $2 billion and extremely well-positioned for substantial future growth and profitability.

<unk> and are applied Q4, 2023 growth rate the guidance mid point of over 20 per cent we.

Speaker 6: We remain confident in the long-term prospects of Raktivian and its potential to drive growth in 2024.

We remain confident in the long term prospects of <unk> and its potential to drive growth in 2024.

Speaker 6: With respect to gross margins, Q3 2023 gross margin of 78.5% is an improvement of 1.6% points as compared to the third quarter of 2022.

With respect to gross margins Q3, 20 twenty-three gross margin of 78.5% is an improvement of 1.6 percentage points as compared to the third quarter of 2022.

Brian Mueller: R&D expense in Q3 2023 was $191 million, and SG&A expense was $224 million, which combined together totaled $415 million and grew 11% versus last year. As compared to 15% revenue growth in the quarter, this aligns with our goal of growing operating expenses slower than revenue to deliver operating margin expansion for the full year. The increase in R&D and SG&A was primarily driven by the development of our early-stage pipeline and global commercial launch activities for VOXZOGO and ROCTAVIAN. As we look ahead to Q4, we anticipate that total SG&A and R&D expense will be higher than Q3 on a dollar basis due to the same drivers. Moving down to P&L, we continue to see an increase in interest income due to higher yields on our cash and investments.

Speaker 6: R&D expense in Q3 2023 was $191 million. And SG&A expense was $224 million, which combined together total $415 million, and grew 11% versus last year.

R. D expanse in Q3, 2023, with $191 million and SG&A experience with $224 million, which combined together total of $415 million and grew 11% versus last year.

Speaker 6: As compared to 15% revenue growth in the quarter, this aligns with our goal of growing operating expenses slower than revenue to deliver operating margin expansions for the full year. The increase in R&D in SGNA was primarily driven by the development of our early stage pipeline and global commercial launch activities for Vaxvogo and Rock Tables.

As compared to 15% revenue growth in the corridor. This aligns with our goal of growing operating expenses slower than revenue to deliver operating margin expansion for the full year the.

The increase in R&D and SG&A was primarily driven by the development of our earliest date pipeline and global commercial launch activities for back so I'll go and <unk>.

Speaker 6: As we look ahead to the fourth quarter, we anticipate that total S-GNA and RD expense will be hired in Q3 at a dollar basis due to the same driver.

As we look ahead to the fourth quarter, we anticipate that total SG&A and Hardy expense will be hired in Q3 at a dollar basis due to the same drivers moving.

Speaker 6: Moving down the P&L, we continue to see an increase in interest in come to the higher yields on our cash and investment.

Moving down the piano, we continue to see an increase in interest income higher yields and our cash and investments.

Jeff Ajer: We have expanded from operating only in the United States when I started with a company with now at that time 300 employees to sitting already seeing in almost 80 countries around the world and more than 3,000 employees.

Speaker 6: on tax expenses for the quarter. I will note that we recognize tax expenses at a lower effective rate during Q3 due to some discrete tax benefits during the quarter. This lower tax expense in Q3 will likely modestly benefit the full year tax as well. But we continue to project that our global annual effective tax rate will be in the 20 to 23% range over the next few years.

Brian Mueller: On tax expense for the quarter, I will note that we recognized tax expense at a lower effective rate during Q3 due to some discrete tax benefits during the quarter. This lower tax expense in Q3 will likely modestly benefit the full-year tax as well, but we continue to project that our global annual effective tax rate will be in the 20% to 23% range over the next few years. On the bottom line, we delivered on our commitment to profitability with $40 million of GAAP net income in Q3 2023, and $89.5 million of non-GAAP income. This level of profitability for Q3 and year-to-date gives us confidence to achieve our stated objectives of sustained and growing profitability on a full year basis going forward.

And tax expense for the quarter I will note that we recognized tax expense at a lower effective rate during two three due to some discrete tax benefits during the quarter. This lower tax expense in Q3 will likely modestly benefit the full year tax as well, but we continue to project that our global annual effective tax rate will be in.

Jeff Ajer: As the company is exposed for its next phase of growth with a recent introduction of Voxogo and Rogue Davien, this is the ideal time to hand over the reins to the next leader. In the search, the board focused on finding a successor with experience driving commercial growth and operational accidents, while at the same time bringing a deep understanding of the science and a strong background in product development. We are confident that Alexander perfectly meets those criteria as CEO of Genentex and Alexander let the commercialization of 10 new products in teophutics areas including cancer and hemophilia.

The 20 to 23 per cent range over the next few years.

Speaker 6: On the bottom line, we delivered on our commitment to profitability with $40 million of GapNet income in Q3, 23, and $89.5 million.

The bottom line, we delivered on our commitment to profitability with $40 million of GAAP net income in Q3 23.

And $89.5 million with non-GAAP income.

Speaker 6: This level of profitability for Q3 and here to date gives us confidence to achieve our stated objective.

This level of profitability for Q3 and year to date gives us confidence to achieve our stated objective of sustaining.

Speaker 6: The ending growing profitability on a full year base is going forward.

Standing growing profitability at a full year basis going forward your.

Brian Mueller: Year-to-date, 2023 non-GAAP diluted earnings per share has increased 33% as compared to Q3 year-to-date last year, and today we have reaffirmed our midpoint guidance for non-GAAP diluted earnings per share for the full year. In closing, we are on track to meet our top-line and bottom-line objectives for 2023 and beyond, with strong double-digit growth in both revenue and profitability. The continued success of VOXZOGO, the momentum building for ROCTAVIAN, and ongoing margin expansion efforts, gives BioMarin a strong foundation to achieve our financial goals of being one of the industry leaders in revenue growth and margin expansion as we close 2023 and head into 2024. Thank you for your attention, and we will now open up the call for questions.

Speaker 6: Year-to-date 2023 non-gap diluted earnings per share has increased 33% as compared to Q3 year-to-date last year. And today we have reaffirmed our mid-point guidance for non-gap diluted earnings per share for the full year.

Year to date 2023, non-GAAP diluted earnings per share has increased 33% as compared to Q3 year to date last year and today, we have reaffirmed our mid point guidance for non-GAAP diluted earnings per share for the full year.

Jeff Ajer: Having a CEO of this industry in innovative and impactful company over the past 18 years has been the highlight of my career. We have built an incredible organization with great potential to be further unlocked by Alexander and the team. I will miss working with our talented and inspiring employees day in and day out. I am confident that the company is well-positioned for future success.

Speaker 6: In closing, we are on track to meet our top line and bottom line objectives for 2023 and beyond with strong double-digit growth in both revenue and profitability.

In closing we are on track to meet our top line and bottom line of Texas for 2023, and beyond with strong double digit growth in both revenue and profitability.

Speaker 6: It continued success of BoxoGo, the momentum building for Octavian, an ongoing margin expansion effort, gives Biomerin a strong foundation to achieve our financial goal of being one of the industry leaders in revenue growth and margin expansion as we close 2023 and head into 2024.

The continued success of boxes go the momentum building for Octavian, an ongoing margin expansion efforts kids by them or in a strong foundation to achieve our financial goal of being one of the industry leaders in revenue growth and margin expansion as we closed 2023 and had into 2024. Thank.

Jeff Ajer: So to do this on the call today, thank you for your engagement and support over the years. I also want to emphasize that I will still be involved with the company and I will still be very involved with biotech in general as you will find out in the future.

Speaker 6: Thank you for your attention and we will now open up the call for questions.

Thank you for your attention and we will now open up the call for questions.

Operator: ... Thank you. If you have a question, please press star one on your telephone keypad. If you wish to remove yourself from the queue, simply press star one again. One moment for your first question. Your first question comes from the line of Sabine Richter of Goldman Sachs. Your line is open.

Thank you if you have a question. Please press star one on your telephone keypad, if you wish to remove yourself from the queue simply press Star One again, one moment for your first question.

Speaker 1: Thank you. If you have a question, please press star one on your telephone keypad. If you wish to remove yourself from the queue, simply press star one again. One moment for your first question.

Jeff Ajer: So now let's turn back to the quality results. We are very pleased with the results in the third quarter. We are recording 15 percent total revenue growth compared to the third quarter last year and continued strong growth in earnings per share faster than revenue. These results were driven primarily by Voxogo which remains on track to become our first blockbuster product. We have so strong demand and uptake in the quarter and we expect this to continue.

Speaker 1: Your first question comes from line of Selvene Richter of Goldman Sachs. Your line is open.

Your first question comes from the line of service Rector of Goldman Sachs. Your line is open.

Speaker 7: Good afternoon, thanks for taking my question. And J.J., it's been a pleasure working with you. Good luck on the next venture here. With regard to my question.

Sabine Richter: Good afternoon. Thanks for taking my question. And JJ, it's been a pleasure working with you. Good luck on the next venture here. With regard to my question, you talked about the, with ROCTAVIAN, the HTC preparedness that's required, and then you also provided some context of 2 to 5 months to prepare patients for treatment. Can you just break that down for us, like, the time period needed for HTC, then kind of the payer dynamics that will play out, the diagnostic aspect as well, and how demand is stacking up as you get a sense from physicians and these centers right now? Thank you.

Salveen Richter: Good afternoon. Thanks for taking my question. And JJ, it's been a pleasure working with you. Good luck on the next venture here. With regard to my question, you talked about the, with ROCTAVIAN, the HTC preparedness that's required, and then you also provided some context of 2 to 5 months to prepare patients for treatment. Can you just break that down for us, like, the time period needed for HTC, then kind of the payer dynamics that will play out, the diagnostic aspect as well, and how demand is stacking up as you get a sense from physicians and these centers right now? Thank you.

Good afternoon. Thanks for taking my question and J J, it's been a pleasure working with you. Good luck on your next venture here Uhm with regard to my question you.

Speaker 7: You talked about the, with Rocktavian, the HTC preparedness that's required, and then you also provided some context of two to five months to prepare patients for treatment. Can you just break that down for us? Like,

And you talked about the with my cable the H T. C. Preparedness, that's required and then you're also provided some context of three to five months to prepare patients for treatment can you just break that down for us like the the time period needed for H T. C. D. Then kind of the pay your dynamics.

Jeff Ajer: In the third quarter more than 300 additional children become treatment with Voxogo since the end of Q2 and additional strong growth is expected in Q4. As the results today we increase full year 2022 Voxogo riders to between 435, 135 million and 455. With recent U.S, and European approvals, Voxogos age expansions, all ages in the U.S, and for children ages, four months and older in Europe. Over 1,000 additional children now have the opportunity to benefit from a longer Voxogos treatment window.

Speaker 7: that the time period needed for HTC, then kind of the payer dynamics that will play out the diagnostic aspect as well. And how demand is stacking up as you get a sense from physicians and the centers right now, thank you.

That will play out the diagnostic aspect as well and and how demand stacking up is you get a sense from from physicians and the centers right now thank you.

Speaker 4: It's all being all take a shot at that one. There's a lot of moving parts in this launch. There is the specific HTC dynamic, which is not unique to rock caveans. So you may be getting some insights and do how that's playing out in a parallel launch in the United States.

Jeff Ajer: Hi, Sabine. I'll take a shot at that one. There's a lot of moving parts in this launch. There is the specific HTC dynamic, which is not unique to ROCTAVIAN, so you may be getting some insights into how that's playing out, you know, in a parallel launch in the United States. Yeah, the HTCs need to be prepared both for the administration of a one-time treatment with ROCTAVIAN, which includes handling a frozen product of very high value. It includes other dynamics like 340B discounting and pricing, relative in some cases to larger healthcare systems that may be a parent organization and have some capabilities that the HTC does or does not have. So there's kind of those dynamics inside of the HTC.

I saw being I'll take a shot at that one there's a lot of moving parts and and this launch there is the specific H D. C dynamic uhm, what which is not unique to <unk>. So you may you may be getting some insight.

Jeff Ajer: We are also pleased to share that based on increased sales finish manufacturing commitments. Voxogos supply is planned to increase from 23 levels. The first and second quarter and 2024 and is expected to be fully unconstrained by mid-year 2024.

And do you have that spelling out you know in a parallel launch in the United States.

Speaker 4: Yeah, the HTC's need to be prepared both for the administration of a one-time treatment with Rotkevian, which includes

Yeah, the H D c's need to be prepared both for the administration of a one time treatment with Roth avian which includes.

Jeff Ajer: Building now to Rotivian, where we have made tremendous progress building and connecting the network that will facilitate patient treatment. We'll be laying the groundwork for patient access to Rotivian. It has taken time but has been an essential step to ensuring access and reimbursement. Well, over a hundred people, a big deal. We have been tested for Rotivian 85 eligibility globally, which is the first step that a patient needs to do to determine if he can be treated for Rotivian.

Speaker 4: Handling a frozen product of very high value, it includes other dynamics like 340B discounting and pricing relative in some cases to larger healthcare systems that may be a parent organization and have some capabilities that the HTC does or does not have. So there's kind of those dynamics inside of the HTC.

Uhm handling a frozen product a very high value.

Includes other dynamics like 340 P discounting in pricing.

Relative in some cases to larger health care systems that may be a parent organization.

Have some capabilities of H D. C does or does not have so there's kind of those dynamics inside of the H D C.

Speaker 4: There's also the payer dynamics going on, which you would expect anyway in the United States. But in this case, the payer dynamics are complicated by the fact that gene therapies in a high value, one time treatment are new and different. We've known and expected the payers.

Jeff Ajer: There's also the payer dynamics going on, which you would expect anyway in the United States. But in this case, the payer dynamics are complicated by the fact that gene therapies, you know, in a high, high-value, one-time treatment, are new and different. We've known and expected the payers will take some time to get their hands around that issue for years, and we've been preparing for that eventuality. There's also patient awareness and interest in a highly competitive and very dynamic, changing standard of care environment. There's physicians that have a number of options available to them. There's all those dynamics going on in parallel, and we're navigating all of those issues in parallel. Some are taking some time. I mentioned the payer dynamics specifically.

There's also the pair dynamics going on which you would expect any way in the United States, but in this case. The <unk> dynamics are are complicated by the fact that gene therapies at.

Jeff Ajer: The zero negativity rates on the samples that were tested today is approximately 70%. 70% of a patient tested are eligible for Rotivian. They still need to believe it's gone, but most of the eligibility comes from the 85 positivity. And we expect the testing momentum to continue as awareness and access increases. Well, it has taken more time than an easy payment to give global healthcare system wire to facilitate Rotivian treatment. We remain encouraged by the high level of interest and demand from patients, physicians, patient advocacy groups, hemophilia treatment centers, and payers.

At a high high value one time treatment, our our new and different we've known and expected the bears.

Speaker 4: We'll take some time to get their hands around that issue for years and we've been preparing for that eventuality.

Will will take some time to get their their hands around that issue for for years and we've been preparing for that eventuality.

Speaker 4: There's also patient awareness and interest in a highly competitive and very dynamic changing standard of care environment. There's positions that have a number of options available to them. There's all those dynamics going on in parallel and we're navigating all of those issues in parallel some are taking some time. I mentioned the pair dynamics.

There's also patient awareness and interest in a highly competitive and very dynamic changing standard of care environment.

There is physician that I have a number of options available to them Uhm, there's all those dynamics going on in parallel and <unk> were navigating all of those issues and in parallel some are taking some time.

Jeff Ajer: In Europe, we have we have strong progress in the quarter and expect to have final German and Italian process for Rotivian published by year and and in the US, significant momentum connecting the network to state treatment is occurring.

Mentioned the pair dynamics specifically.

Jeff Ajer: What I believe is that the two-thirds of US population with issued coverage policies four months after approval, I think is, is really encouraging and very, very fast. The uptake of the warranties, which is a brand-new thing for payers, at the level that I described, is very fast and very encouraging. And we're seeing the patient funnel now starting to take shape in the United States. So I've been describing the patient funnel over the last year in Germany, and, you know, the US patient population is substantially larger than that of Germany. But early signals in the US look like the taking shape of that patient funnel is going to have some similarities to what we've experienced in the United States.

Speaker 4: What I believe is that the 2-3rd, the U.S. population with issued coverage policies four months after approval, I think is really encouraging and very, very fast.

What I believe is that the two thirds the U S population with issued coverage policies for months. After approval. I think is is really encouraging and very very fast the uptake of the warranties, which is a brand new thing four pairs.

Jeff Ajer: Yes, we provide more detail on lost progress and our updated expectations for Rotivian. According to Bowie's record here today, revenues of really nearly $1.8 billion in the first nine months of the year represents 14% year over year growth, even with lower than expected contributions from Rotivian. These results underscore the strength of our business and the achievement of double digit revenue growth and our profitability objectives as set out as the start of the year.

Speaker 4: The uptake of the warranties, which is a brand new thing for pairs At the level that I described is very fast and very encouraging

But the level that I described as very fast and very encouraging them and we're seeing the patient bottle now starting to shape takes shape in the United States. So I've been describing the patient final over the last year in Germany, and you know the U S patient population is substantially large.

Speaker 4: And we're seeing the patient funnel now starting to shape, take shape in the United States. So I've been describing the patient funnel over the last year in Germany and the US patient population is substantially larger than that of Germany, but early signals on in the US

Jeff Ajer: The business is supposed for strong growth in 24 and beyond. We've also been continuing to excited expectations. Sorry, and we're giving access to re-enversation in place for a meaningful update next year. We believe our revenues are set up to approach $3 billion in 2024.

Than that of Germany, but early signals on in the U S.

Speaker 4: look like the taking shape of that patient funnel is going to have some similarities to what we've experienced.

Look like the the taking shape of that patient final is going to have some similarities to what we've experienced in the United States all of which gives me a great deal of confidence that uhm as we.

Jeff Ajer: In summary, we have built an industry in company. The strategy to create a sustainably profitable and growing business provides patients with innovative and essential medicine is coming to fruition. This future looks very bright for Baham Ren and I want to thank my colleagues for the tremendous collaboration and for what we have built together.

Speaker 4: in the United States, all of which gives me a great deal of confidence that as we finish up this year hopefully treat some patients in Q4 that were really set up with pair of approvals in Italy and Germany and the dynamics in the United States falling into place, all of which point to greater uptake in 2024. Thanks for the question.

Jeff Ajer: All of which gives me a great deal of confidence that, as we, you know, finish up this year, hopefully treat some patients in Q4, that we're really set up with payer approvals in Italy, Germany, and the dynamics in the United States falling into place, all of which point to greater uptake in 2024. Thanks for the question.

Finish up this year hopefully treat some patients in queue for that were really set up with pair approvals in Italy, and Germany and the dynamics in the United States falling into place all of which 0.2 greater uptake in 2024. Thanks for the question.

Jeff Ajer: Thank you for continuing support.

Jeff Ajer: I will not turn the call over to Geoff Hager to discuss the commercial business of this. Thank you, JJ, in so many ways. I'm very pleased with our commercial performance in the third quarter, resulting in $581,000 in total revenues, and representing 15% growth year-over-year, including Cuban, and 20% growth, excluding Cuban. Turning to Vox Sago, as JJ mentioned, today we updated full-year guidance to between $435,000,000 and $455 million, representing approximately 165% year-over-year growth at the midpoint.

Operator: Your next question comes from the line of Geoff Meacham of Bank of America. Your line is open.

Your next question comes from the line of just <unk>.

Speaker 1: Your next question comes from the line of Jess uh, meet-men of Bank of America. Helene is open.

Bank of America. Your line is open.

Speaker 5: Great. Hey guys, thanks for the question. JJ also wanted to offer up a congrats on the retirement. So not surprisingly, you have a few on rock TV as well. So the first one is, I know you're not giving more detail. 24 guidance than the three billion in revenue. But at a high level, how would you characterize the rock TV and contribution?

Geoff Meacham: Great. Hey, guys, thanks for the question. JJ, also wanted to offer up congrats on the retirement. So not surprisingly, I have a few on ROCTAVIAN as well. So, you know, the first one is, I know you're not giving more detailed 2024 guidance than the, you know, $3 billion in revenue, but at a high level, you know, how would you characterize the, the ROCTAVIAN contribution? And the second question is, I guess when you use Germany as a, as a proxy, you know, you have 60 patients, you know, that are pretty late in the onboarding process. It's been on the market, you know, officially for over a year. Is there, is there what you characterize as a second wave or, or is the 60 mostly it?

Great Hey, guys. Thanks for the question J J also wanted to offer up a congrats on the retirement.

So not surprisingly I have a few on rock saving as well so.

The first one is I know, you're not giving more detailed 24 guidance and the.

3 billion in revenue, but at a high level you know how would you characterize the the rock T V and contribution and the second question is I guess, when you used German Germany as a as a proxy you know you have 60 patients that are pretty late in the onboarding process. It's been on the market you know officially for over a year.

Speaker 5: And the second question is, I guess when you use German, Germany as a proxy, you know, you have 60 patients, you know, that are pretty late in the onboarding process. It's been on the market, you know, officially for over a year or a year.

Jeff Ajer: Appreciating that the run rate for the fourth quarter in all year 2023 may seem conservative based on Vox Sago revenues of $123 million in the third quarter, we note the impact from supply constraints discussed last quarter, continued limit growth through the end of the year. While we continue to closely manage new patient growth and limit inventory stocking, we expect to fulfill smaller orders from various regions in Q4 while at the same time adding hundreds of new patients in the fourth quarter.

<unk> is there would you characterize as a second wave or or is the 60, mostly it wasn't I wasn't sure kind of you know if there's a bit of a ripple effect once you get formal reimbursement. Thank you.

Speaker 5: Is there, you characterize it as a second wave or is this 60 mostly it? I wasn't sure if there's a bit of a ripple effect once you get formal reimbursement. Thank you.

Geoff Meacham: I wasn't sure kind of, you know, if there's a bit of a ripple effect once you get formal reimbursement. Thank you.

Jeff Ajer: You want me to start? Yeah. Hey, thanks, Jeff. Brian here. I'll start with your first question. On the $3 billion, you know, as you noted, haven't given the specifics behind that. We'll plan on doing that early next year like we typically would. But importantly, there's both continued growth in VOXZOGO over 2023 and a meaningful contribution from ROCTAVIAN. So we'll ask you to stay tuned for the details, but you understand, you know, what has been the typical growth trajectory for the mature enzyme products, you know, still growing healthily here, plus substantial growth in VOXZOGO, plus meaningful ROCTAVIAN contribution, gets us to that approximately, or approaching $3 billion.

Brian Mueller: You want me to start? Yeah. Hey, thanks, Jeff. Brian here. I'll start with your first question. On the $3 billion, you know, as you noted, haven't given the specifics behind that. We'll plan on doing that early next year like we typically would. But importantly, there's both continued growth in VOXZOGO over 2023 and a meaningful contribution from ROCTAVIAN. So we'll ask you to stay tuned for the details, but you understand, you know, what has been the typical growth trajectory for the mature enzyme products, you know, still growing healthily here, plus substantial growth in VOXZOGO, plus meaningful ROCTAVIAN contribution, gets us to that approximately, or approaching $3 billion.

Speaker 6: Can be in the start. Yeah, hey, thanks Jeff Brian here. I'll start with your first question. On the three billion, as you noted, haven't given the specifics behind that. We'll plan on doing that early next year like we typically would. But importantly, there's both continued growth in Voxfogo over 23 and a meaningful contribution from Rocktable.

<unk> yeah, he answered that extra Brian here I'll start with your first question on the 3 billion. You know as you noted haven't given the specifics behind that plan on doing that early next year like we typically would.

Jeff Ajer: As a reminder, new patient starts with Vox Sago in the fourth quarter will represent only a fraction of a full-year revenue patient in 2023. Importantly, we expect approximately 2600 patients to be receiving Vox Sago treatment by year end 2023. Vox Sago supply is planned to increase from 2023 levels through the first and second quarters of 2024, and is expected to be fully unconstrained by mid-year next year. These commitments will ensure that we have ample supply to exceed full-year 2024 consensus, currently at $624 million, and support growth beyond 2024.

But importantly, there's both continued growth inbox they'll go over twenty-three and a meaningful contribution from <unk>. So I'll ask you to stay tuned for the details, but you understand what has been the you know typical growth trajectory for the mature enzyme <unk>.

Speaker 6: So let's get a stay tuned for the details, but you understand what has been the typical growth trajectory for the mature enzyme products, still growing healthily here, plus substantial growth in VoxoGo, plus meaningful rock-giving contribution to that approximately, or approaching three billion.

<unk> you know still feel growing healthily here plus substantial growth inbox will go plus meaningful rocked heaving contribution.

To that approximately are approaching 3 billion.

Speaker 4: And as usual, we will give more detailed guidance when we report to four in every week of next year. And I think great question about

Jean-Jacques Bienaimé: As usual, we will give more detailed guidance, you know, when we report Q4 in February of next year.

As usual, we will give more detailed guidance you know when we report to do for you.

Next year.

Jeff Ajer: And I think great question about the 60 AAV5-eligible patients that have yet to be treated in Germany. Actually, the pace of CDX testing in Germany slowed down over the summer. That's probably a reflection both of the general slowdown in August, in particular in Germany, and also a reflection that, as I'd noted previously, our you know our earlier plan to treat patients in the free pricing period while we were negotiating with GKV, wasn't paying out the way we had hoped it would. So we know the HTCs were slowing down in Germany, waiting for the GKV process to be finished.

And I said, great question about the 60, a V five eligible patients that have yet to be treated in Germany.

Jeff Ajer: At the end of the third quarter, more than 2300 children with ACounder Plasia in 38 markets were being treated with Vox Sago. Uptake to date represents 12% penetration of the indicated patients in Biomurin's commercial footprint, highlighting the significant growth potential remaining. With the opportunity to now treat up to an additional 1000 children following the recent age expansion approvals in the US and Europe, we look forward to the possibility of the youngest children in these regions benefiting from Vox Sago therapy.

Speaker 4: AAV-5 eligible patients that have yet to be treated in Germany.

Speaker 4: But actually the pace of CDX testing in Germany is flowed down over the summer. That's probably a reflection both of the general flow down in August in particular in Germany and also a reflection that is I've noted previously our earlier plan to

The actually the pace of C. D X testing in Germany slowed down over the summer that's probably a reflection both of the general slowdown in in August in particular in Germany, and also a reflection that as I've noted previously R. R. R earlier plan.

To treat patients in the free pricing period, while we were negotiating with T. K V wasn't.

Speaker 4: treat patients in the free pricing period while we are negotiating with TKV.

Jeff Ajer: Turning now to Octavian, we are pleased to share that the second commercial patient was treated today in Germany. As JJ highlighted, we continue to make progress connecting the network that will facilitate treatment. 2023 has turned out to be the year of building that network to ensure and support meaningful patient uptake in 2024. Starting with Europe, we have tentative agreement with the German National Association of Statutory Health Insurance Funds, or GKB, on the final German prize for Octavian, which we expect to be formalized and published by the end of the year.

Speaker 4: paying out the way we had hoped it would. So we know the HTC's were slowing down in Germany waiting for the GKB process to be finished. My perception is a pickup.

Paying out the way we had hoped it would so we know the H T. C is we're slowing down in Germany waiting for the T. K V process to be finished my perception is a pick up following the end of the summer and C. D X activity and lining up patients to be treated in <unk>.

Jeff Ajer: My perception is a pickup following the end of the summer in CDX activity and lining up patients to be treated in Germany is both reflective of, one, getting past the summer, and two, an expectation that these HTCs know essentially that we've come to a conclusion on price. They're just waiting for that price to be published so they can move ahead with patient treatment. And while we're on the subject of Germany, which the physicians in Germany and the data on other launches would bear out, Germany moves slow for adoption of new hemophilia options. Having 60 patients in Germany that are AAV5 eligible on a population base-adjusted basis in the United States would equate to about 240 to 250 patients.

Speaker 4: Following the end of the summer in CDX activity.

Speaker 4: and lining up patients to be treated in Germany is both reflective of one getting past the summer and to an expectation that these HTC's know, eventually, that we've come to conclusion on price. They're just waiting for that price to be published so they can move ahead with patient treatment. While we're on the subject of Germany, which the physicians in Germany and the data on other launches would bear out,

<unk>, both reflective of one getting past the summer and to an expectation that V. H D sees no essentially that we've come to a conclusion on price, they're just waiting for that price to be published so they can move ahead with patient treatment and while we're on the subject of of Germany, which.

Jeff Ajer: As we have said previously, we believe this important step, given the unique treatment and reimbursement profile of Octavian, will open up the access bundle to treatment in Germany. We are pleased to share that in Germany, 60 people are known to be eligible based on CDX testing for AAV 5 antibodies. The vast majority of those are in follow-up for next steps. As expected, the seronegativity rate of about 70% is in line with our study results and consistent with expectations.

The physicians in Germany, and and the the data on other launches with bear out Germany move slow for the option of <unk> options, having 60 patients in Germany that R. A V.

Speaker 4: Germany moved slow for the option of new hemophilate.

Speaker 4: Having 60 patients in Germany that are AAV-5 eligible on a population of a base-adjusted basis in the United States would equate to about 240 to 250 patients. So just benchmarking what may be the possibilities in the US.

I've eligible on a population of <unk> face adjusted basis in the United States would equate to about 240 to 250 patients. So just benchmarking <unk>, maybe the possibilities in the U S. Thank you.

Jeff Ajer: Just benchmarking what may be the possibilities in the US. Thank you.

Jeff Ajer: We are also pleased to report that there are now 10 sites in Germany testing for eligibility. With a large full of eligible patients and the final German price expected to be published soon, we are optimistic about patient uptake rates in 2024. Important to note is that the first patient treated resulted in net revenue of approximately $800,000. Gross to net revenue adjustments going forward under the GKV agreement will be different than for the first patient reported.

Speaker 1: Your next question comes from a line of film The Dole of Titi Cowan. Your line is open.

Operator: Your next question comes from the line of Phil Nadeau of TD Cowen. Your line is open.

Your next question comes from the line of <unk> of T. D. Cohen Your line is open.

Phil Nadeau: Good evening. Thanks for taking our questions. JJ, let me add our congratulations on a very successful tenure, and your retirement from BioMarin. Again, a couple of follow-ups on ROCTAVIAN, specifically on the, on patient demand. On the last earnings call, you mentioned that patient consent forms had been trending well, and everyone was happy with the rate that they had started to come in. Any update on how patient consent forms are trending, and is there any visibility on when the first US patients could be treated? That's question one. And then question two: In terms of the institutional challenges, we've had some physicians mention things like training staff, beds need to be set up, equipment needs to be ordered. Are those challenges at the HTCs? And if so, is there anything BioMarin can do to remove those bottlenecks?

Speaker 8: Good evening, thanks for taking our questions. JJ, let me add our congratulations on a very successful tenure and your retirement from Bimer. And again, a couple of follow-ups on rock-tavian, specifically on patient demand. In the last earnings call, you mentioned that patient consent forms had been trending well and Bimer was happy with the rate that they had started to come in. Any update on how patient consent forms are trending.

Good evening, Thanks for taking our questions J J, let me at our congratulations on a very successful tenure any retirement from Biomarin again, a couple a couple of follow ups on rock T V in specific around the patient demand.

I'll start this call you mentioned that patient consent forms had been training well and environment was happy with the rate that the head start to come in any update on how patient consent forms are trending and is there any visibility on when the first U S patience can be treated.

Jeff Ajer: But our overall expectations are to maintain similar net revenue per patient in Germany, noting the impact of weight-based dosing and variable weight of patients. Italy negotiations with the Italian medicine agency are going well and we expect to have a final price published by year end. Italy represents then our second major European market that has agreement on price and reimbursement opening the door to Octavian access likely in early 2024. We continue to work with the authorities in France and expect pricing negotiations to extend into 2024.

Speaker 8: and is there any visibility on when the first US patients could be treated? Let's question one. And then question two, in terms of the institutional challenges, we've had some physicians mention things like training staff.

That's a question one in a question to you in terms of institutional challenges. We've had some physicians mentioned things like training staff birds need to be set up equipment needs to be ordered.

Speaker 8: beds need to be set up, equipment needs to be ordered.

Speaker 8: Are those challenges at the HTC's and if so, is there anything by or can do to remove those bombs?

Are those challenges at the eighties H D C's and if so is there anything <unk> can do too.

To remove those bottlenecks.

Phil Nadeau: Thanks.

Jeff Ajer: Thank you for the question, Phil. So, let me start with the second point first, the institutional challenges. In the prepared remarks, I addressed HTC site readiness and some of the elements of that that I think are one-time events that won't be recurring. The types of things that you just mentioned, I would say generally are captured in that HTC site readiness and some of the institutional things that they're doing to get ready for ROCTAVIAN treatment in particular, and taking a little bit of time, but which should be resolved and facilitate patients to move expeditiously through those type of things going forward.

Thank you for the question Bill So let me start with the second point Forsee institutional challenges and and the prepared remarks, I addressed H D. C site readiness and some of the elements of that that I think are are one time event.

Speaker 4: Thank you for the question, Phil. So let me start with the second point, for see, institutional challenges.

Jeff Ajer: It has taken several months in the U.S, following approval to line stakeholders and to facilitate first patients treated. In general terms, this is a result of that unique profile of one-time reimbursement and treatment with Octavian. Navigating hospital administration and finance adds a level of complexity for him affiliate treatment centers or HTC's leading to the final step of patient treatment with Octavian. We expect that these administrative steps taken at the HTC level while time consuming are one-time events and essential to facilitate patient access going forward. In short, groundwork completed in 2023 is expected to enable simplified access to Octavian. Live more and more AAD-5 eligibility testing and giving us confidence in the prospects for 2024.

Speaker 4: And in the prepared remarks, I addressed HCC site readiness.

Speaker 4: And some of the elements of that that I think are one time events that won't be recurring. The types of things that you just mentioned, I would say generally are captured in that HTC site readiness and some of the institutional things that they're doing to get ready for rock baby and treatment in particular.

<unk> that won't be recurring uhm the types of things that you just mentioned I I would say generally are captured in that H T. C site readiness and some of the institutional things that they're doing to get ready for <unk> treatment in particular.

Speaker 4: and taking a little bit of time, which should be resolved and facilitate patients to move expeditiously through those type of things going forward. In terms of patient demand in the United States,

And taking a little bit of time, but which should be resolved and facilitate patients to move expeditiously through those type of things going forward in terms of patient demand in the United States and we're <unk>, we're not disclosing patient level of detail in the United States, but we're <unk>.

Jeff Ajer: In terms of patient demand in the United States, we're not disclosing patient-level detail in the United States, but we're continuing to see, as I noted, that patient funnel build-up in the US, and I'm qualitatively encouraged by what we're seeing so far. In the US, we have this hub operation, and in prior launches, you know, all of the patients move through our hub essentially first before they move through the next steps towards treatment. Given the nature of the HTCs and their capabilities and the fact that they operate in this multi-sponsor environment, we've been seeing patients come into our hub maybe later in the process and not the very beginning of the process. So this is a little bit of a different dynamic for us. We're getting our hands around that one.

Speaker 4: We're not disclosing patient level detail in the United States, but we're continuing to see as I noted that patient tunnel builds up in the U.S. And I'm qualitatively encouraged by what we're seeing so far.

<unk> C. As I noted that patient, but I'll build up in the U S and I'm I'm qualitatively encouraged by what we're seeing so far.

Jeff Ajer: However, with less than two months remaining in the air and the holiday season beginning soon, we are lowering full year Octavian 2023 guidance to less than $10 million. Moving now to specific steps completed this quarter, starting with the issuance of coverage policies across payers, both private and public, we are pleased to share that nearly two thirds of all covered lives in the United States have been issued coverage policies today. Representing approximately 205 million people.

Speaker 4: In the US, we have this hub operation. And in prior launches, all of the patients move through or hub essentially first before they move through the next steps sort of treatment.

And the U S. We have this hub operation and in prior launches all of the patients move through our hub essentially first before they move through the next steps or its treatment given the nature of the H D C as in their capabilities and the fact that they operate in this <unk>.

Speaker 4: Given the nature of the HTC's and their capabilities.

Speaker 4: and the fact that they operate in this multi-sponter environment.

Ah sponsor environment, we've been seeing patients come into our hub, maybe later in the process and not the very beginning of the process. So this is a little bit of a different dynamic for us. We're we're getting our hands around that one but yes encouraged by the patient demand that we're seeing in the United States.

Speaker 4: We've been seeing patients come into our hub maybe later in the process and not the very beginning of the process. So this is a little bit of a different dynamic for us. We're getting our hands around that one. But yes, encouraged by the patient demand that we're seeing in the United States and the patient funnel building up. In addition to the funnel breathing of the Kapi, also having.

Jeff Ajer: Importantly, the vast majority are in line with label or clinical trial criteria and are thus favorable and meeting expectations, patients. Additionally, we are encouraged by the rapid take of the outcomes-based warranty with signed warranties in place representing more than 95 million U.S, lives. We have also received notice that the J-code for Raktavian has been assigned effective January 1, 2024. The permanent J-code is expected to accelerate Pair's prior authorization review process, ultimately speeding time to patient treatment.

Jeff Ajer: But yes, encouraged by the patient demand that we're seeing in the United States and the patient funnel building up.

The patient Bunnell building up in addition to the federal building up though I mean.

Jean-Jacques Bienaimé: Yeah, in addition to the federal building up, though, I mean, it is possible, I, I think that was your question, could we treat some US patients by year-end? I would say, yes, it is possible. We have 3 US patients who have now cleared all ROCTAVIAN screens for treatment, like the AAV5, liver scan, liver health, and all that. So the timing of their treatment isn't guaranteed in 2023, but they have, they have completed all eligibility testing for treatment, so it, it will very likely occur in the not-too-distant future.

Speaker 3: It is possible that I think that was your question. Can we treat some investigations by Iran? I would say yes, it is possible. We have...

It is possible that I think there was your question, we treat somebody reservations by you and I would say, yes. It is possible we have three.

Speaker 3: Three US patients who have now cleared all of Kavian screens for treatment, like the AV5, Leavers-Kaan, Leavers-Hell, and all that. So the timing of the treatment isn't guaranteed in 23, but they have complete eligibility testing for treatment. So it will very likely occur in and not to be seen.

<unk>, we have now appear all <unk> screens for treatment.

85, <unk> <unk>.

[noise] house and all that so the timing of their treatment isn't guaranteed in 23, but they have they have some <unk> for treatment. So it it.

Jeff Ajer: Akin together, these developments are a strong indication of the value of the U.S, health system places in a cost-effective one-time gene therapy. We stated in our approval in Q2 earnings call, we expect it could take patients two to five months to complete the steps necessary before treatment with Raktavian. The commercial team has been hard at work, implementing a number of tactics to prepare physicians, HTC's and patients for the opportunity to benefit from Raktavian.

He will very likely occur eaten or not too distant future.

Operator: Your next question comes from the line of Robyn Karnauskas of Truist. Your line is open.

Your next question comes from the line of Robyn Curnow scarce of Trust. Your line is open.

Speaker 1: Your next question comes from the line of Robin Karnowskas of Truth. Your line is open.

Robyn Karnauskas: Great. I have three quick questions. Number 1, JJ, you have mentored so many people on the street, so appreciative. Is Hank retiring, too? Because then we will have a panic attack. So, would you like an announcement on that call? Is Traci getting a promotion? That's our first question. The second question would be, ROCTAVIAN. There was such a focus, and you misguided this year on the outcome of what you could achieve. Do you think in 2024 you can do better? And then I think on VOXZOGO, I mean, that came away from our analyst meeting as, like, the big takeaway. So how do you think about letting analysts understand the opportunity there and the timelines for that? And I'll go back to Traci's promotion. Is Hank leaving? I'll leave it at that.

Speaker 9: Great, I have three quick questions. Number one, JJ.

Great I have three quick question number one J J.

Speaker 9: You have mentored so many people on the street. So appreciative. Is Hingretary too, because we will have the other tax.

You have been through so many people on the street so appreciative.

Hey, great hurry to cause that we will have to ask like would you like it out the announcement on that call is Tracy <unk>. That's our first question. The second question would be <unk>. There was such a focus and you misguided this year on the outcome of what you could.

Speaker 9: Would you like an announcement on that call? Is Tracy getting a promotion? That's our first question.

Jeff Ajer: With respect to site readiness, HTC's are being prepared and educated on how to administer Raktavian. We have targeted the largest and most capable HTC's for site readiness by year end, a number of which have readiness plans before the end of this year. Increasing HTTP and patient awareness in the U.S, was also an important focus in the quarter. A number of activities were conducted, including national HTTP and patient webinars and the third annual global gene therapy scientific summit sponsored by Biomorin.

Speaker 9: Second question would be, Rock Tatian, there was such a focus and you misguided this year on the outcome of what you could achieve. Do you think in 2024 you can do better? And then I think on VoxoGo, I mean that came away from our analyst meaning it's like the big takeaway.

A cheese do you think in 2024, you can do better and then I think <unk> I mean that Kimberly from our analysts, meaning like the big take away.

Speaker 3: So how do you think about letting analysts understand the opportunity there in the time list for that? And I'll go back to Tracy's promotion is Hank leaving. I'll leave it at that. Thank you, Robin. I mean, I think there was a previous question from I think from Salveen and we had a detail answer from, I know it was from Jeff.

So how do you think about <unk>.

Leading analysts understand the opportunity there at the time lines for that.

And I'll go back to Ya Tracy's promotion is Hank leaving I'll leave it at that.

Jeff Ajer: We are especially encouraged by the number of local patient advocacy grips which have been willing to host branded patient awareness programs for Raktavian. Medical eligibility, including CDX testing, is underway, and as a result, the U.S, patient funnel is beginning to take shape, giving us confidence in the prospects for treating patients going forward.

[noise]. Thank you and I mean, I think they were there was a previous question from from Salve and then you'll get a detailed answer from from.

Jean-Jacques Bienaimé: Thank you, Robyn. I mean, I think there was a previous question from, I think from Sabine, and, you know, we had a detailed answer from, I know it was from Jeff. Indeed, you know, it taken longer than we anticipated to get the to connect the patient with the treatment centers, to get the whole system wired out. So again, big picture, you know, when you have such a big-ticket item like ROCTAVIAN, $3 million WAC, it attracts a lot of people, you know, so essentially, it's a big pie that attracts a lot of players that you know don't get involved in the decision making because they all kind of want a piece of the action. Just, Jeff mentioned the 340B, you know, twenty...

<unk> from from jazz.

Speaker 3: Indeed, you know, it take it longer than we anticipate it to get the Connected patient with the treatment centers because the whole system wired. I was so again big picture You know when you have such a big ticket item like like Rockabian $3 million dollars wax Tracks a lot of people, you know, so essentially it's a big pie Is that it tracks a lot of players that you know you don't get involved in the decision making?

Indeed take.

Taken longer than we anticipated to get the <unk>.

Connected patients with a treatment centers because the whole system wide I was <unk>.

Big Picture you know when you have such a big ticket item like rotarian.

Jeff Ajer: In conclusion, we are on track to achieve 2023 full-year total revenue guidance driven by the strength of our inside-product revenues and continued high demand for BotsoGO globally. Looking ahead, we've leave the network built to facilitate treatment with Raktavian, combined with our high expectations for BotsoGO and strong enzyme business will enable Biomorin to approach close to $3 billion in total revenues in 2024.

$3 million <unk> <unk>.

<unk> a lot of people you know so essentially it's a big pie that attract a lot of players that you know you don't get involved in the decision making.

Speaker 3: because they all come out of one piece of the action. Just just mentioned this 340 V, you know.

Because they all got a lot of piece of the action just just.

<unk> mentioned this before T V you know.

Speaker 3: 2340 B is 23, I mean, all basically all human three patients in the US, such a terrible way lies in 340 B patients. Will you talk to?

Jean-Jacques Bienaimé: 340B is – I mean, all, basically all hemophilia patients in the US, statutory-wise, are 340B patients. So you're talking 20%, you know, 340B discount of a $3 million WAC drug. You're talking about $600,000. As you can understand, many, many people get interested in that pie and want a slice of it, which just slows things down, but eventually, long term, it's actually good news, and we'll be taken care of. So the fact that it's – the takeoff has been delayed, to me and to us at BioMarin, has no impact on the, on the future potential of this drug. It's taking longer than planned.

<unk> 20th <unk>, all basically all he must be patient and use statutory wait like three.

Hank Fuchs: Thank you for your attention and I will now turn the call over to Hank, provide an R&D update. Hank? Thanks, Jeff. Thank you, JJ. The privilege is to ours. And thank you all for joining us today.

<unk> <unk> 20 per cent.

Speaker 3: You know, 340, we discounted with $3 million, WackDriver, talking about $600,000. And you can understand many, many people get interested in that pie. You know, on a slice of it, which just slows things down, but eventually, don't try to be exactly good user, we'll be taken care of. So the fact that it's, the takeoff has been delayed.

340, we just got on with premium those wag drug you're talking about $600000.

You can understand many many people get interested in that.

Hank Fuchs: We provided a thorough update on our earlier state pipeline at R&D day in September, so if you missed it, please find the full presentation on Biomorin's IR website. Starting with BotsoGO in the quarter and shortly beyond, we received some important updates from global health authorities resulting in expanded access to children, younger children in both the U.S, and where it is now approved in all age groups and in Europe for children four months of age and older.

No and a slice of it we just know things done, but eventually longterm, it's actually good use I will be taken care of.

So the.

The fact that is to take off his be delayed.

<unk> <unk> <unk>.

Speaker 3: still impacts on the future potential of these drugs. It's taking longer than planned. It took longer elsewhere, the first time we got a product of proving you not before the US.

No impact on the on the future Joshua this drug is taking longer than plan.

Jean-Jacques Bienaimé: It took longer also. It was the first time we got a product approved in Europe before the US, and ROCTAVIAN got approved in Germany, and based on our previous experiences, you know, we thought we could be able to treat some patients during the free pricing period in Germany, which is slowly disappearing. Didn't happen then. We are basically, you know, very, very close to a final deal with the German authorities, so which generally takes about 12 to 13 months. So in this respect, we're not that far behind versus the European approval. So again, we believe ROCTAVIAN has great potential. We believe ROCTAVIAN will be a significant contributor in 2024, and, and, and beyond.

It took longer I'm, sorry, what was the first time, you've got a product proving you out before the U S.

Speaker 3: and we've taken care of improving Germany and based on our previous experiences, we thought we could be able to treat some patients during the free pricing period Germany, which is slowly disappearing, didn't happen. Then we are basically very, very close to the final deal with the German authority. So we generally take about 12 to 13 months. So in this respect, we're not that far behind versus the European approval.

And what's even got to put in Germany and based on our previous experiences no. We thought we could be able to treat some patients during the the free price interior in Germany, which is slowly disappearing didn't happen. Then we are basically very very close to a final deal with a gentleman authority, so which generally takes about 12 to 13 miles. So in this respect we're not that far behind.

Hank Fuchs: We're gratified that the youngest children now have the possibility of greater potential long-term benefits from treatment of BotsoGO. And I want to thank not just the usual clinical and regulatory teams at these finish line efforts, but also the extended Biomorin team of safety professionals, biometrics professionals, operations, pharmacology, really the whole entire fully integrated team, platy strong role in enabling this advancement and access of patients.

<unk> the the the European approval. So again, we believe productive is great potential we believe <unk> would be a significant contributor.

Speaker 3: Again, we believe that this is a great potential, we believe that Rock Taven would be a significant contributor.

Hank Fuchs: So on behalf of the families and children with acondriplasia, thank you all. As mentioned last quarter, we have solidified our plans to begin the pivotal program using BotsoGO for the treatment of hypochondriplasia with the natural history arm of the study expected to begin this quarter. The pathogenesis in epidemiology are similar between acondriplasia and the hypochondriplasia. Well, hypochondriplasia presents with a milder phenotype in some patients in terms of short stature, disproportionately in clinical manifestations.

Speaker 3: And beyond obviously not as much as Voxloga, which is turning into a very, very large drag.

24, and and and beyond obviously not as much as of October which is turning into a very very large dry.

Jean-Jacques Bienaimé: Obviously, not as much as VOXZOGO, which is turning into a very, very large drug, based on, you know, our Q3 results, particularly Q4, and the fact that, you know, thanks to the efforts of our drug supply team, you know, all the constraints in terms of packaging that we had and field finishes that we had this year are starting to go away, and we'll, and we'll be much more substantial. We'll have much more substantial capacity in 2023 and 2024. In Q1 and Q2, and then be free of all capacity constraints by the middle of the year. So hope that answers your question, and I'll get back to you regarding Traci.

Speaker 3: based on, you know, or two, three results, participatory Q4 and the fact that, you know, thanks to the efforts of our drug supply team, you know, all the constraints in terms of...

Based on you know R. Two to three resolve such as <unk> and the fact that you're thanks to the efforts of our drug supply team you know the <unk>.

Speaker 3: packaging that we had and feel finished with that we had this year are starting to go away and will be much more substantial we have much more substantial capacity in 23 and in Q1 and Q2 and then if we have all capacity constraints by the middle of the year. Hope that answers your question and I'll get back to you regarding Tracy.

Packaging that we had in seal cities that we had this year are starting to go away and we will I will be much more substantial.

Hank Fuchs: We plan to target the population with the most severe form of the disease, and therefore the highest bond met needs with high deficits beyond three standard deviations below that it means. With a line-in from FDA on the single registration enabling 52-week randomized double-blind placebo controlled study with a primary outcome of annualized growth velocity, we look forward to initiating the treatment arm of the study in 2024, following the natural history run in portion.

More substantial capacity in 23, <unk> and Q2 and then.

Free of all capacity constrained by the need over here.

Okay, Sir answered your question and I'll get back to you regarding trees.

[laughter].

Speaker 1: Your next question comes from Malayana of Joseph Schwartz of Lerink Partners. Your line is open.

Operator: Your next question comes from the line of Joseph Schwartz of Leerink Partners. Your line is open.

Your next question comes from the line is Joseph Schwartz of link partners. Your line is open.

Hank Fuchs: Foundation. In addition, we plan to initiate two new clinical programs next year with Fox OGO, one in idiopathic, or polygenic short stature, and the other in defined genetic short stature conditions. We know that CNP is a pivotal physiologic stimulator of endocundral bone growth on the level of the growth plate. Further, human genetic studies of CNP and not FGFR-3 demonstrate that even modest nudges and CNP signaling have a significant impact on final adult height.

Hank Fuchs: This supports our confidence that Fox OGO can deliver on the transformational improvement in growth across many forms of skeletal impairment, while retaining the strong safety profile demonstrated in acondriplasia. For these reasons, we look forward to starting the clinical programs in these additional stature indications in 2024.

Speaker 10: Great, thanks very much and best wishes to JJ. That's truly the end of an era. So I guess.

Joseph Schwartz: Great. Thanks very much, and best wishes to JJ. That's truly the end of an era. So, I guess, I was wondering if you can help us understand what kinds of initiatives you've undertaken to address the administrative delays and site readiness issues and, you know, what kinds of success have you had where you have implemented these things, and what initiatives do you have left to implement still to support ROCTAVIAN's growth? Any more color there would be very helpful.

Great. Thanks, very much and best wishes to J J.

That's true.

So I guess.

Speaker 10: I was wondering if you can help us understand what kinds of initiatives you've undertaken to address the administrative delays and site readiness.

Was wondering if you can help us understand what kinds of initiatives you've undertaken to address the administrative.

Administrative delays and uhm slight readiness issues and you know what.

Speaker 10: issues and, you know, what kinds of success have you had where you have implemented these things? And what initiatives do you have left to implement still to support rock TV and growth? Any more color there would be very helpful.

Kinds of success have you had where you have implemented these things and what initiatives do you have left to implement.

Still to support <unk>.

Any more color there would be very helpful.

Jeff Ajer: Thanks, Joe, for your question. I would characterize the answer to that as less specific initiatives and more just, you know, the nuts and bolts and mechanics of selling, of providing scientific support to HTCs, the physicians, and patient advocacy groups, including local chapter events. You know, all the things that help them through those little steps and lots of little decisions, including the big decision of affirmatively making the decision to treat. And as Hank and I have discussed, just this week, that we're really focused on those small, tactical, on-the-ground elements with our teams to just keep those things moving through the various little challenges that are popping up.

Thanks for your question I would characterize the answer to that is less specific initiatives and more just you know the the nuts and bolts and mechanics of selling or providing scientific support H T C.

Speaker 4: Thanks, Joe, for your question. I would characterize the answer to that is less specific initiatives and more just, the nuts and bolts and mechanics of selling, of providing scientific support to HTC's, physicians, patient advocacy groups, including local chapter events.

Hank Fuchs: Please take a look at the full R&D day presentations of the latest update on our seven portfolio candidates discussed in September, and we look forward to keeping you apprised as they progress through development.

Brian Mueller: And thanks for your support, and I'll turn the caller to Brian to update financial results in the quarter of Brian. Thank you, Hank, and thank you, JJ, as well.

The physicians patient advocacy groups, including local chapter events.

Speaker 4: You know, all the things that help them through those little steps and lots of little decisions, including the big decision of Affirmatively making the decision to treat and as Hank and I have discussed this week, we're really focused on those

Brian Mueller: Please refer to today's press release, summarizing our financial results for full details on the third quarter of 2023. Then, JJ and Jeff spoke to our revenue performance for the quarter and future revenue outlook. I will primarily focus on the remainder of our P&L and other key financial updates this quarter. As usual, all results will be available in our upcoming form 10Q, which we expect to file tomorrow. We are pleased with our year-to-date revenue growth of 14 percent and non-gap earnings per share growth of 33 percent, which well positions us to achieve our top and bottom line financial objectives in 2023, as indicated by our narrowed guidance provided today on total revenue and earnings per share.

You know all the things that help them through those little steps in and lots of little decisions, including the big decision of affirmatively, making the decision to treat and as as Hank and I have discussed just this week, we're really focused on those.

Speaker 3: small tactical on the ground elements with our teams to just keep those things moving through the various little challenges that are popping up. So it's more about dealing with those little things, getting to the other side of many of which are one time events and less about introducing new initiatives. Thank you. Anytime you ask me, are you getting Germany again? Until you have your precious don't pay for drugs.

Small tactical on the ground elements with our teams to just keep.

Keep those things moving through various little challenges that are that are popping up. So it's it's more about dealing with those little things getting to the other side of many of which are one time events and less about introducing new initiatives. Thank you.

Jeff Ajer: So it's, it's more about dealing with those little things, getting to the other side of them, many of which are one-time events, and less about introducing new initiatives.

Joseph Schwartz: Thank you.

Jean-Jacques Bienaimé: And if I may ask you, regarding Germany again, so in Europe, you know, patients don't pay for drugs. They basically don't pay either for doctor's visits, so they don't know the price of a drug. For one, so the key thing in Europe is to have official government reimbursement for your drug. That's about to happen in Germany and Italy. We basically, as we've said, we have an agreement on the price in Germany already. Now we need to have this price published, which should occur over the next few weeks. Once that happens, there is no barrier whatsoever left, you know, in Germany, because the 60 patients that are AAV5 negative in Germany, it's very likely that the vast majority of them-

And you have to ask you are you getting Germany again.

Brian Mueller: As underscored in today's guidance updates, we continue to expect double-digit revenue growth, coupled with meaningful P&L leverage with gap and non-gap earnings per share growth faster than revenue. Moving on to Q3 results, we have earned $581 million of total revenue in the third quarter of 2023 through 15 percent, compared to the third quarter of 2022, and 19 percent on a constant currency basis. Boxogo's strong performance and healthy growth in the enzyme products marketed by Biamarin drove this result with some partial offsets of lower QVAN revenue due to continued generic competition, and now there is time timing of order fulfillment to Sonofi.

<unk> don't pay for drugs.

Speaker 3: They don't they don't pay either for doctors visit so they don't they don't know the price of a drug But once so the key thing in Europe is to have official government reimbursement for your drug That's about to happen in Germany and Italy we basically as we say we have Agreement on the price in Germany already now we need to have this price published We should occur over the next few weeks once that happens

They don't really busy you don't pay either for a doctor's visit so they don't they don't know the price of a dry but one so the key thing in Europe is to have official government reimbursement for Ya drive that's about to happen in Germany recently, we basically as we said we have an agreement on the price in Germany already know we need to have this price pug.

<unk>, we should occur over the next few weeks once that happens.

Speaker 3: There is no barrier whatsoever left in Germany from the 16 patients that are 85 negative in Germany. It's very likely that the vast majority of them will be treated. Obviously most of them next year because they are so few weeks left in the year because of the holiday season coming up pretty soon. And the fact that the price probably won't be published for another three, four weeks.

There is no barrier whatsoever less.

<unk> the 60 patients 85, and you can even Germany, it's very likely the vast majority of them will be treated.

C. Greg Guyer: ... will be treated. Obviously, most of them are next year because there are so few weeks left in this year because of the holiday season is coming up, you know, pretty soon, and the fact that the price probably won't be published for another, you know, three, four weeks.

Jean-Jacques Bienaimé: ... will be treated. Obviously, most of them are next year because there are so few weeks left in this year because of the holiday season is coming up, you know, pretty soon, and the fact that the price probably won't be published for another, you know, three, four weeks.

Obviously most of them next year.

Brian Mueller: The strength of our portfolio, consisting of eight commercial products, allows us continued patience on the pioneering nature of the rock-tvian launch, specifically the ability to maintain our original full year 2023 total revenue growth expectation, which represents 16 percent of year over year growth at the midpoint, and are implied Q4 2023 growth rate at the guidance midpoint of over 20 percent. We remain confident in the long-term prospects of rock-tvian and its potential to drive growth in 2024.

Because there are so few weeks left.

Because.

Because of the holiday season is coming up pretty soon is the fact that the.

Price probably won't be published for another three four weeks.

Speaker 1: Your next question comes from a line of Chris Raymond of Piper Sandler. Your line is...

Operator: Your next question comes from the line of Chris Raymond of Piper Sandler. Your line is open.

Your next question comes from the line of Chris Raymond Piper Sandler Your line is open.

Speaker 11: Thanks and congrats from us to JJ and sorry to see you leave JJ and really enjoyed working with you all these years. I've got two questions maybe one a commercial one maybe for Jeff and then one for Hank.

Chris Raymond: Thanks, and congrats from us, too, to JJ, and sorry to see you leave, JJ, and really enjoyed working with you all these years. I've got two questions, maybe, one, a commercial one, maybe for Jeff, and then one for Hank, if that's okay. So maybe just on VOXZOGO, Jeff, just on that supply constraint issue, maybe could you talk a little bit about the triage process that you've instituted with new patient starts? I guess I'm just kind of wondering, what kind of retention programs do you have in place for those new patients who want to be on therapy but can't?

Thanks, and uhm, congrats from us to to J, J and sorry to see you leave J J and really enjoyed working with you all these years.

I've got two questions, maybe one a commercial one maybe for Jeff and then one for Hank if that's okay. So maybe just <unk> <unk>, Jeff just done that supply constraint issue, maybe could you talk a little bit about the triage process that you've instituted with new patient starts.

Brian Mueller: With respect to growth margins, Q3 2023 growth margin of 78.5 percent is an improvement of 1.6 percentage points, as compared to the third quarter of 2022. R&D Expense in Q3 2023 was $191 million, and SG&A Expense was $224 million, which combined together a total of $415 million and grew 11% versus last year. As compared to 15% revenue growth in the quarter, this aligns with our goal of growing operating expenses slower than revenue to deliver operating margin expansions for the full year.

Speaker 11: So maybe to send Vax, Vaxogo, Jeff.

Speaker 11: Just on that supply constraint issue, maybe could you talk a little bit about the triage process that you've instituted with new patient starts? I guess I'm just kind of wondering, what kind of retention programs do you have in place for those new patients?

I guess I'm, just kind of wondering what what kind of retention programs do you have in place for those new patients.

Speaker 11: who want to be on therapy but can't. And can you give us a sense of that number now and where you expect it to grow to, you know, when you get back up to supply mid next year and start to treat those patients.

I Wanna be on therapy, but can't.

Chris Raymond: Can you give us a sense of that number now and where you expect it to grow to, you know, when you get back up to supply mid-next year and start to treat those patients? Then maybe on the R&D side, Hank, just noticing back at your R&D Day, which was just in September, I think, your cardiomyopathy programs, I think 293 was supposed to have its initial data proof of concept in 2025, and I think 365 was the next year after. It looks like those have been pushed a year. It's only been a, you know, few weeks since your R&D Day. Maybe is there a sense of what's happening there? Thanks.

And can you give us a sense of that number now.

Where do you expect it to grow too you know when you get back up to supply mid next year and start to to treat those patients.

Speaker 11: And then maybe on the R&D side, Hank, just noticing back at GRND Day, which was just in September , I think you, Cardiomaiopathy programs, I think 293 was supposed to have its initial data proof of concept in 2025.

Then maybe on the on the R&D side Hank.

Brian Mueller: The increase in R&D in SG&A was primarily driven by the development of our early stage pipeline and global commercial launch activities for Voxfogo and Rock-Tagging. As we look ahead to the fourth quarter, we anticipate that total SG&A and R&D expense will be higher than Q3 at a dollar basis due to the same drivers. Moving down the P&L, we continue to see an increase in interest income due to higher yields on our cash and investment.

Just noticing back it uhm guarantee day, which was just in September I think uhm your cardio <unk>, you're cardiomyopathy programs. I think 293 was supposed to have its initial data proof of concept in 2025.

And I think 365 was the near your after it looks like those have been pushed a year.

It's only been a few weeks since you're already day, maybe is there a sense of what's what's happening there. Thanks.

Brian Mueller: On tax expense for the quarter, I will note that we recognize tax expense at a lower effective rate during Q3 due to some discrete tax benefits during the quarter. This lower tax expense in Q3 will likely modestly benefit the full year tax as well, but we continue to project that our global annual effective tax rate will be in the 20 to 23 percent range over the next few years. On the bottom line, we delivered on our commitment to profitability with $40 million of gap net income in Q3, $23, and $89.5 million of non-gap income.

Speaker 4: I think, great. So let's start with the box. So goes like in strain issue and I'll ask Greg and Brian to jump in if they think I'm missing any here, but qualitatively what I can tell you is if by the time we were looking at our patient demand and the resulting unit demand forecast.

Jeff Ajer: Thanks, Chris. So let's start with the VOXZOGO supply constraint issue, and I'll ask Greg and Brian to jump in if they think I'm missing any here. But qualitatively, what I can tell you is, you know, by the time we were looking at our patient demand and the resulting unit demand forecast, the very beginning of this year, we anticipated that we would be getting to a point during the course of 2023 that we would not be able to satisfy all of the patient demand that we saw springing up in all of our commercial footprint. A reminder that we're active now in 38 markets. So our objective was twofold during the course of the year.

Thanks, Great. So let's start with the <unk> issue and I'll I'll ask Greg and Brian to jump in if they think I am I missing any here, but qualitatively what I can tell you is yeah, but by the time, we were looking at our our patient to me.

And in the resulting unit demand forecasts the very beginning of this year, we anticipated that we would be getting to a point during the course of 2023 that we would not be able to satisfy all of the patient dinette demand that.

Speaker 4: The very beginning of this year, we anticipated that we would be getting to a point during the course of 2023 that we would not be able to satisfy all of the patient demand that we saw springing up in all of our commercial footprint or reminder that we're active now in 38 market.

Brian Mueller: This level of profitability for Q3 and year-to-date gives us confidence to achieve our stated objectives of sustaining growing profitability on a full year basis going forward. Year-to-date 2023 non-gap diluted earnings per share has increased 33 percent as compared to Q3 year-to-date last year, and today we have reaffirmed our mid-point guidance for non-gap diluted earnings per share for the full year. In closing, we are on track to meet our top line and bottom line objectives for 2023 and beyond with strong double-digit growth in both revenue and profitability.

But we saw springing up in all of our commercial footprint reminder, that were active now in 38 markets. So our our objective was two fold during the course of the year at the first <unk> sure or do our best to ensure that if we were starting page.

Speaker 4: So our objective was twofold during the course of the year. And the first was to ensure or do our best to ensure that if we were starting patients on Boscogo therapy in any of those markets.

Jeff Ajer: The first was to ensure or do our best to ensure that if we were starting patients on VOXZOGO therapy in any of those markets, that we could assure the supply continuity behind those specific patients and that we would minimize or eliminate the risk that patients starting therapy would have to go off therapy for either a very short or a prolonged time, which required a lot of forward planning. A reminder, you hear a lot about the different dynamics of markets. You know, the different markets look very different. So having, you know, a 38 market footprint to be projecting into, projecting those patient demands, looking at our labeled product supply, going out, you know, 6 and 9 months forward, was a pretty dynamic situation to manage.

<unk>, So I'll go therapy in any of those markets.

Speaker 4: that we could assure the supply continuity behind those specific patients.

We could assure the supply continuity behind those specific patience and we would minimize or eliminate the risks the patient starting therapy would have to go off therapy for either a very short for a prolonged time, which required a lot of forward <unk>.

Speaker 4: and that we would minimize or eliminate the risk that patients starting therapy would have to go off therapy for either a very short or a prolonged time, which required a lot of forward planning. And a reminder, you hear a lot about the different dynamics of market.

Brian Mueller: We continue to success the box-o-go, the momentum building for Octavian and ongoing margin expansion efforts gives Biomerin a strong foundation to achieve our financial goal of being one of the industry leaders in revenue growth and margin expansion as we close 2023 and head into 2024.

<unk> and and a reminder, you hear a lot about the different dynamics of markets. Yeah. The different markets look very different so having you know 38 market footprint to be projecting indu projecting those price and demand looking at are labeled products supply going out.

Speaker 4: You know, the different markets look very different. So having, you know, a 38 market footprint to be projecting into projecting those patient demand.

Operator: Thank you for your attention, and we will now open up the call for questions. Thank you. If you have a question, please press star one on your telephone keypad. If you wish to remove yourself from the queue, simply press star one again. One moment for your first question.

Speaker 4: Looking at our labeled product supply, going out six and nine months forward, was a pretty dynamic situation to manage. But getting on top of that early allowed us to say in certain markets.

Six nine months forward was a pretty dynamic situation to manage but getting getting on top of that early allowed us to say in certain markets.

Salveen Richter: Your first question comes from line of Selvian Richter of Goldman Sachs. Your line is open. Good afternoon. Thanks for taking my question. And, JJ, it's been a pleasure working with you. Good luck on the next venture here. With regard to my question, you talked about the, with Octavian, the HTC preparedness that's required, and then you also provided some context of two to five months to prepare patients for treatment. Can you just break that down for us?

Jeff Ajer: But getting on top of that early allowed us to say, in certain markets, we're gonna delay our entry into that market because we can't be assured of keeping patients on therapy once we start them. But also having a forward-looking date that we'd be able to communicate and say, "Look, if we can't get into this market on, you know, May of this year," for example, with an increasing supply, still supply constrained, but supply increasing, when could we project that market to come online for patients to be treated? Those are the kind of dynamics that we were working through.

Speaker 4: We're going to delay our entry into that market because we can't be assured of keeping patients on therapy once we start them, but also having a forward looking date that would be able to communicate and say, look, if we can't get into this market on...

We're gonna delay our our entry into that market, because we can't be assured of of keeping patients on therapy. Once we start them, but also having a forward looking date that we'd be able to communicate say look if we can't get into this market on.

Salveen Richter: Like the time period needed for HTC, the then kind of the payer dynamics that will play out the diagnostic aspect as well, and how demand is stacking up as you get a sense from physicians and the centers right now. Thank you.

Speaker 4: May of this year, for example, with an increasing supply, still supply constraint, but supply increasing, when could we project that market to come online for patients to be treated?

May of this year for example, with with an increasing supply still supply constrained, but supply increasing when could we project that market to come online for patients to be treated those are the kind of dynamics that we were working through N. As you can.

Speaker 4: Those are the kind of dynamics that we were working through. And as you can tell by the different quarterly results in our remarks during the year, we've now kind of reached that point where there are patients in certain markets that we are attempting to not get started on therapy because we can't.

Jeff Ajer: And as you can tell by the different quarterly results and our remarks during the year, we've now kind of reached that point where there are patients in certain markets that we are attempting to not get started on therapy because we can't guarantee supply. And there are markets that we have earmarked for the future to open up and begin treating patients because we couldn't guarantee the continuity of supply for earlier starts. Maybe I'll pause there and look at Brian or Greg for additional comments.

You can tell by the different quarterly results in our remarks during the year. We've we've now kind of reached that point, where there are patients in certain markets that we are are attempting to not get started on therapy, because we can't.

Jeff Ajer: Hi, Salveen, I'll take a shot at that one. There's a lot of moving parts in this launch. There is the specific HTC dynamic, which is not unique to Octavian, so you may be getting some insights and do how that's playing out, you know, in a parallel launch in the United States. The HTC's need to be prepared to be prepared both for the administration of a one-time treatment with Octavian, which includes handling a frozen product, a very high value.

Jeff Ajer: It includes other dynamics like 340B discounting and pricing, relative in some cases to larger healthcare systems that may be apparent organization and have some capabilities that the HTC does or does not have. So there's kind of those dynamics inside of the HTC. There's also the paired dynamics going on, which you would expect anyway in the United States. But in this case, the paired dynamics are complicated by the fact that gene therapies, you know, in a high value, one-time treatment are new and different.

Speaker 12: Guarantee supply and there are markets that we have earmarked for the future to open up and begin treating patients because we couldn't guarantee the continuity of supply for earlier starts Maybe I'll pause there and look at Breiner Greg for additional comments. Yeah, Chris. This is Greg. I'll just add So they should think about it is and Jeff stated it for the end of the to the fourth quarter

Guarantee supply and there are markets that we have earmarked for the future to open up and begin treating patients because we couldn't guarantee the continuity of supply for earlier starts maybe I'll pause Erin look at Brian or Greg for additional comments Christmas. This is Greg I'll just add so what you should think about.

C. Greg Guyer: Yeah, Chris, this is Greg. I'll just add, so the way you should think about it is, and Jeff stated it, for the end of Q4, we'll add 300 new patients, as you said. What you have to think about is we have done a lot of work since we were last on a call with you. Q1 is less constrained, Q2 is almost close to not constrained, and by midyear, we'll have no constraint. So it is getting better by the day, so it's not like some event happens in midyear that gets us to totally unconstrained. We're gonna be building inventory and building supply, and able to support more and more of those patients.

Greg Guyer: Yeah, Chris, this is Greg. I'll just add, so the way you should think about it is, and Jeff stated it, for the end of Q4, we'll add 300 new patients, as you said. What you have to think about is we have done a lot of work since we were last on a call with you. Q1 is less constrained, Q2 is almost close to not constrained, and by midyear, we'll have no constraint. So it is getting better by the day, so it's not like some event happens in midyear that gets us to totally unconstrained. We're gonna be building inventory and building supply, and able to support more and more of those patients.

It it is and and just stayed there for the end of the <unk> to the fourth quarter.

Speaker 12: We kind of will add 300 new patients as he said. What you have to think about is we have done a lot of work since we were last on a call with you.

We kind will add 300, new patient says you said what you have to think about is we have done a lot of work since we were last on the call with you and first quarter is less constrained second quarter is.

Speaker 12: And first quarter is less constrained. Second quarter is

Speaker 12: almost close to not constrained and by mid year will have no constraint. So it is getting better by the day. So it's not like some event happens in mid year. It gets us to totally unconstrained. We're going to be building inventory and building supply and enable to support more and more of those patients. There is a significant uplift in patients in the first half the next year that we'll be able to support. And then by mid year we'll be fully unconstrained for all those markets, all those patients.

Close to not constrained by mid year will have no constraint. So it is it is getting better by the day. So it's not like some of it happens in mid year. It gets us to totally unconstrained, we're gonna be building inventory and building supply and enable to support more and more of those patients. There is a significant uplift.

C. Greg Guyer: There is a significant uplift in patients in the first half of next year that we'll be able to support, and then by midyear, we'll be fully unconstrained for all those markets, all those patients. So think of it as a taper over time, that we will start to go after those patients, as opposed to some event that will consume all those patients in midyear, if that helps.

Greg Guyer: There is a significant uplift in patients in the first half of next year that we'll be able to support, and then by midyear, we'll be fully unconstrained for all those markets, all those patients. So think of it as a taper over time, that we will start to go after those patients, as opposed to some event that will consume all those patients in midyear, if that helps.

And patients in the first half of next year that we'll be able to support and then by mid year will be fully unconstrained for all those markets. All those patients. So <unk> think of it as a taper overtime that we will start to go after those patients as opposed to some event that will consume all those patients and this and that.

Jeff Ajer: We've known and expected the payers will take some time to get their hands around that issue for years, and we've been preparing for that eventuality. There's also patient awareness and interest in a highly competitive and very dynamic changing standard of care environment. There's positions that have a number of options available to them. There's all those dynamics going on in parallel, and we're navigating all of those issues in parallel some are taking some time.

Speaker 12: So think of it as a taper over time that we will start to go after those patients as opposed to some event that will consume all the patients in the mid year if that helps.

In mid year, if that helps.

Traci McCarty: Then, Chris, I just want to address your question about-

And then Chris I, just want to address your question about uhm certain earlier stage program shifting they haven't and that's part of our ongoing efforts to streamline communications were gonna be updating the street when we have clinical proof of concept. So those dates just the point in time when we're communicating at eight.

Speaker 2: I just want to address your question about um Certain earlier stage programs shifting they haven't as part of our ongoing efforts to streamline communications We're going to be updating the street when we have clinical proof of concept so those dates just the points in time when we're communicating updates Have changed but none of the actual advances have changed

Brian Mueller: ... certain earlier stage programs shifting, they haven't. As part of our ongoing efforts to streamline communications, we're gonna be updating the street when we have clinical proof of concept. So those dates, just the points in time when we're communicating updates, have changed, but none of the actual advances have changed. So the next question?

Traci McCarty: ... certain earlier stage programs shifting, they haven't. As part of our ongoing efforts to streamline communications, we're gonna be updating the street when we have clinical proof of concept. So those dates, just the points in time when we're communicating updates, have changed, but none of the actual advances have changed. So the next question?

Change, but none of the actual advances have changed.

Jeff Ajer: I mentioned the paired dynamics specifically. What I believe is that the two thirds of US population with issued coverage policies four months after approval, I think is really encouraging and very, very fast. The uptake of the warranties, which is a brand new thing for pairs at the level that I described is very fast and very encouraging. And we're seeing the patient funnel now starting to shape take shape in the United States.

Yeah.

For the next.

Operator: Your next question comes from the line of Paul Matteis of Stifel. Your line is open.

Your next question comes from Might've Palm Metastasis Stifel. Your line is open.

Speaker 1: The next section comes from mine of Paul Mateus of Stefel. The line is open.

Speaker 8: Great, thanks so much. And I'll add my congrats to JJ as well. I appreciate the question. I would curious when you were undertaking the CEO search and succession planning over the past couple of years, what were the two to three key things that you were prioritizing? And as you were thinking on selecting new leadership, how important was it that a new CEO would really stay the course and bomber and course strategy over the past few years? ...

Great. Thanks, so much and I'll add my congrats to J J as well I. Appreciate the question I was curious when you were undertaking B C O searching succession planning over the past couple of years what were the two to three key things that you were prioritizing and as you were thinking I think you are selecting new leadership.

Paul Matteis: Great, thanks so much. And, I'll add my congrats to JJ as well. Appreciate the question. I was curious, when you were undertaking the CEO search and succession planning over the past couple of years, what were the 2 to 3 key things that you were prioritizing? And as you were thinking on selecting new leadership, how important was it that a new CEO would really stay the course in BioMarin's core strategy over the past few years? Or, I guess what I'm kinda trying to say is, as we think forward, you know, what are the chances here that Alexander Hardy, you know, does actually enact material changes over the next 6 to 12 months, and BioMarin in certain ways could look differently? Thanks so much.

How important was it that a new C E O would really stay the course and bombers core strategy over the past few years or I guess, what I'm trying to trying to say as as we as we think forward.

Jeff Ajer: So I've been describing the patient funnel over the last year in Germany. And the US patient population is substantially larger than that of Germany, but early signals in the US look like the taking shape of that patient funnel is going to have some similarities to what we've experienced in the United States. All of which gives me a great deal of confidence that as we finish up this year, hopefully treat some patients in Q4 that were really set up with pair of approvals in Italy and Germany and the dynamics in the United States falling into place. All of which point to greater uptake in 2024.

Speaker 13: or I guess what I'm kind of trying to say is as we think forward, you know, what are the chances here that Alexander Hardy, you know, does actually enact material changes over the next six to 12 months and bomber and in certain ways could look differently. Thanks.

What are the chances here that that Alexander hearty, you know actually enact material changes over the next six to 12 months in bomber in in certain ways could look differently. Thanks, so much.

Jean-Jacques Bienaimé: Yes. So, let me try to answer your question. So, we've done a pretty comprehensive search, mainly over the past year or so, that accelerated about, you know, a few months ago. And we had several very good candidates, but the board thought that Alexander was probably the best candidate. As you know, he served as CEO of Genentech since 2019. Over there, he was responsible for the company's $25 billion a year business and leading 13,500 employees. So we believe that he has demonstrated ability to deliver growth, which is what, you know, continues to be needed at BioMarin.

Yeah. So let me try to answer your question so.

Speaker 3: Yeah, so let me try to answer your question. So we got a pretty comprehensive search mainly over the past year or so that accelerated about a few months ago. And we had several very good candidates and the board thought that Alexander was probably the best candidate.

So we've got a pretty comprehensive search.

Mainly over the past year or so that excited about you know a few months ago.

And we had several good candidate and.

Ward.

That that Alexander was probably the best the best candidate.

Speaker 3: As you know, it served as CEO of Genentex in 2019.

As you know he served as you have genetic since 2019.

Jeff Ajer: Thanks for the question.

Speaker 3: Over there he went responsible for the companies. $25 billion dollars a year of business and leading 13,000 500 employees.

Over there he was responsible for the company's $25 billion a year business and.

Helene: Your next question comes from the line of just a meet of Bank of America. Helene is open.

13500 employees.

Speaker 3: So we believe that he has demonstrated ability to deliver growth.

Brian Mueller: Great, hey guys, thanks for the question, JJ also wanted to offer up congrats on the retirement. So, not surprisingly, you have a few on Rock Tavian as well. So, the first one is, I know you're not giving more detail, 24 guidance than the, you know, three billion in revenue, but at a high level, you know, how would you characterize the Rock Tavian contribution? And the second question is, I guess when you use German, Germany as a proxy, you know, you have 60 patients, you know, that are pretty late in the onboarding process.

So we we believe that he has demonstrated the ability to deliver growth.

Speaker 3: which is what is, you know, which he needs to be needed at Bound Rennes. You know, he steered genetics through the, the loss of exclusivity of three nature drugs during that time, that together accounted for 40%.

Which is worse what is.

It seems to be needed a bad man you know he he's tier genetics through the <unk> the loss of exclusivity of three nature drags you in that time.

Jean-Jacques Bienaimé: You know, he steered Genentech through the loss of exclusivity of three major drugs during that time, that together accounted for 40% of the company's revenues in 2019, and he returned the portfolio to strong growth by the end of 2022. So we believe that, you know, he has the ability to grow, to continue to grow our commercial business pretty successfully. We understand from, you know, reference checks, that he's a very good leader, and he, you know, he's been evolving and working in competitive markets, across a range of therapeutic areas, including, you know, hemophilia, as you might have noticed.

That together accounted for 40%.

Speaker 3: of the company's revenues in 2019. And he returned the portfolio to strong growth by the end of 2022. So we believe that he has the ability to grow, to continue to grow our commercial business, very successfully. We understand from reference checks that he's a very good leader. And he's been

Of the company's revenues in 2019, and he returned the portfolio too strong gross by the end of the <unk> of 2022. So so we believe that you know he has the ability to.

<unk> could you to grow our commercial business very successfully we I understand from you know reference checks that he's he's a very good leader and and you know his bill.

Brian Mueller: It's been on the market, you know, officially for over a year. Is there, is there what you characterize as a second wave, or is this 60 mostly it? I wasn't sure kind of, you know, if there's a bit of a ripple effect once you get formal reimbursement. Thank you.

Speaker 3: evolving and working in competitive markets, of course, a range of therapeutic areas, including no hemophilia, as you might have noticed.

Evolving and working in in competitive markets of course, a range of COPD areas, including.

Brian Mueller: Yeah, hey, thanks, Jeff Brian here. I'll start with your first question on the three billion, you know, as you noted, haven't given the specifics behind that. We'll plan on doing that early next year, like we typically would. But importantly, there's both continued growth in Voxfogo over 23 and a meaningful contribution from Rock Tavian. So let's get a stay tuned for the details, but you understand, you know, what has been the, you know, typical growth trajectory for the mature enzyme products, you know, still, still growing healthily here, plus substantial growth in Voxfogo, plus meaningful Rock Tavian contribution to that approximately approaching three billion. And as usual, we will give more detailed guidance, you know, when we report to 240 every week of next year.

No hemophilia is you might <unk> might have noticed.

Jean-Jacques Bienaimé: He's overseen the launch of 10 new molecular entities during his time as CEO of Genentech. So he has a strong background and understanding in drug development and science. Alexander hasn't been hired to make some major changes within BioMarin. Now he's going to assess the situation and will determine, you know, what needs to be changed, if anything, but I think we all look forward to working with Alexander.

Speaker 3: And he's always seen the launch of 10 new molecular entities during his time at CEO of Genentech. So here's a strong background and understanding in drug development and science. So, you know, it was, it was, it hasn't been hard to make some major changes within Bahamurain, are these things?

And.

And he's always seen the launch of 10 <unk>.

S C O genetic so so he has a strong background in understanding and in in drug development and science. So you know you are he was hired to make to make some major changes within biomarin, obviously to assess the situation.

Speaker 3: It sets the situation and will determine what needs to be changed. If anything, but I think we all look forward to working with an examiner.

<unk> and will determine no what what needs to be changed if anything but I think we all look forward to working with with an extender.

Speaker 1: Your next question comes from the line of a cash Ferrari of Jeffries. Your line is open.

Your next question comes from the line of a cash to Rory Jeffries. Your line is open.

Operator: Your next question comes from the line of Akash Tewari of Jefferies. Your line is open.

Speaker 14: Thanks for taking our questions. This is Ivy Alpher Cush and GJ Congress Monday retirement. The forward questions, so I guess for 2024, I know you haven't given any products with the details of four bucks tape.

[Analyst] (Jefferies): Hi, thanks for taking our questions. This is Ivy on for Akash, and JJ, congrats on the retirement. So for our questions, so I guess for 2024, I know you haven't given any product-specific details for ROCTAVIAN. What will be the cadence of new patients to add next year for both US and ex-US? 'Cause consensus right now has $350 million contribution from ROCTAVIAN, which looks quite high compared to the below $10 million guidance you have for this year. So what do you think needs to happen to hit that target, or is that, like, a fair target to say? And secondly, I think as for your mid-decade revs go up around $4 to 5 billion, are there any updated thoughts to that target?

Ivy Wang: Hi, thanks for taking our questions. This is Ivy on for Akash, and JJ, congrats on the retirement. So for our questions, so I guess for 2024, I know you haven't given any product-specific details for ROCTAVIAN. What will be the cadence of new patients to add next year for both US and ex-US? 'Cause consensus right now has $350 million contribution from ROCTAVIAN, which looks quite high compared to the below $10 million guidance you have for this year. So what do you think needs to happen to hit that target, or is that, like, a fair target to say? And secondly, I think as for your mid-decade revs go up around $4 to 5 billion, are there any updated thoughts to that target?

Hi, Thanks for taking all my questions. This is Ivy on <unk> and T. J congrats on the retirement.

Questions. So I guess for 2024, and then you'll have an gave it any product specific details for my case, well that'll be the hidden Avenue patience and next year for both the U S and <unk>.

Brian Mueller: And I think great question about the 60 AAB 5 eligible patients that have yet to be treated in Germany. Actually, the pace of CDX testing in Germany is flowed down over the summer. That's probably a reflection both of the general flow down in August in particular in Germany. And also a reflection that is I've noted previously are, you know, our earlier plan to treat patients in the free pricing period while we were negotiating with TKV wasn't paying out the way we had hoped it would.

Speaker 14: We'll be the cadence of new patients to add, next year for both US and XUS.

Contact us by now had 350 million contribution from Octavian, which looks quite high compared to the pillow 10 guidance you happy to hear that what do you think needs to happen to hit that target or is that like a fair target to say.

Speaker 15: Compared to the below 10 guidance you have for this year. So what do you think me to happen to hit that target or is it like a fair target to say and secondly, I think it's for your mid decade revs go off around four to five billion. Are there any update off to that target and if you are to break down this five billion by products, how much of it is from a single and how much of it is from a chain. Thanks. Thanks. Maybe I'll start with the big picture.

I think it's <unk> offer around four to 5 million are there any updates to that target.

[Analyst] (Jefferies): If you were to break down this $5 billion by products, how much of it is from VOXZOGO and how much of it is from ROCTAVIAN? Thanks.

Ivy Wang: If you were to break down this $5 billion by products, how much of it is from VOXZOGO and how much of it is from ROCTAVIAN? Thanks.

Q breakdown, if five yoga and byproduct how much of it is from <unk> and how much of it.

Thanks.

Brian Mueller: And so we know the HTC's were slowing down in Germany waiting for the GKV process to be finished. My perception is a pickup following the end of the summer in CDX activity and lining up patients to be treated in Germany is both reflective of one getting past the summer and to an expectation that these HTC's know, eventually, that we've come to conclusion on price. They're just waiting for that price to be published so they can move ahead with patient treatment.

Brian Mueller: Maybe I'll start with the big picture, revenue question. This is Brian, then, and Jeff can maybe touch on the cadence briefly. So I touched on this little bit in Jeff's question earlier. The key takeaway from the approaching $3 billion of revenue expectation for 2024 is that ROCTAVIAN is expected to come online globally, mostly Germany and the US. In that, the challenges and delays that we've discussed for 2023, in the context of 2024, we expect to be timing. Jeff and JJ touched on the German price dynamics. We've got the patient funnel filling up with what we believe to be the final gate, which is concluding the price and getting the publication in the National Register. That should open up that pool of patients for 2024.

Maybe I'm, sorry, I'll start with the Big picture revenue questions. This is Brian then and Jackson <unk>. The cadence briefly so touched on this a little bit in and just question earlier.

Speaker 6: Maybe I'll start with the big picture, revenue questions, this is Brian , then Jeff can maybe touch on the cadence briefly. So I touched on this a little bit in Jeff's question earlier that the

The key take away from.

Speaker 6: The approaching $3 billion of revenue expectation for 2024 is that...

The approaching 3 billion dollar revenue expectation for 2024.

Is that.

Speaker 6: Octavian is expected to come online globally, mostly Germany and the US.

<unk> is expected to come online globally, mostly Germany in U S.

Speaker 16: and that the challenges and delays that we've discussed for 2023.

And that the challenges and delays that we've discussed for 2023.

Brian Mueller: And while we're on the subject of Germany, which the physicians in Germany and the data on other launches would bear out. So Germany moves slow for adoption of new hemophilic options. Having 60 patients in Germany that are AAV live eligible on a population based adjusted basis in the United States would equate to about 240 to 250 patients. So just benchmarking what may be the possibilities in the U.S. Thank you.

In the context of 2024, we expect to be timing.

Speaker 16: In the context of 2024, we expect to be timing.

Speaker 16: Yeah, F&JJ touched on the German price dynamics. We've got the patient funnel filling up with what we believe to be the final gate, which is concluding the price and getting the publication in the national regi-

Yeah, J J touched on the German price dynamics, we've got the patient funnel filling up with what we believe to be the final gate, which is including the price and getting the fabrication in the National Register.

Speaker 16: That should open up that pool of patients for 2024. In the US, Jeff, on a couple of the answers has talked about these dynamics that are coming together positive momentum, but that's why we use the terminology network to bring all of those pieces together.

That should open up.

<unk> pull of patience for 2024, and the newest Jeff on a couple of the answers has talked about these dynamics that are coming together positive momentum, but that's why we use the terminology network to bring all those pieces together.

Brian Mueller: In the US, Jeff, on a couple of the answers, has talked about these dynamics that are coming together, positive momentum, but that's why we use the terminology network to bring all those pieces together. Patient, physician interest, site readiness, contracting and reimbursement. They all need to come together to have patients come online. That's taken longer in 2023, but we expect to be making significant progress over the next few months, where we'll start to see those patients come online in 2024. So the key takeaway from that $3 billion is that there is a meaningful piece of ROCTAVIAN there, which is a bridge from where we're at in 2023. And then briefly, no changes to the $4 to 5 billion. Obviously, we need to continue to-

Speaker 16: patient physician interest, sight readiness, contracting and reimbursement. They all need to come together to have patients come online. That's taken longer in 2023, but we expect to be making significant progress over the next few months where we'll start to see those patients come online in 24.

<unk> physician interests site readiness contracting reimbursement they all need to come together to have patients come online that's taken longer than 2023.

Phil Nadeau: Your next question comes from the line of Phil Nadeau of TD Cowan. Your line is open. Good evening. Thanks for taking our questions.

Back to be making significant progress over the next few months, where it will start to see those patients come on line and 24. So the key take away from that $3 billion is that there is a meaningful piece of rock T V. In there, which is a bridge from where we're at 23 and then briefly no changes to the four to 5 billion, obviously, we need to continue to modern.

Phil Nadeau: JJ, let me add our congratulations on a very successful tenure and your retirement from Biomarin. Again, a couple follow-ups on Rock Tavine, specifically on patient demand. In the last earnings call, you mentioned that patient consent forms had been trending well and environment was happy with the rate that they had started to come in. Any update on how patient consent forms are trending and is there any visibility on when the first US patients could be treated.

Speaker 6: So the key takeaway from that $3 billion is that there is a meaningful piece of rock TV in there, which is a bridge from where we're at in 23. And that briefly, no changes to the $4 to $5 billion. Obviously, we need to continue to...

Speaker 16: Monitor and execute on both box, so go and rock TV and we've commented previously that that four to five billion is roughly made up of half of the base business with continued growth in half of rock TV and box.

Jeff Ajer: ... monitor and execute on both VOXZOGO and ROCTAVIAN. We've commented previously that $4 to 5 billion is roughly made up of half of the base business with continued growth and half of ROCTAVIAN and VOXZOGO.

Brian Mueller: ... monitor and execute on both VOXZOGO and ROCTAVIAN. We've commented previously that $4 to 5 billion is roughly made up of half of the base business with continued growth and half of ROCTAVIAN and VOXZOGO.

<unk>.

Execute on both of <unk>. We've commented previously that that $4 billion to $5 billion is roughly made up of half of the base business with continued growth and half of <unk>.

Phil Nadeau: Let's question one. In terms of the institutional challenges, we've had some physicians mention things like training staff, beds need to be set up, equipment needs to be ordered. Are those challenges at the HTC's and if so, is there anything Biomarin can do to remove those bottlenecks? Thanks. Thank you for the question, Phil.

Jean-Jacques Bienaimé: But I think we refer you to some comments that answers to questions that we had previously on the call that should address your questions because they were asked earlier. Sorry, so we're running out of time, so.

Speaker 3: But I think we refer you to some comments that answers to questions that we had previously on the call that Dwets your questions because they were asked earlier. Sorry, so we were waiting our time

But I think we refer you to some comments.

Answers to questions that we had previously on the call that suited to answer your questions because.

They were asked earlier, sorry, we're running out of time.

Speaker 1: Your next question comes from Line of Jessica Phi of JPMorgan. Your line is open.

Your next question comes from the line of Jessica five J P. Morgan Your line is open.

Operator: Your next question comes from line of Jessica Fye of JP Morgan. Your line is open.

Phil Nadeau: Let me start with the second point for the institutional challenges. In the prepared remarks, I addressed HTC site readiness and some of the elements of that that I think are one time events that won't be recurring. The types of things that you just mentioned, I would say generally are captured in that HTC site readiness and some of the institutional things that they're doing to get ready for Rock Tavine treatment in particular, and taking a little bit of time, which should be resolved and facilitate patients to move expeditiously through those type of things going forward.

Speaker 17: Hey guys, thanks for taking my question and JJ Best wishes for your retirement.

Jessica Fye: Hey, guys. Thanks for taking my question, and JJ, best wishes for your retirement. Recognizing what you're doing to manage supply with VOXZOGO, can you just talk qualitatively about the geographic mix of VOXZOGO sales and which regions or countries have the most growth left in front of them in achondroplasia?

Hey, guys and thanks for taking my question and J J task vicious for your retirement.

Speaker 17: Recognizing what you're doing to manage supply with Voxogo, can you just talk qualitatively about the geographic mix of Voxogo sales and which regions or countries have the most growth left in front of them in a contract pleasure?

Recognizing what you're going to need to supply with Foxtel, though can you just talk qualitatively about the geographic next <unk> sales and which regions or countries have been lost across left in front of them any contemplation.

Speaker 4: Yeah, I don't think that we're disclosing market level detail. Yes, but overall mentioned that we're 12% patient penetrated of the indicated patient population.

Jeff Ajer: Yeah, I don't think that we're disclosing market-level detail, Jess, but overall, mentioned that there were 12% patient penetration of the indicated patient population, which includes the four months and up in Europe and from birth, essentially, in the United States. There is some variability in uptake. I think I've mentioned previously that early rapid uptake in places like Germany and Japan leave them at a relatively higher penetration rate so far without ceilings, but a higher rate in other places, importantly, like the United States, which just had a huge expansion of the patient population open to us.

Yeah, I don't think that word disclosing market level detail, yes, but overall mentioned that there were 12% patient penetrated of the indicated.

Patient population, which includes the four months and up in Europe and from from birth essentially in the United States. There is some variability in uptake I think I've mentioned previously that early rapid uptake in in places like Germany and and <unk>.

Speaker 4: the four months and up in Europe and from birth essentially in the United States. There is some variability in uptake. I think I mentioned previously that early rapid uptake in places like Germany and Japan leave them at a relatively higher penetration rate so far without ceilings, but a higher rate places, importantly like the United States, which is had a

Phil Nadeau: In terms of patient demand in the United States, we're not disclosing patient level detail in the United States, but we're continuing to see as I noted that patient will build up in the US and I'm qualitatively encouraged by what we're seeing so far. In the US, we have this hub operation and in prior launches, all of the patients move through our hub essentially first before they move through the next steps towards treatment.

Pan wave them at a relatively higher penetration rate, so far without ceilings, but higher rate and other places importantly, like the United States, which just had a huge expansion of of the patient population open to us and that patient population.

Speaker 4: expansion of the patient population opened to us, and that patient population, very young patients, having the strongest value proposition for treatment, opens up a lot of penetration room in the United States, for example, probably too much detail to get into from...

Phil Nadeau: Given the nature of the HTC's and their capabilities and the fact that they operate in this multi-sponsor environment, we've been seeing patients come into our hub maybe later in the process, and not the very beginning of the process. So this is a little bit of a different dynamic for us. We're getting our hands around that one. But yes, encouraged by the patient demand that we're seeing in the United States and the patient funnel building up.

Jeff Ajer: That patient population, very young patients having the strongest value proposition for treatment, opens up a lot of penetration room in the United States, for example, probably too much detail to get into for other markets.

Very young patients, having a the strongest value proposition for treatment opens up a lot of penetration room in the United States for example, probably too much detail to get into for other markets.

Operator: Your last question comes from the line of Ellie Merle of UBS. Your line is open.

And your last question comes from the line of <unk> of UBS. Your line is open.

Operator: Your last question comes from the line of Ellie Merle of UBS. Your line is open.

Speaker 1: And your last question comes from the line of Ellie Merle of UBS. Your line is open.

Speaker 18: Yeah, I think so much for squeezing in. Just in terms of the flexible expansion studies, from a disease biology perspective, how should we think about the potential mechanistically for CNP to expand into other indications relative to FGFR inhibitors? I guess other indications where you think the biology might make more sense for CNP and not FGFR or Vifersa, or where you think CNP is particularly likely to be active? Thanks

Ellie Merle: Hi. Hey, guys, thanks so much for squeezing me in. Just in terms of the VOXZOGO expansion studies, just from a disease biology perspective, how should we think about the potential mechanistically for CNP to expand into other indications relative to FGFR inhibitors? I guess, are there indications where you think the biology might make more sense for CNP and not FGFR or vice versa? Or where you think CNP is particularly likely to be active? Thanks.

Oh, thanks, so much for sleeping ma'am just in terms of the Occidental expansion studies.

Phil Nadeau: In addition to the federal building, I mean, it is possible, I think that was your question. Let me treat some US patients by Iran. I would say, yes, it is possible. We have three US patients. We have now cleared all repayment screens for treatment, like the AV-5, Lever Scan, Lever Health, and all that. So the timing of their treatment isn't guaranteed in 23, but they have they have complete eligibility testing for treatment. And so it will very likely occur in and out to this. You sure?

I'm in disease Biology perspective, how should we think about the potential mechanistically for C. N P to expand into other indications Martin to actually have fine hamburgers and it's R. There are indications, where you think the biology might make more sense for C. N T and not actually offer or vice versa. Alrighty. Thanks, the M. P S, particularly likely to be active.

Thanks.

Speaker 5: Lesson on you, Ellie, for asking questions so that I could both say unequivocally to Robyns earlier question I'm here in very committed to Bimeron and partly for exactly this reason I think that when you look at the genetics of

Hank Fuchs: Blessings on you, Ellie, for asking the question, so that I could both say unequivocally to Robin's earlier question, I am here and very committed to BioMarin, and partly for exactly this reason. I think that when you look at the genetics of statural impairments, and our group has actually published quite nicely on this, that gain-of-function mutations in CNP confer a height benefit, loss-of-function mutations convey short stature across a polygenic background. That, I think, is very strong evidence that CNP is gonna be a dominant or, you know, regulator of bone growth. You know, interestingly, you can find loss of function, or sorry, gain-of-function variance in FGFR3. When they are associated with stature, they're also associated with profound other abnormalities.

Blessings on your early for asking the question so that I could both say unequivocally to Robbins earlier question I'm here and very committed to buying ranch and partly for exactly. This reason I think that when you look at the genetics of of Satchel impairments in our group is actually published quite nicely on.

Robyn Karnauskas: Your next question comes from the line of Robyn Karnauskas of Truth.

Robyn Karnauskas: Your line is open. Great.

Speaker 5: of of of of of statureal impairments in our group is actually published quite nicely on this that gain a function mutations in CNP can for a high benefit loss of function mutations convey short stature across a polygenic background that I think is very strong evidence that CNP is going to be a dominant or you know regular or bone growth. You know interestingly you can find loss of function or start gain a function variance in FTFR 3 when they are associated with stature they're also associated with profound other abnormalities

Robyn Karnauskas: I have three quick questions. Number one, JJ, you have mentored so many people on the street. It's so appreciative. Is Hank Rotary too? Because that we will have the other pack. So, like, would you like an announcement on that call? Is Tracy getting a promotion? That's our first question.

<unk> that gain a function mutations in C. N P can for height benefit loss of function mutations convey short stature cross apologetic background that I think is very strong evidence that C. N. P is gonna be a dominant regular.

Regular <unk> you know interestingly you can find lots of function or sorry gene function variance in F. G. F. R. Three when they are associated with stature. There also associated with profound other abnormalities. So that's why we think that the C. N. P. Access is gonna be the perfect target for improving stature across a wide range of both Jeanette.

Robyn Karnauskas: Second question. Question would be, Rock Tavian, there was such a focus, and you misguided this year on the outcome of what you could achieve. Do you think in 2024 you can do better? And then I think on Vaxogo, I mean, that came away from our analyst meeting as like the big takeaway. So, how do you think about letting analysts understand the opportunity there at the time lunch for that? And I'll go back to Tracy's promotion. It's Hank Leyvis. I'll leave it at that.

Hank Fuchs: So that's why we think that the CNP axis is gonna be the perfect target for improving stature across a wide range of both genetic and polygenic indications, and why FGFR3 approaches are primarily gonna be limited to FGFR3 abnormalities. You can get a lot of height benefit nudging the CNP axis safely. You have to hit the FGF axis quite hard in non-FGF indications and probably too toxic effect. At least that's based on human genetic data. So thanks for the question. We're very excited about that future.

Speaker 5: So that's why we think that the CNP access is gonna be the perfect target for improving fetcher across a wide range of both genetic and polygenic indications, and why FGFR3 approaches are primarily gonna be limited to FGFR3 abnormalities. You can get a lot of height benefit nudging the CNP access safely. You have to hit the FGF access quite hard in non-FGF indications, and probably two toxic effects. At least that's based on human genetic data.

<unk> indications N Y F. G F. R. Three approaches primarily going to be limited to.

F. G F. R. Three abnormalities you can get a lot of hype benefit nudging. The C. N. P. Access safely you have to hit the F. G F access quite hard and non F. G F indications and probably too toxic effect at least that's based on human genetic data. So thanks for the question, we're very excited about the future.

Jeff Ajer: Thank you, Robin. I mean, I think there was a previous question from I think from Salveen, and we had a detailed answer from Jeff. Indeed, you know, we take it longer than we anticipated to get to connect to patients with the treatment centers because the whole system wired. I would say, again, big picture, you know, when you have such a big ticket item, like, like, Rock Tavian, $3 million, whack. It attracts a lot of people, you know, especially it's a big pie that attracts a lot of players that you already don't get involved in the decision making, because they all kind of want a piece of the action.

Speaker 5: Thanks for the question. We're very excited about this.

Speaker 1: There are no further questions at this time. I'll now turn the call over to JJBNME, Chairman and CEO .

Operator: There are no further questions at this time. I'll now turn the call over to JJ Bienaimé, Chairman and CEO.

There are no further questions at this time I'll now turn the call over to J, J B enemy chairman and CEO.

Speaker 3: Thank you, operator, and thank you for all for joining us today. We are pleased with our result, this quarter, and our prospect for that. The violin is now a profitable, fully integrated industry leader. The annual revenues, well over $2 billion, and an extremely well-positioned for substantial future growth and profitability. Thank you for your continued support and have a good afternoon or evening.

Jean-Jacques Bienaimé: Thank you, operator, and thank you, all, for joining us today. We are pleased with our results this quarter and our prospects ahead. So BioMarin is now a profitable, fully integrated industry leader with annual revenues well over $2 billion and is extremely well-positioned for substantial future growth and profitability. Thank you for your continued support, and have a good afternoon or evening.

Thank you operator, and thank you all for joining US today, we are pleased with our resolve this quarter and our prospects ahead.

<unk> profitable 40 integrated industry leader has annual revenues well over $2 billion and is extremely well positioned for substantial future growth and profitability. Thank.

Jeff Ajer: Just just mentioned the 340V, you know, 340V is 25. I mean, all basically all human beings are patients in the US. It's a statutory device or a 340V patient. You know, 340V discount of a $3 million whack drug, you're talking about $600,000. As you can understand, many, many people get interested in that pie, you know, on a slice of it, which just slows things down, but eventually, don't try to be sexually good user, we'll be taking care of.

Thank you for your continued support and have a good afternoon or evening.

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

This concludes today's conference call you may know disconnect.

Speaker 19: This concludes today's conference call. If you may now disconnect. Well, bye, all.

[music].

Jeff Ajer: So the fact that it's the takeoff has been delayed, V and to us at Bahram Reinhard, showing facts on the on the future potential of these drugs is taking longer than plan. It took longer also it was the first time we got a product of proving you know, before the US and Rock Tavian got to prove in Germany and based on our previous experiences, you know, we thought we could be able to treat some patients during the free pricing period in Germany, which is slowly disappearing.

Jeff Ajer: It didn't happen. Then we are basically very, very close to the final deal with the German authority. So we generally take about 12 to 13 months, so in this respect, we're not that far behind versus the European approval. So again, we believe productiva is great potential. We believe Rock Tavian would be a significant contributor in 2024. And beyond, obviously not as much as Voxov, which is turning into a very, very large drug based on, you know, or two, two, three results.

Jeff Ajer: And the fact that, you know, thanks to the efforts of our drug supply team, you know, all the constraints in terms of packaging that we had and feel finished with that we had this year are starting to go away. And we'll be much more substantial. We have much more substantial capacity in 23 and in Q1 and Q2. And then if we have all capacity constraints by the middle of the year, so hope that answers your question and I'll get back to you regarding Tracy.

Joseph Schwartz: Your next question comes from the line of Joseph Schwartz of Lurink Partners. Your line is open. Great. Thanks very much. And best wishes to JJ. That's really the end of an era.

Jeff Ajer: So I guess it was wondering if you can help us understand what kinds of initiatives you've undertaken to address the administrative delays and site readiness issues. And, you know, what kinds of success have you had where you have implemented these things and what initiatives you have left to implement still to support rock TV and growth. Any more color there would be very helpful. Thanks, Joe, for your question. I would characterize the answer to that is less specific initiatives and more just, you know, the nuts and bolts and mechanics of selling, providing scientific support to HTC's positions, patient advocacy groups, including local chapter events.

Jeff Ajer: You know, all the things that help them through those little steps and lots of little decisions, including the big decision of, of permanently making the decision to treat. And as Hank and I have discussed just this week that we're really focused on those small tactical on the ground elements with our teams to just keep those things moving through the various little challenges that are popping up.

Jeff Ajer: So it's more about dealing with those little things, getting to the other side of many of which are one time events, and less about introducing new initiatives. Thank you. Anytime you ask me, are you getting Germany again? So in Europe, you know, patients don't pay for drugs. They don't, you don't pay either for doctors visit. So they don't, you don't know the price of a drug. So the key thing in Europe is to have official government reimbursement for your drug.

Jeff Ajer: That's about to happen in Germany and Italy. We basically, as we've said, we have an agreement on the price in Germany already. Now we need to have this price published. We should occur over the next few weeks. Once that happens, there is no barrier whatsoever left in Germany from the 60 patients that are 85 negative in Germany. It's very likely that the vast majority of them will be treated. Obviously, most of them next year. Because there are so few weeks left in here because of the holiday season coming up pretty soon. And the fact that the price probably won't be published for another three, four weeks.

Chris Raymond: Your next question comes from a line of Chris Raymond of Piper Sandler. Your line is open. Thanks and congrats from us to JJ and sorry to see you leave JJ and really enjoyed working with you all these years. I've got two questions. Maybe one, a commercial one, maybe for Jeff and then one for Hank. If that's okay. So maybe just send Vox, Voxogo, Jeff, just on that supply constraint issue. Maybe could you talk a little bit about the triage process that you've instituted with new patient starts?

Chris Raymond: I guess I'm just wondering what kind of retention programs do you have in place for those new patients who want to be on therapy but can't? And can you give us a sense of that number now and where you expect it to grow to, you know, when you get back up to supply mid next year and start to treat those patients? And then maybe on the R&D side, Hank, just noticing back at GR&D Day, which was just in September.

Chris Raymond: I think you, Cardiama, your Cardiomyopathy programs, I think 293 was supposed to have its initial data proof of concept in 2025. And I think 365 was the year after it looks like those have been pushed a year. It's only been a few weeks since your R&D Day. Maybe is there a sense of what's happened in there? Thanks. Thanks, Chris.

Jeff Ajer: Let's start with the boxogos, Lycan strain issue, and I'll ask Greg and Brian to jump in if they think I'm missing any here. But qualitatively, what I can tell you is, by the time we were looking at our patient demand and the resulting unit demand forecasts, the very beginning of this year, we anticipated that we would be getting to a point during the course of 2023 that we would not be able to satisfy all of the patient demand that we saw springing up in all of our commercial footprint or a reminder that we're active now in 38 markets.

Jeff Ajer: So our our objective was twofold during the course of the year and the first was to ensure or do our best to ensure that if we were starting patients on boxogos, we could go therapy in any of those markets that we could assure the supply continuity behind those specific patients and that we would minimize or eliminate the risk that patients starting therapy would have to go off therapy for either a very short or a prolonged time, which required a lot of forward planning and a reminder, you hear a lot about the different dynamics of markets. The different markets look very different.

Jeff Ajer: So having a 38 market footprint to be projecting into projecting those patient demands, looking at our labeled product supply going out six and nine months forward was a pretty dynamic situation to manage. But getting getting on top of that early allowed us to say in certain markets, we're going to delay our entry into that market because we can't be assured of of keeping patients on therapy once we start them, but also having a forward looking date that would be able to communicate, say, look, if we can't get into this market on, you know, may of this year, for example, with an increasing supply, still supply constraint, but supply increasing, when could we project that market to come online for patients to be treated.

Jeff Ajer: Those are the kind of dynamics that we were working through. And as you can, as you can tell by the different quarterly results in our remarks during the year, we've now kind of reached that point where there are patients in certain markets that we are attempting to not get started on therapy because we can't guarantee supply. And there are markets that we have earmarked for the future to open up and begin treating patients because we couldn't guarantee the continuity of supply for earlier starts. Maybe I'll pause there and look at Brian or Greg for additional comments.

Greg Geier: This is Greg. I'll just add, so we should think about it is and Jeff stated it. For the end of the fourth quarter, we'll add 300 new patients as he said. What you have to think about is we have done a lot of work since we were last on a call with you. And first quarter is less constrained. Second quarter is almost close to not constrained. And by mid year, we'll have no constraint.

Greg Geier: So it is, it is getting better by the day. So it's not like some event happens in mid year. It gets us totally unconstrained. We're going to be building inventory and building supply and enable to support more and more of those patients. There is a significant uplift in patients in the first half of next year that we'll be able to support. And then by mid year will be fully unconstrained for all those markets, all those patients. So think of it as a taper over time that we will start to go after those patients as opposed to some event that will consume all those patients in the mid year. If that helps.

Hank Fuchs: I think Chris, I just want to address your question about certain earlier stage programs, shifting they haven't as part of our ongoing efforts to streamline communications. We're going to be updating the streets when we have clinical proof of concept. So those dates, just the points in time when we're communicating updates have changed, but none of the actual advances have changed.

Paul Matteis: The next question comes from my enough Paul Mateus of Stiefel, the line is open. Great, thanks so much, and I'll add my congrats to JJ as well. I appreciate the question.

JJ Ajer: I would curious when you were undertaking the CEO search and succession planning over the past couple of years, what were the two to three key things that you were prioritizing? And as you were thinking on selecting new leadership, how important was it that a new CEO would really stay the course and Biomarin's course strategy over the past few years? Or I guess what I'm kind of trying to say is as we think forward, what are the chances here that Alexander Hardy does actually enact material changes over the next six to 12 months and Biomarin in certain ways could look differently? Thanks so much.

JJ Ajer: So let me try to answer your question. So we got a pretty comprehensive search, mainly over the past year or so, that accelerated about a few months ago. And we had several very good candidates and the board thought that Alexander was probably the best candidate. As you know, he served as CEO of Genentex in 2019, over there he was responsible for the company's 25 billion dollars a year business and leading 13,500 employees.

JJ Ajer: So we believe that he has demonstrated the ability to deliver growth, which is what is continues to be needed at Biomarin. Again, he steered Genentex through the loss of exclusivity of three nature drugs during that time that together accounted for 40% of the company's revenues in 2019. And he returned the portfolio to strong growth by the end of 2022. So we believe that he has the ability to grow, to continue to grow our commercial business, very successfully.

JJ Ajer: We understand from reference checks that he's a very good leader and he's been evolving and working in competitive markets. Of course, a range of therapeutic areas, including, you know, hemophilia, as you might have noticed. And he's always seen the launch of 10 new molecular entities during his time as CEO of Genentex. So he has a strong background and understanding in drug development and science.

JJ Ajer: So, you know, it wasn't exactly hard to make some major changes within Biomarin. I'll be going to assess the situation and will determine what needs to be changed if anything. But I think we all look forward to working with an examiner.

Akash Tewari: Your next question comes from the line of Akash Tewari of Geoffrey. Your line is open. Hi, thanks for taking our questions.

Brian Mueller: This is Ivy Young for Akash, and GJ Congress Monday Retirement. The far more questions, so I guess for 2024, I know you haven't given any products with the details for a bucket tape, what will be the cadence of new patients at next year for both US and XUS? Because Constance right now has three to three million contributions from Octavian, which looks quite high compared to the below 10 guidance you have for this year.

Brian Mueller: So what do you think needs to happen to hit that target or is it like a fair target to say? And secondly, I think it's for your mid-second revs go off around four to five billion. Are there any updates off to that target? And if you are to break down this five billion by products, how much of it is from Octogo and how much of it is from Octavian?

Brian Mueller: Thanks. Maybe I'll start with the big picture revenue questions. This is Brian, then Jeff can maybe touch on the cadence briefly. So I touched on this a little bit in and Jeff's question earlier that the key takeaway from the approaching $3 billion of revenue expectation for 2024 is that Octavian is expected to come online globally, mostly Germany and US. And that the challenges and delays that we've discussed for 2023, in the context of 2024, we expect to be timing.

Brian Mueller: Jeff and JJ touched on the German price dynamics. We've got the patient funnel filling up with what we believe to be the final gate, which is concluding the price and getting the publication in the national register. That should open up that pool of patience for 2024. In the US, Jeff, on a couple of the answers has talked about these dynamics that are coming together positive momentum, but that's why we use the terminology network to bring all those pieces together.

Brian Mueller: Patient, physician, interest, sight readiness, contracting and reimbursement, they all need to come together to have patients come online. That's taken longer in 2023, but we expect to be making significant progress over the next few months where we'll start to see those patients come online in 24. So the key takeaway from that $3 billion is that there is a meaningful piece of Octavian there, which is a bridge from where we're at in 23.

Brian Mueller: And that briefly no changes to the $4.5 billion. Obviously, we need to continue to monitor and execute on both Boxelgo and Octavian. We've commented previously that that $4.5 billion is roughly made up of half of the base business with continued growth in half of Octavian Boxelgo. But I think we refer you to some comments that answers to questions that we had previously on the call that should address your questions because they were asked earlier.

Operator: Sorry, so we've only got time.

Jessica Fye: Your next question comes online of Jessica 5 of JP Morgan. Your line is open. Hey, guys, thanks for taking my question and JJ best wishes for your retirement, and recognizing what you're doing to manage supply with Voxogo.

Jeff Ajer: Can you just talk qualitatively about the geographic mix of Voxogo sales and which regions or countries have the most growth left in front of them in a contract pleasure? Yeah, I don't think that we're disclosing market level detail. Yes, but overall mentioned that we're 12% patient penetrated. The indicated patient population, which includes the four months and up in Europe and from birth essentially in the United States, there is some variability in uptake.

Jeff Ajer: I think I mentioned previously that early rapid uptake in places like Germany and Japan, we've been at a relatively higher penetration rate so far without any change. Sealings, but a higher rate and other places, importantly, like the United States, which just had a huge expansion of the patient population opened to us and that patient population, very young patients having the strongest value proposition for treatment opens up a lot of penetration room in the United States, for example, probably too much detail to get into for other markets.

Hank Fuchs: And your last question comes from the line of Elie Merle of UBS, your line is open. Yeah, thanks so much for squeezing me in. Just in terms of the flexible expansion studies, just some of the disease biology perspective, how should we think about the potential mechanistically for CNP to expand into other indications relative to FGFR inhibitors? I guess are there indications where you think the biology might make more sense for CNP and not FGFR or vice versa, or where you think CNP is particularly likely to be active?

Hank Fuchs: Thanks. Lessing on you, Elie, for asking questions so that I could both say unequivocally to Robyn's earlier question, I'm here in very committed environment. And partly, for exactly this reason, I think that when you look at the genetics of statural impairments in our group, it's actually published quite nicely on this, that gain-of-function mutations in CNP can for a high benefit loss of function mutations convey short stature across a polygenic background. That, I think, is very strong evidence that CNP is going to be a dominant or regular or bone growth.

Hank Fuchs: You know, interestingly, you can find loss of function or start gain-of-function variants in FGFR 3 when they are associated with stature, they're also associated with profound other abnormalities. So that's why we think that the CNP axis is going to be the perfect target for improving stature across a wide range of both genetic and polygenic indications. And why FGFR 3 approaches are primarily going to be limited to FGFR 3 abnormalities. You can get a lot of height benefit nudging the CNP axis safely. You have to hit the FGF axis quite hard in non-FGF indications and probably too toxic effect. At least that's based on human genetic data. Thanks for the question. We're very excited about that.

Operator: There are no further questions at this time.

JJ Ajer: I'll now turn the call over to JJ B&May, Chairman and CEO. Thank you, Operator, and thank you for all for joining us today. We are pleased with our results, this quarter, and our prospect ahead. The Biomarin is now a profitable, fully integrated industry leader. The annual revenue is well over $2 billion, and it's extremely well positioned for substantial future growth and profitability. Thank you for your continued support and have a good afternoon or evening.

Operator: This concludes today's conference call.

Operator: You may now disconnect.

Q3 2023 Biomarin Pharmaceutical Inc Earnings Call

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