• Revenue Growth: 15% increase in total revenues for Q1 2025, reaching $745 million.
• VOXZOGO Revenue: $214 million, a 40% year-over-year increase.
• Enzyme Therapies Revenue: $484 million, an 8% year-over-year increase.
• PALYNZIQ Revenue Growth: 22% increase compared to Q1 2024.
• Non-GAAP Earnings Per Share: $1.13, a 59% year-over-year increase.
• Non-GAAP Operating Margin: 35.7% for Q1 2025, expanding by 11.9 percentage points year-over-year.
• Non-GAAP R&D Expense: $147 million, lower than the same quarter in 2024.
• Non-GAAP SG&A Expense: $183 million, decreased year-over-year.
• Operating Cash Flow: $174 million, a 271% increase over Q1 2024.

• Warning! GuruFocus has detected 2 Warning Sign with TRUP.

Release Date: May 01, 2025

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• Biomarin Pharmaceutical Inc (NASDAQ:BMRN) reported a strong financial performance with a 15% revenue growth in the first quarter of 2025.
• Non-GAAP earnings per share increased by 59% year-over-year, highlighting significant profitability expansion.
• VOXZOGO's global revenue reached $214 million, marking a 40% increase year-over-year.
• The company completed enrollment in its pivotal Phase III study with VOXZOGO in children with hypochondroplasia, aiming for a potential launch in 2027.
• Biomarin Pharmaceutical Inc (NASDAQ:BMRN) has a strong global presence, with approximately 2/3 of its total revenues originating from outside the United States, providing insulation from potential US tariffs.

Negative Points

• There is uncertainty regarding potential pharmaceutical tariffs, which could impact Biomarin Pharmaceutical Inc (NASDAQ:BMRN)'s financials if imposed.
• VOXZOGO's revenue growth in the first half of 2025 is expected to be lower due to global order dynamics, with more substantial growth anticipated in the second half.
• Non-GAAP SG&A expenses are expected to increase throughout 2025 as the company invests in strategic priorities, potentially impacting operating margins.
• The company faces long-term competition concerns for VOXZOGO, particularly beyond 2030.
• There are challenges in correlating VOXZOGO's PK data with growth outcomes, which could impact the development of BMN 333.

Q & A Highlights

Q: Can you clarify the expected revenue trends for VOXZOGO in the second quarter and the impact of potential US tariffs on EU pharmaceuticals? A: Brian Mueller, CFO, explained that VOXZOGO's revenue growth is expected to be higher in the second half of the year due to global order dynamics. Regarding tariffs, BioMarin is modeling various scenarios and potential mitigation strategies, but specific impacts will be discussed once more clarity is available.

Q: What is the company's interest and timing for business development (BD) activities, and what details can you share about BMN 333 data? A: Alexander Hardy, CEO, stated that BioMarin is methodically pursuing BD opportunities aligned with their strengths and aims to complete at least one deal this year. Gregory Friberg, Chief R&D Officer, mentioned that BMN 333 Phase I data will be shared later this year, with more detailed data expected in the first half of next year.

Q: What strategies are being implemented to drive further adoption of VOXZOGO among patients with achondroplasia? A: Cristin Hubbard, Chief Commercial Officer, highlighted initiatives targeting new patient uptake, including increasing field personnel and expanding the prescriber base, particularly among pediatric endocrinologists. The focus is on driving awareness and adoption across all age groups.

Q: How is BioMarin addressing market concerns about long-term competition for VOXZOGO? A: Cristin Hubbard emphasized the focus on a start-and-stay treatment paradigm, supported by a growing body of evidence on VOXZOGO's benefits beyond height. Gregory Friberg added that ongoing R&D efforts aim to leverage long-term data and explore new indications.

Q: Can you provide insights into the potential approval pathway for BMN 333 and its global plans? A: Gregory Friberg explained that the pivotal program for BMN 333 will include a comparative effectiveness study versus VOXZOGO, aiming for superior efficacy. BioMarin is working with global regulators to define the approval pathway.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.