BioMarin Pharmaceutical Inc.

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. In addition, we will use non-GAAP financial metrics as defined in Regulation G during the call today. These non-GAAP measures should not be considered in isolation from, as substitutes for, or superior to financial measures prepared in accordance with U.S. GAAP, and you can find the related reconciliations to U.S. GAAP in the earnings release and earnings presentation, both of which are available in the investor relations section of our website. Please note that our commentary on today's call will focus on non-GAAP financial measures unless otherwise indicated. Beginning on slide three and introducing Biomarin's management team joining today's call, Alexander Hardy, President and Chief Executive Officer, Brian Mueller, Chief Financial Officer, Kristen Hubbard, Chief Commercial Officer, and Greg Freiberg, Chief R&D Officer. I will now turn the call over to Biomarin's President and CEO, Alexander Hardy.

Thank you, Tracy. Good afternoon, everyone, and thank you for joining us on today's call. Starting on slide five, I am very pleased with the strong results across the business, leading us to raise full year total revenues guidance at the midpoint and reaffirm our Voxogo revenue outlook for 2025. In addition to top line performance, Biomarin has delivered expanding profitability and significant growth in operating cash flow, bringing our cash and investments balance to approximately $2 billion at the end of the third quarter. We are focused on finishing the year strong, positioning ourselves to achieve record commercial results for the full year. To date this year, we have delivered 11% increase in top-line growth year over year. These strong results are driven by the performance of our global enzyme therapies and skeletal conditions business units. as we deliver for patients around the world. We've built the enzyme therapies business unit into a $2 billion plus franchise over the last 12 months with continued growth anticipated. In the skeletal conditions business unit, our first indication with our first product, FOXOGO, the treatment of achondroplasia is expected to generate more than $900 million in revenues this year. leading us to reaffirm our full year 2025 FOXOGO outlook and representing 25% growth at the midpoint of our guidance range. As the established standard of care in achondroplasia, FOXOGO revenue has increased 24% year-to-date compared to 2024. Now in the fourth year of its global launch, FOXOGO has represented a breakthrough therapy for families seeking treatment for their children with achondroplasia. Building on this innovation, we're excited to bring VoxOgo's second indication forward for the treatment of children with hypochondroplasia. We have high confidence in the upcoming pivotal data readout for hypochondroplasia expected in the first half of next year, based on proof of concept data of more than 10,000 patient years of safety and efficacy data in achondroplasia. Building on the rapid and successful global commercialization of Oxogo in achondroplasia, now available in 55 countries, we are well positioned to execute a strong global launch in hypochondroplasia, targeting 2027 should the data be supported. Kristin and Greg will provide more details in a moment. Turning now to our broader strategic objectives. Over the past 18 months, we have undertaken a series of initiatives designed to prepare BioMarin for future growth and expansion. As part of this effort, we have made difficult decisions, including the discontinuation of multiple research programs that did not meet our criteria for advancement. Accordingly, today we are announcing the decision to pursue options to divest Roktavian and remove it from our portfolio as we focus on the business unit aligned with our strategic priorities. Roktavian is an innovative gene therapy that holds potential within the treatment landscape for severe haemophilia A. As a result, we are working to find an alternative that will allow for Roktavian to reach its full potential by ensuring access to those interested in gene therapy treatment. In the meantime, Roptavian will be commercially available in the United States, Italy, and Germany. Throughout this process, we will support and monitor patients who have received Roptavian as their well-being is our top priority. As we look to the future, we are pleased that our breakthrough medicines are in high demand and reaching thousands of patients around the world. Our financial performance so far this year reflects strategic investments in the enzyme therapies and skeletal conditions business units, both of which remain central to our growth strategy, along with increased business development opportunities and our own advancing internal pipeline. Building on this momentum, we look forward to the many data readouts and potential new approvals ahead, along with new business development opportunities as we focus on the next stage of biomass growth. With that, I will now turn it over to Brian.

Thank you, Alexander. Please refer to today's press release for detailed third quarter 2025 results, including reconciliations of GAAP to non-GAAP financial measures. All 2025 results will be available in our upcoming Form 10-Q, which we expect to file in the coming days. Starting on slide seven, Strong global demand across our portfolio of innovative medicines drove a year-to-date total revenue increase of 11% compared to the same period in 2024. Revenues from Voxogo and Palantik each increased by more than 20% on a year-to-date basis. As we shared last quarter, we anticipate Voxogo revenue in Q4 to reach its highest level of the year due to the timing of contracted orders, as well as increasing numbers of patients on therapy. We expect full year 2025 Voxogo revenue of between $900 million and $935 million. We are pleased with 8% year-to-date growth of the enzyme therapies business unit led by Pal and Zeke. Due to large orders for Naglazyme and Vimizem in the second quarter, we note that enzyme therapies revenue was lower in Q3 quarter over quarter. And compared to Q3 2024, alzheimer's therapy revenue in the quarter was relatively flat, primarily due to a higher volume of Durazine quarter last year. Given the strong top-line performance so far this year and our expectations for the fourth quarter, we are raising the lower end of our full-year 2025 total revenue guidance to $3.15 billion, with the midpoint of the range representing double-digit year-over-year growth. Now moving to slide 8, Q3 2025 results include a charge of $221 million for acquired in-process research and development, or IPR&D, on a pre-tax basis related to the Innozyme Pharma acquisition completed on July 1st of this year. This acquisition-related expense represents an approximate impact of $1.10 on a per-share basis. the IPR&D charges significantly increased both GAAP and non-GAAP R&D expenses in the third quarter. And along with higher SG&A investments across our skeletal conditions and enzyme therapies business units resulted in lower year-over-year operating margin and diluted earnings per share, both on a GAAP and a non-GAAP basis. However, looking past the IPR&D charge, Biomarin's underlying strong revenue performance and operational efficiencies drove increased year-to-date GAAP and non-GAAP diluted earnings per share. Further, we recognized lower tax expense during the third quarter due to the timing impact of the Innozyme IPR&D charge on our quarterly estimated tax rate, as well as some tax benefits from the recently enacted tax law. Taking these dynamics into account, alongside our expectations for strong revenue growth and continued operational execution in Q4 2025. We are updating full-year 2025 non-GAAP operating margin guidance to between 26% and 27%, and non-GAAP diluted earnings per share guidance to between $3.50 and $3.60. Biomarin continues to generate robust operating cash flows. reaching $369 million in the third quarter, $728 million year-to-date, contributing to the company's total cash and investments balance of approximately $2 billion at the end of Q3. We expect this momentum to continue, both solidifying the sustainability of our profitability and cash flows and building incremental capital to invest in future growth through business development. Now moving to slide nine, and to summarize, we have updated our full year 2025 guidance across total revenues, non-GAAP operating margins, and non-GAAP diluted earnings per share, incorporating the impact of the Q3 IPR&D charges. This update reflects a net improvement in our expected financial performance, net of the IPR&D charge of about 15 cents per share for non-GAAP diluted earnings per share. We are executing on our business plan so far this year, and today's guidance updates reflect both our year-to-date performance and our confidence in strong top-line and profitability growth in the final quarter of 2025. Finally, we are sharing an update on our previously provided 2027 revenue outlook, given the high level of interest. Based on the many unknowns and variables impacting our revenue over the next two years, mostly the impact of VoxOgo potential competition. We recognize that there are a range of outcomes from which a single scenario cannot be predicted with enough certainty at this point in time. We have developed a number of scenarios and will share that the lower end of our range of estimates is in line with current 2027 top line consensus per fact set, excluding Roctavian. And on the higher end of the range of estimates, there are scenarios that reach $4 billion in total 2027 revenues, also excluding Roctavian. But again, given the many unknowns between now and then, we are not providing a specific estimate or more narrow range. Going forward, we will continue to execute on our strategy and monitor the most impactful variables. However, we do not plan to provide additional estimates of 2027 revenues and we plan to follow our usual process of providing full-year guidance at the beginning of each year with the usual quarterly updates. Thank you for your attention, and I will now turn the call over to Kristin for a commercial update. Kristin?