BioMarin Pharmaceutical Inc.

Hello and welcome to the Biomarin Pharmaceutical 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. We'll 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 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 10Q, 10K, and 8K reports. On the call today from BioMarin Management are JJ Bien-Aimé, Chairman and Chief Executive Officer, Jeff Ager, Executive Vice President, Chief Commercial Officer, Hank Fuchs, President, Worldwide Research and Development, Greg Geyer, 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 p.m. Pacific time, and we thank you for your understanding. I will now turn the call over to Biomarine's Chairman and CEO, JJ Bien-Aimé.

Thank you, Tracy. Good afternoon. Good morning, everybody. As you will have seen this afternoon, we announced that I have decided after over 18 years with Biomarine to retire as the Chairman and Chief Executive Officer, effective December 1st of this year. And as many of you are aware, I recently actually turned 70. I cannot believe it myself. So it will not be a surprise to you that succession 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. 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 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 Lean Independent Director, Richard Meyer, with 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 of patients. Biomarine is now a profitable, fully integrated industry leader and one of the most successful biotech companies 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. Biomarine 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 at CU 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. Balmory 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 a company with now at that time 300 employees to sitting already seen in almost 80 countries around the world and more than 3,000 employees. As the company is posed for its next phase of growth with a recent introduction of Voxogo and Roktavion, 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. 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. But 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 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. So now let's turn back to the quarterly results. We were very pleased with the results in the third quarter, 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 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 Voxogo since the end of Q2, and additional strong growth is expected in Q4. As a result, today we increased full-year 2023 Voxogo guidance to between $435 million, $135 million, and $455 million. With recent U.S. and European approvals, Voxogo's 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 Voxogo treatment window. We are also pleased to share that based on increased field finish manufacturing commitments, VoxAugust supply is planned to increase from 23 levels to the first and second quarter in 2024, and is expected to be fully unconstrained by mid-year 2024. Turning now to Roktavian, where we have made tremendous progress building and connecting the network that will facilitate patient treatment. We're relaying the groundwork for patient access to Roktavian. It has taken but has been an essential step to ensuring access and reimbursement. Well over 100 people have been tested for roctavian A85 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 Roktavian, 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 Roktavian treatment, we remain encouraged by the high level of interest and demand from patients, physicians, patient advocacy groups, chemotherapy treatment centers, and payers. In Europe, we have strong progress in the quarter and expect to have final German and Italian prices for Octavian published by year-end. And in the U.S., significant momentum connecting the network to facilitate treatment is occurring. Jeff will provide more detail on large progress and our updated expectations for Octavian. Turning to Biomain's record year-to-date revenues of nearly $1.8 billion in the first nine months of the year. This represents 14% year-over-year growth, even with lower than expected contributions from Octavia. 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. The business is poised for strong growth in 2024 and beyond. With VoxHogo continuing to exceed expectations, Sorry, an Octavian Access reimbursement in place for a 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, a 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, the tremendous collaboration and fort we have built together. Thank you for continued support. I will now turn the call over to Jeff Hager to discuss the commercial business update. Jeff.