Financial Data and Key Metrics Changes - BioMarin reported a revenue growth of 15% in Q1 2025, totaling $745 million compared to the same period last year [12] - Non-GAAP earnings per share reached $1.13, representing a 59% year-over-year increase, indicating significant profitability expansion [5][19] - Non-GAAP operating margin expanded to 35.7%, an increase of 11.9 percentage points year-over-year [15] Business Line Data and Key Metrics Changes - Voxzogo's global revenue reached $214 million, a 40% increase year-over-year, continuing its strong growth trajectory since approval [13] - Revenue from the Enzyme Therapies business unit grew 8% year-over-year to $484 million, with Palynziq contributing a 22% increase compared to Q1 2024 [13][22] - Full-year revenue expectations for Voxzogo are between $900 million and $950 million, representing a 26% growth at the midpoint [14] Market Data and Key Metrics Changes - Approximately two-thirds of BioMarin's total revenues originate from outside the United States, providing insulation from U.S. macroeconomic conditions [7][9] - The company is analyzing potential exposure to pharmaceutical tariffs and believes its global revenue base offers protection against such impacts [8][9] Company Strategy and Development Direction - BioMarin is focused on implementing changes to its strategy and operating model announced in September 2024, aiming for record full-year performance in 2025 [6][11] - The company is advancing its innovation strategy, with pivotal studies for Voxzogo and plans for new product applications in the second half of 2025 [10][24] - BioMarin aims to leverage its established global capabilities to support growth and innovation, particularly in rare disease treatments [11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in BioMarin's ability to navigate market uncertainties due to its focus on rare conditions and strong cash generation [6][7] - The company anticipates continued strong performance and innovation in the coming quarters, supported by its strategic transformation [11][19] Other Important Information - Non-GAAP R&D expenses were $147 million, lower than the same quarter in 2024, reflecting a reprioritization of R&D efforts [14] - Non-GAAP SG&A expenses decreased to $183 million due to cost transformation initiatives [15] Q&A Session Summary Question: Will Q2 revenue for Voxzogo decline? - Management indicated that while Q1 showed growth, Q2 revenue may appear flat due to global order dynamics, with expectations for stronger growth in the second half of the year [35][36] Question: How could potential U.S. tariffs impact financials? - Management stated that current guidance includes modest impacts from existing tariffs, but they are modeling various scenarios for potential future tariffs [36][37] Question: What strategies are being implemented to drive further adoption of Voxzogo? - The company is focusing on increasing awareness and expanding the prescriber base, particularly among pediatric endocrinologists, to drive adoption across all age groups [55][56] Question: What is the timeline for BMN 333 and its potential indications? - BMN 333 is in a Phase I study, with plans for pivotal studies in 2027, and the company is evaluating its future role in additional indications [84] Question: How have U.S. versus ex-U.S. sales evolved for Voxzogo? - Approximately 75% of Voxzogo revenues come from outside the U.S., and this split is expected to fluctuate as the brand matures [88]

Financial Data and Key Metrics Changes - BioMarin reported a revenue growth of 15% in Q1 2025, totaling $745 million compared to the same period last year [14] - Non-GAAP earnings per share reached $1.13, representing a 59% year-over-year increase, indicating significant profitability expansion [6][20] - Non-GAAP operating margin expanded to 35.7%, an increase of 11.9 percentage points year over year, driven by strong revenue performance and a reset in spending [18][20] - Positive operating cash flow was $174 million, a 271% increase over Q1 2024, supporting future growth investments [22] Business Line Data and Key Metrics Changes - VOXZOGO's global revenue reached $214 million, a 40% increase year over year, continuing its strong growth trajectory since approval [15] - Revenue from the Enzyme Therapies business unit grew 8% year over year to $484 million, with Palynziq contributing a 22% increase compared to Q1 2024 [15][27] - The company expects VOXZOGO full-year revenues to be between $900 million and $950 million, representing a 26% growth at the midpoint [16] Market Data and Key Metrics Changes - Approximately two-thirds of BioMarin's total revenues originate from outside the United States, providing insulation from U.S. macroeconomic conditions [9][10] - The company is focused on expanding access to VOXZOGO in more than 60 countries by 2027, with current access in 49 countries [24][26] Company Strategy and Development Direction - BioMarin is implementing a strategic transformation aimed at enhancing growth and profitability, with a focus on innovation and expansion [8][13] - The company is making progress on its innovation strategy, including pivotal studies for new products like Voxogo and Palynziq [12][28] - BioMarin is exploring business development opportunities aligned with its strengths in genetically defined conditions and clinical stage assets [45] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in navigating uncertainties in the pharmaceutical sector, citing the company's strong fundamentals and global revenue base [9][10] - The company anticipates continued strong performance and cash generation, supporting its growth agenda for 2025 and beyond [22][13] Other Important Information - BioMarin's R&D expenses were $147 million in Q1 2025, lower than the same quarter in 2024, reflecting a reprioritization strategy [17] - SG&A expenses decreased to $183 million year over year, attributed to cost transformation initiatives [18] Q&A Session Summary Question: Will Q2 revenue for VOXZOGO decline? - Management indicated that while revenue growth is expected to be stronger in the second half of the year, Q2 may show a slight decline due to global order dynamics [36][37] Question: How could U.S. tariffs impact financials? - Management stated that current guidance includes modest impacts from existing tariffs, but they are evaluating potential future scenarios and mitigation strategies [39][40] Question: What strategies are being implemented to drive further adoption of VOXZOGO? - The company is focusing on increasing awareness and expanding the prescriber base, particularly among pediatric endocrinologists, to drive adoption across all age groups [55][56] Question: What is the timeline for BMN 333 and its potential indications? - Management anticipates data from ongoing studies in 2027, with plans for pivotal studies based on the outcomes of BMN 333 [84] Question: How does the U.S. versus ex-U.S. sales for VOXZOGO compare? - Approximately 75% of VOXZOGO revenues come from outside the U.S., with expectations for this split to fluctuate as the brand matures [88]

First Quarter 2025 Earnings May 1, 2025 Table of Contents 2 ● Business and Financial Highlights ● First Quarter 2025 Key Financial Metrics ● 2025 Guidance ● BMN 351 Model ● Non-GAAP Reconciliations Forward-Looking Statements This non-confidential presentation and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expe ...