BioMarin Pharmaceutical Inc.

Non-GAAP diluted earnings per share increased 69%. This financial strength puts us on the path for double-digit revenue growth in 2025 and enables our ongoing reinvestment in further innovation and growth. Our confidence is supported by Biomarin's increasingly profitable commercial portfolio. Leadership across skeletal conditions is anchored to global expansion VOXOGO for achondroplasia, which grew 56% year-over-year, with plans to expand into hypochondroplasia in 2027, as well as four additional skeletal conditions should data be supportive. Strengthen from our enzyme therapies, growing at 9% in the fourth quarter year-over-year, and now approaching 2 billion annual revenues, gives us confidence in our long-term outlook. high single-digit CAGR from this business unit. The strategic prioritization of our pipeline last year resulted in the advancement of BMN351 for Duchenne muscular dystrophy and BMN333, a non-acting CNP, two candidates that may provide highly differentiated treatment options for the conditions they address. We look forward to early clinical data results later this year for both candidates. This year, we plan to share results from our phase three study with Palantzeek for adolescents. This potential age expansion represents an opportunity to provide 12 to 17 year olds significant and sustained fee response, as well as unrestricted diet, should data be supportive. In summary, we're excited by the progress we're making at Biomarin. The strategic and operational decisions made last year yielding tangible results and allowing us to make an even greater impact on our patients, our employees, and our shareholders. Looking ahead in 2025, we expect to share clinical data from three of our advancing programs, drive double-digit global revenue growth, execute on our business development strategy, continue our journey towards delivering the mid- and long-term financial outlook provided at Invest Today last year. I would like to thank our employees around the world for their contributions in 2024. I look forward to the progress we will make together in 2025. Thank you for your attention. I will now turn the call over to Brian to provide an overview of our financial highlights for the quarter.

Thank you, Alexander. Please refer to today's press release for detailed fourth quarter and full year 2024 results, including reconciliations of GAAP to non-GAAP financial initiatives. All 2024 results will be available in our upcoming Form 10-K, which we expect to file in the coming days. We are very pleased with Biomarin's execution in 2024, especially during a year of significant transformation. Full-year total revenues grew 18% to $2.85 billion and set the stage for record results in 2025. Fourth quarter 2024 revenues increased 16% year-over-year to $747 million. Double-digit increases from Voxogo were a strong contributor to growth in the fourth quarter and full year. Also contributing to record results in 2024, the enzyme therapy business totaled over $1.9 billion for the full year, a 12% increase versus the full year 2023, with consistent growth across BioMarin's marketed brands. Moving to expenses, for the full year 2024, non-GAAP R&D expense was slightly higher compared to the full year 2023, primarily due to spending on development of VoxOGO and five new indications offset by a reduction in spend for deprioritized programs. In the fourth quarter of 2024, non-GAAP R&D expense was $159 million and lower compared to the same quarter in 2023 as the impact of discontinued programs was fully realized during the fourth quarter. We expect that R&D expense will increase in 2025 as we ramp up our BMN 351, BMN 333, and VoxOGO indication expansion studies. For both the full year and fourth quarter of 2024, non-GAAP SG&A expense increased year over year, primarily due to a bad debt reserve, higher VoxOgo commercialization expenses, and costs associated with our ERP implementation, partially offset by lower Roctavian spending. Moving to profitability, we are pleased with the positive impact of Biomarin's strong revenue performance, coupled with the ongoing progress of our cost transformation program outlined last year. As a result of this momentum, Non-GAAP operating margin reached 28.6% for the full year 2024, an increase of 9.2 percentage points versus the full year 2023. Fourth quarter non-GAAP operating margin of 31.1% was boosted by the benefit from the cost transformation initiatives, lower R&D spend related to discontinued programs, and profitability leverage from our strong revenue growth. In addition to significant revenue growth in 2024, Biomarin achieved its 2024 objective of accelerating profitability growth at meaningful and sustainable levels. Our full year non-GAAP diluted earnings per share increased 69% to $3.52, and our Q4 non-GAAP diluted earnings per share increased 88% to $0.92. Biomarin's increasing profitability is also generating significant levels of operating cash flow, with $573 million of operating cash flow for the full year 2024, a 260% increase over the full year 2023, and further puts Biomarin on track to achieve our target of greater than $1.25 billion of operating cash flow in 2027. By Marin's ability to generate meaningful positive cash flows is a key enabler of our top capital allocation priority of investing in long-term revenue growth, both through our internal research and development investments and external innovation through our business development strategy. Turning to our 2025 outlook and full year 2025 total revenues, we expect between 3.1 billion and $3.2 billion of total Biomarin revenue, which represents 10% growth compared to 2024. In 2025, strong growth is expected from Voxogo, Imizin, Palantzeek, and Brineuro. And we expect the majority of year-over-year revenue growth in the second half of the year, as growth in the business in the first half of the year translates to an increased revenue base in the second half of the year. In 2024, the enzyme therapies business benefited from unusually high alderazine contributions in the third quarter. Similarly, naglazine demonstrated very strong growth and benefited from new patient additions as well as order timing. Due to these unique dynamics, we expect lower year-over-year growth rates for naglazine and alderazine in 2025. Importantly, Biomarin's 2024 revenue performance and the expected growth in 2025 keep us on track for achieving our target of $4 billion of total revenues in 2027. FOGO is expected to be a strong contributor to full-year 2025 total revenue growth, and we estimate it will contribute between $900 million and $950 million to 2025 total revenue. While we are not changing our overall guidance structure of only guiding the total revenue These directional insights are meant to provide a framework to align your expectations with ours. Moving to non-GAAP operating margin for the full year 2025, we are guiding to between 32% and 33%, which represents 3.9 percentage points of expansion at the midpoint versus the 2024 non-GAAP operating margin of 28.6%. Our guidance is supported by continued strong revenue growth along with the impact of the ongoing cost transformation initiatives. While noting that 2025 also represents an important investment year for critical activities in our prioritized pipeline and sales and marketing to enable our long-term growth profile. Based on the continued implementation of these activities in 2025, this is an important stepping stone towards the achievement of our target 40% non-GAAP operating margin next year. Finally, For non-GAAP diluted earnings per share, we expect between $4.20, $4.40 per share for the full year 2025, which at the midpoint represents a two-times top-line growth rate over 2024, driving towards another expected year of leveraged profitability growth for BioMarin. Building on our strong execution in 2024, we expect continued high performance as we benefit from Biomarin's revamped corporate strategy and operating model in 2025 and beyond. I will now pass the call to Kristen to discuss the drivers of our commercial performance. Kristen?

Thank you, Brian. The team delivered strong growth in 2024, led by the continued global expansion of Voxogo, increasing palenzyme penetration, and demand for enzyme replacement products, and we expect 2025 to build on this momentum. Record VoxOga results and 56% year-over-year growth for the full year were driven by strong demand globally with a significant number of new patient starts in infants and young children. In the United States, the expanding prescriber base and strong demand from families with children in the zero to five-year-old age cohort drove increased growth in 2024. Consistent with prior quarters, the majority of new patient starts in the U.S. were for infants and young children under five years of age. In international markets, we also saw significant VoxOgo year-over-year growth throughout 2024. In deeply penetrated markets, such as Germany, we focused on early diagnosis and treatment of the incident population. In markets with significant expansion potential, we are leveraging Biomarin's robust global footprint and commercial infrastructure to drive awareness and adoption in the eligible patient population. To provide insight into these global dynamics, Today, we are providing the percentage of actual total Vox Sogo revenues split between the U.S. and the combined contributions from outside the U.S., or OUS. For full year 2024, of Vox Sogo total revenue of $735 million, 24% was from the U.S., and 76% was from all countries outside of the U.S. For fourth quarter 2024, of Vox Sogo total revenue of $208 million, 26% was from the U.S., and 74% was from OUS. Now moving to expansion strategies in 2025. In the United States, we look forward to realizing the growth opportunity ahead and building on the momentum of our growing prescriber base and continued demand from families with infants and younger children based on VoxOgo's proven safety and efficacy profile. Outside of the US and in our larger strategic markets, such as Germany and Japan, we will continue to focus on early diagnosis and treatment of the incident population to drive treatment with Voxogo from infancy to provide maximum therapeutic benefit. Now turning to growth strategies in other international markets, we intend to more deeply penetrate countries where Voxogo is already available and include patient starts in new countries that are added this year. By 2027, we are focused on increasing Voxogo access to more than 60 countries. To facilitate these growth plans, we are investing in commercialization efforts including but not limited to increased field personnel to enhance the referral and prescriber basis, additional platforms to broaden reach, and the introduction of new initiatives to raise awareness and adoption of VoxOgo treatment. Further supporting our expansion efforts, we are very pleased to see two publications highlighting the importance of early VoxOgo treatment. Recently published international guidelines recommend early diagnosis, followed by early VoxOgo treatment. These guidelines were independently created to facilitate maximum clinical benefit for children with achondroplasia and to provide families confidence when choosing Voxogo treatment for infants and young children with achondroplasia. Also, as published in the December issue of MED, Voxogo is the only treatment for achondroplasia to have demonstrated a statistically significant improvement in proportionality versus an untreated control arm after three years of follow-up. We are confident that this will be another year of strong execution and record growth for Voxogo, the only approved treatment for infants and children with achondroplasia. At the same time, we are using our experience in achondroplasia to prepare for the 2027 launch of Voxogo for the treatment of hypochondroplasia should phase three data be supportive. The commercial and medical team are creating programs to raise awareness and increase the diagnosis of hypochondroplasia so we will be ready to provide VoxOgo to those interested in treatment upon potential approval. Now, moving over to our enzyme therapies, global demand drove strong results across all of Biomarin's marketed products. Double-digit Palenzyk revenue growth in the quarter was the result of substantial patient uptake in the U.S. and ongoing expansion in Japan. In addition, we saw significant year-over-year growth in Maglozyme, Simazem, and Brunera during the quarter, enabled by our ongoing efforts to find new patients and maintain treatment continuity for those on therapy. We continue to make progress in finding and starting new patients with our enzyme replacement therapies. For instance, we have seen considerable success in certain regions where we have expanded our gene panel testing programs to help diagnose patients with MPS and CLN2. For example, in Brazil, these initiatives have resulted in an increase in new diagnoses, We expect to roll out these programs into multiple countries over the coming quarters. Now, moving to Palenzec, with its highly differentiated profile and that it works across all PKU phenotypes, growth has been driven by new patient starts and reinitiation of treatment from adults with PKU. We are also excited that adolescents with PKU may soon have access to the only substitute enzyme therapy that can deliver normal fee and an unrestricted diet. With phase three data coming mid-year, we look forward to potentially submitting our applications in the U.S. and Europe in the second half of this year. In conclusion, I am very pleased with the team's execution in 2024 during a year of transformation across the organization. The transition to defined business units is resulting in higher focus, accountability, and performance, and we are already seeing the benefits of this operating model. We have set the stage for greater performance in 2025 and are on course to achieve our target of $4 billion of total revenues in 2027. Thank you for your attention, and I'll now turn the call over to Greg to provide an R&D update. Greg?