BioMarin Pharmaceutical Inc.

pharmaceutical first quarter 2024 conference call. Today's conference is being recorded. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press the star key followed by the number one on your telephone keypad. If you would like to withdraw your question, press star one again. At this time, I'd like to turn the conference over to Tracy McCarthy, Group Vice President, Investor Relations. Please go ahead.

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. In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today. These non-GAAP measures should not be considered in isolation from, as substitutes for, or superior to financial measures prepared in accordance with U.S. GAAP, and you can find the related reconciliations to U.S. GAAP in the earnings release and earnings presentation. both of which are available in the investor relations section of our website. On the call from Biomarin Management today are Alexander Hardy, President and Chief Executive Officer, Hank Fuchs, President of Worldwide R&D, and Brian Mueller, Executive Vice President, Chief Financial Officer. Jeff Ager, Executive Vice President, Chief Commercial Officer, and Greg Geyer, Executive Vice President, Chief Technical Officer, are here with us to answer questions during the Q&A portion of the call. I will now turn the call over to Biomarin's President and CEO, Alexander Hardy.

Thank you, Tracy, and good afternoon, everyone. Thank you all for joining us today. In addition to strong financial results, we made significant progress in the quarter, developing the components of Biomarin's new corporate vision and strategy, all in the interests of positively impacting patients' lives while creating value for shareholders. and with a number of strategic initiatives ongoing to finalize these components, to be communicated and invested today, which we have now set for September the 4th. We are pleased to share the first chapter update today, the results from our strategic assessment of Biomarin's R&D portfolio. As innovation has fueled Biomarin's success to date, we undertook a prioritization of our overall portfolio from early to lifecycle stage assets. The goal of this assessment was to accelerate the delivery of those assets which add the greatest value to all of our stakeholders and align with the timelines for our strategic growth plans. We added a commercial lens early in the portfolio peer review process to ensure that return on investment, resource allocation, patient impact were all thoroughly considered. As a result, we chose to accelerate three assets that we believe offer the most transformative potential for patients and value creation for shareholders. We are also discontinuing four programs that did not meet our new higher bar for continued development. Moving briefly to progress made in the quarter on our four strategic priorities outlined in January. Beginning with our opportunity to accelerate and maximize VoxOga, we made significant progress during the quarter. With 74% revenue growth year over year and more than 500 additional children receiving therapy in the first quarter, VoxOga in achondroplasia continues towards blockbuster status. The majority of new U.S. prescriptions in the quarter were for children under the age of five, an important trend since FDA's age expansion approval last quarter, albeit we continue to see expansion in the over-five population as well. Global access to Voxergo from infancy is having a significant impact on rapid uptake as families pursue maximum therapeutic benefit by starting treatment early. In the United States, we continue to observe an increase in the breadth and depth of our prescriber base as real-world experience drives confidence in VoxOgo's extensive safety and efficacy profile. Leveraging our established leadership in achondroplasia, we also made important strides in our plans to expand the multiple other growth-related conditions. Our pivotal program with VoxOgo for the treatment of children with hypochondroplasia will begin treatment study mid-year with a target of completing enrollment by the first half of 2025. Based on ongoing discussions with global health authorities on study protocols with VoxOgo, theopathic short stature, and multiple genetic short stature pathway conditions, we expect to begin enrollment into these programs later this year. The momentum we're seeing with VoxOgo in achondroplasia supports our belief that CNP can potentially unlock clinical benefit for children across a number of growth-related conditions for many years to come. Hank will share perspective on the opportunities ahead with VoxOgo based on strong proof of concept and indications beyond achondroplasia, as well as the potential for longer-acting formulations. The second priority, establishing the Roctavian opportunity, continues to be complex. With pricing and reimbursement established in the United States, Germany, and Italy, aligning the required steps leading to patient treatment continues to pose different challenges. For example, while the Octavian national German price was established and published in December, the sub-insurers inserted new barriers to reimbursement. In the U.S., the complexity of local site reimbursement contracting continues to be an obstacle. We remain confident in the clinical profile of Roktavian, and we're pleased with the Roktavian update at the TH-SNA meeting, showing durable hemostatic efficacy, improved quality of life, and no safety signals at four years. With respect to the additional Roktavian development programs, we are proceeding with Roktavian development in Japan and in the prior inhibitor population, and have paused other programs until we observe more meaningful Roktavian commercial uptake. We recognize the importance of allocating our resources to the highest value-creating opportunities, and with the current levels of Roktavian investments and continued challenges with commercial uptake, we plan to communicate our evaluation criteria for Roktavian in terms of its place in our portfolio and related timing for potential next steps at the investor day in September. The third priority is our focus on the most productive R&D assets. And as already mentioned, I'm pleased we have completed the initial chapter of that work. Beyond our refresh pipeline, centered around three key assets to be accelerated, we're in a strong position to leverage external innovation in conjunction with our internal capabilities to fortify our mid- and longer-term pipeline. At Investor Day, we will also share more about our innovation strategy and plans and how they fit into our capital allocation and internal external portfolio innovation strategies. Lastly, our fourth priority to increase profitability faster than originally planned. As demonstrated by our first quarter results, we're tracking well towards achieving this priority. As I complete my first quarter as CEO, I hope it's evident that we're taking decisive and thoughtful action to realize our priorities, all designed to align with our broader operational and cost transformation strategies to be shared at Investor Day. Our full-year 2024 guidance reflects double-digit revenue growth, non-GAAP operating margin expansion, and non-GAAP earnings per share growing faster than revenues. These four-year guidance items allow you to track our financial progress as we transform Biomarin's operating model to produce the best outcomes for the patients we serve, our employees, and our shareholders. So, in summary, we are making tangible progress across the enterprise to reshape Biomarin's corporate vision and strategy. This is an enormous body of work that remains in process. But as you can see from our first quarter updates, we're working with a sense of urgency, purpose, and we are making definitive progress. We're excited to continue this work over the coming months with the goal of sharing our vision for a successful future with you at Investor Day on September 4 in New York. Thank you for your attention. I will now turn the call over to Hank to provide an update on key R&D highlights.