BioMarin Pharmaceutical(BMRN) - 2025 Q3 - Earnings Call Transcript

Financial Data and Key Metrics Changes - BioMarin reported an 11% year-over-year increase in total revenue, with cash and investments reaching approximately $2 billion at the end of Q3 2025 [5][6][15] - The company raised its full-year 2025 total revenue guidance to a range of $3.15 billion, reflecting strong performance and operational efficiencies [12][15] - Non-GAAP diluted earnings per share guidance was updated to between $3.50 and $3.60, with an operating margin guidance of 26% to 27% [15][17] Business Line Data and Key Metrics Changes - The Enzyme Therapies business unit experienced 8% year-to-date growth, with Palynziq and VoxoGo each increasing by over 20% year-to-date [12][19] - VoxoGo revenue is expected to reach between $900 million and $935 million for the full year 2025, with a 24% increase year-to-date compared to 2024 [6][12][20] - The Skeletal Conditions business unit, driven by FOXOGO, is projected to generate over $900 million in revenue for 2025, representing a 25% growth at the midpoint of guidance [6][19] Market Data and Key Metrics Changes - Approximately 75% of year-to-date VoxoGo revenue was generated outside the U.S., indicating strong international demand [20][21] - VoxoGo is now available in 55 countries, with significant growth in patient numbers quarter-over-quarter [20][21] Company Strategy and Development Direction - BioMarin is focusing on strategic investments in the Enzyme Therapies and Skeletal Conditions business units while pursuing options to divest ROCTAVIAN [9][10] - The company aims to expand its global footprint and deepen penetration in existing markets, particularly for VoxoGo and upcoming indications [20][21] - Future growth is anticipated through new product launches and business development opportunities, with a focus on advancing internal pipelines [11][15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving record commercial results for the full year, despite challenges from potential competition [5][11] - The company is monitoring various market dynamics and has developed multiple scenarios for future revenue, particularly concerning VoxoGo competition [17][42] - Management emphasized the importance of maintaining value-creating activities while pursuing operational efficiencies [75] Other Important Information - A charge of $221 million for acquired in-process research and development related to the Enizyme Pharma acquisition impacted Q3 results [14] - The company is preparing for pivotal data readouts and potential new approvals in the coming quarters, particularly for hypochondroplasia and BMN 333 [36][37] Q&A Session Summary Question: Can you elaborate on the 2027 guidance and the changes since it was initially issued? - Management indicated that various factors, including potential VoxoGo competition and the acquisition of Inozyme, influenced the revised outlook, leading to a range of estimates rather than a specific forecast [39][42] Question: Why were VoxoGo sales down quarter-over-quarter? - Management noted that the decline was due to timing shifts in larger orders and reaffirmed the total revenue range for VoxoGo for the year [48][50] Question: What is the company's strategy for business development? - Management highlighted a focus on early-stage collaborations and Phase III assets, emphasizing that business development remains a high priority [52][54] Question: What are the expectations for the upcoming BMN 333 data? - Management stated that they are looking for significant increases in free CNP levels to demonstrate clinical superiority over VoxoGo [64][66] Question: Can you clarify the 2027 cash flow targets? - Management confirmed that the cash flow targets are tied to revenue scenarios and emphasized ongoing optimization initiatives [79][80]