BioMarin Pharmaceutical(BMRN) - 2024 Q4 - Earnings Call Transcript

Financial Performance and Key Metrics - Full year 2024 total revenues grew 18% to $2.85 billion, with fourth quarter revenues increasing 16% year-over-year to$ 747 million [16][9] - Non-GAAP operating margin expanded over 900 basis points, reaching 28.6% for the full year 2024, and 31.1% for the fourth quarter [21][10] - Non-GAAP diluted earnings per share increased 69% to $3.52 for the full year and 88% to$ 0.92 for the fourth quarter [22][10] - Operating cash flow for the full year 2024 was $573 million, a 260% increase over the previous year [22] Business Line Performance - VOXZOGO for achondroplasia grew 56% year-over-year, contributing significantly to revenue growth, with total revenue of$ 735 million for 2024 [11][30] - Enzyme therapy business totaled over $1.9 billion for the full year, a 12% increase compared to 2023, with consistent growth across marketed brands [17][10] - PALYNZIQ revenue growth was driven by substantial patient uptake in the U.S. and ongoing expansion in Japan [37] Market Performance - VOXZOGO's revenue split for 2024 was 24% from the U.S. and 76% from international markets, indicating strong global demand [32] - The company plans to expand VOXZOGO access to over 60 countries by 2027, focusing on early diagnosis and treatment [34] Company Strategy and Industry Competition - The company is prioritizing its most promising pipeline candidates and reorganizing its operating model around business units [8] - A$ 500 million cost transformation program is underway to enhance profitability and support long-term growth [8] - The company aims for double-digit revenue growth in 2025, with a target of $4 billion in total revenues by 2027 [23][25] Management Commentary on Operating Environment and Future Outlook - Management expressed confidence in achieving double-digit revenue growth in 2025, supported by a strong commercial portfolio and ongoing innovation [10][13] - The company anticipates that the majority of revenue growth in 2025 will occur in the second half of the year [24] - Management highlighted the importance of early diagnosis and treatment in driving future growth, particularly for VOXZOGO [33] Other Important Information - The company expects non-GAAP operating margin for 2025 to be between 32% and 33%, reflecting continued strong revenue growth and cost transformation initiatives [26] - Non-GAAP diluted earnings per share for 2025 is expected to be between$ 4.20 and $4.40, indicating a strong growth trajectory [28] Q&A Session Summary Question: Insights on VOXZOGO guidance and growth expectations - Management acknowledged that while VOXZOGO is expected to grow by$ 200 million year-over-year, the growth rate is decelerating as the revenue base increases [50][52] - The U.S. market is a significant focus, with efforts to expand the prescriber base and increase patient starts in the 0 to 5 age cohort [56][57] Question: Business development priorities - The company is focused on both pipeline and commercial assets, with a strong interest in genetically defined conditions [62][68] - Management highlighted the importance of their global commercialization capabilities in attracting potential partners [70] Question: Durability and safety of growth hormone and CNP combo - The company aims to develop a single agent CNP drug that delivers best-in-class growth while ensuring convenience and safety for patients [79] Question: DMD program and dystrophin levels - Management indicated that the 25-week data will provide a clearer indication of whether the target of 10% dystrophin levels is achievable [86][88] Question: Diagnostic harmonization and enzyme therapy growth - The company is actively working on harmonizing diagnostic protocols across geographies to enhance patient identification and treatment [90][93]