BioMarin Pharmaceutical Inc.

Thank you for standing by and welcome to the Biomarin Pharmaceutical fourth quarter and full year 2023 conference call. I would now like to welcome Tracy McCarty, Head of Investor Relations, to begin the call. Tracy, over to you.

Thank you, Mandeep. Thank you everyone 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 program, 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 SEC, 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 on 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 Biomart's President and CEO, Alexander Hardy.

Thank you, Tracy, and good afternoon, everyone. Thank you all for joining us today. As I said in January and reiterate today, the opportunity at Biomarin to positively impact patients' lives through transformative therapies while delivering value-creating revenue growth and profitability to shareholders has never been more evident. Touching on today's financial results, total revenue grew 20% in the fourth quarter of 2023 compared to the fourth quarter of 2022. total revenue grew 15% for the full year of 2023 compared to the prior year. This is a very strong performance. On a constant currency basis, total revenue growth was 25% in the fourth quarter and 20% for the full year. Equally as important to Biomarin's growth story are bottom line results. Non-GAAP earnings per share increased 48% in the fourth quarter versus the fourth quarter of 2022, percent year over year. These 2023 results, along with our growth expectations as implied by our full 2024 financial guidance provided today, solidifies our position as a financially self-sustaining business that can grow revenue, expand operating margins, and accelerate earnings per share. Brian will provide more details on the financials in a moment, so I will now turn to update you on our progress on the priorities I outlined in January. The first, to accelerate and maximize the Voxergo opportunity, remains front and center. As demonstrated by the Voxergo financial results today, 178% growth year over year, with close to 300 new patients added in Q4. The launch in achondroplasia is on a path to blockbuster status. We were pleased that 70% of new U.S. prescriptions in Q4 were for children under the age of five following FDA's age expansion approval last October. The U.S. and EU approvals last quarter allowing treatment from infancy sets Voxergo up to be a major multi-year growth driver. Beyond having an expanded age label, Voxergo's profile benefits from more than 1,000 patient years of long-term safety and efficacy data beyond just height. We believe this substantial clinical track record will encourage families to pursue VoxOgo treatment as early and for as long as possible to enable maximum essential therapeutic benefit. As a result of the growing global demand for VoxOgo treatment and the scope of our extensive long-term clinical data, We're seeing an increase in the breadth of our prescriber base. Our work to build prescribing confidence and relationships with pediatric endocrinologists in the United States has been extremely well received. With recent Voxergo uptake for children with achondroplasia under five being twice as fast as launch uptake for children over five, our plan to drive earlier and longer intervention with a goal of greater therapeutic outcomes is on track. We plan to build on our established leadership in achondroplasia treatment to expand into multiple other statural conditions. In the fourth quarter, Biomarin began the enrollment in the six-month observational run-in portion of the pivotal program with Voxergo for the treatment of children with hypochondroplasia. And we are actively engaging global health authorities regarding development programs in idiopathic short stature, and multiple genetic short stature pathway conditions with plans to begin pivotal studies later this year. As Hank will discuss later, we believe that there is strong proof of concept and indications beyond achondroplasia. As we expand into these indications, we hope VoxOgo treatment will empower patients and families across a spectrum of structural conditions to live their lives to the fullest. For this reason, VoxOgo acceleration, achondroplasia and expansion other indications remains the top priority of Biomarin. The second priority is establishing the Roctavian opportunity. As I said in January, we believe 2024 and 2025 will inform Roctavian's uptake curve and long-term potential. We have been very pleased with the strong and positive payer response to the value proposition associated with Roctavian and how this is translated into published payer policies and lives covered. Furthermore, we continue to be confident with the clinical profile of the product, which is evidenced by the warranty agreement, which has been equally well received in the marketplace. We have also continued to make good progress in activating the global marketplace, including the recent publishing of the Italian Octavian price. We want to reiterate, however, that the complexity of aligning the required pre-infusion checklist will take time. As I outlined last month, for successful Octavian treatment, we need a motivated patient, a supportive payer, and a treatment site with a physician who's willing and able to use the product. For a pioneering new therapy, this isn't a surprise and is the reason why we intend to let the results do the talking for Octavian uptake. We do expect patients to be treated with Octavian in 2024, as implied by its inclusion in our 2024 total revenue guidance. In the meantime, we will continue our work to activate the global marketplace and look forward to reporting Roptavian revenues on a quarterly basis. The third priority is our focus on the most productive R&D assets, those with transformational benefits for patients and high commercial potential. I've been spending a lot of time with the R&D team to understand the unique profile of each pipeline asset currently under development. I've been impressed by the level of innovation and expertise in developing transformational therapies. Keeping with our ambitious financial goals, we intend to hold a very high bar in terms of discipline spend and prioritization of the most impactful medicines. To that end, we're undertaking a strategic portfolio review to determine which pipeline assets will advance and which will not. A complete update on prioritized R&D assets, those with the highest potential patient impact and highest potential value creation for shareholders, will be communicated at our investor day later in 2024. In the meantime, we have a number of promising candidates advancing, and Hank will provide an update on those in a moment. And lastly, our fourth priority is increasing profitability faster than originally planned. Our 2023 results and our four-year guidance for 2024 both demonstrate our transition to growing profitability and significant operating leverage. Our four-year 2024 guidance reflects double-digit revenue growth, regardless of Roptavian contributions, and non-GAAP earnings per share growing faster than revenues. Otsogo is expected to be a major driver of year-over-year growth, and is reflected in our streamlined guidance, total revenues guidance item, which includes all Biomarin commercial products. Non-GAAP operating margin is a new line item added to Biomarin's 2024 four-year guidance, primarily because it reflects our focus on leverage across the P&L and anticipated margin expansion this year, as well as providing you with a clear line of sight into our business performance. We believe these streamlined, full-year guidance items will allow you to track our financial progress as we strive to achieve the four strategic priorities I just described. So in summary, it's truly an exciting time at Biomarin, and I see tremendous opportunity to create value for patients and for shareholders. The entire leadership team is hard at work on shaping the future corporate strategy, which will include views on R&D and capital allocation, as well as setting and taking steps to achieve ambitious long-term financial targets. The entire organization is mobilized and approaching this work with a sense of urgency. We will be making significant progress that we will want to share externally on a timely basis. That could be incrementally and at the investor day for which we are evaluating the specific timing. Please stay tuned for additional updates. Thank you for your attention and I will now turn over the call to Hank provide an update on key R&D highlights.