BIOMARIN PHARMACEUTICAL INC Q1 FY2025 earnings call

• Strong financial performance with 15% revenue growth and non-GAAP earnings per share of $1.13, a 59% year-over-year increase. - Progress on strategic transformation, with focus on implementing changes while delivering growth. - Completed enrollment in VOXZOGO's pivotal Phase 3 study for hypochondroplasia, with potential launch in 2027. - PALYNZIQ on track for US and European submission in the second half of 2025 for adolescent treatment. - Early clinical results expected later in the year for BMN 351 and BMN 333. - Focus on business development to support growth agenda, with plans to do at least one deal this year.

In the first quarter of 2025, total revenues rose 15% to $745 million. VOXZOGO's global revenue reached $214 million, a 40% year-over-year increase. Revenue from the Enzyme Therapies business unit grew 8% year-over-year to $484 million, with PALYNZIQ contributing 22% growth compared to Q1 2024. VOXZOGO accounted for a significant portion of the revenue growth, and the Enzyme Therapies unit also showed strong performance.

• Reaffirmed full year non-GAAP operating margin guidance of 32% to 33%. - Non-GAAP earnings per share guidance reaffirmed, with earnings not expected to be smooth quarter-to-quarter but accumulating to double-digit growth for the full year. - Anticipated higher revenues in the second half of 2025 compared to the first half due to order timing dynamics, with VOXZOGO full year revenues expected between $900 million and $950 million (midpoint 26% growth).

• Potential pharmaceutical tariffs: Analyzing exposure and mitigation tactics, with limited exposure to US tariffs for China, Mexico, and Canada, and global revenue base (two-thirds outside US) providing some insulation. However, impact of future tariffs not reflected in current guidance.

Q: On VOXZOGO, whether there could be a decline in Q2 revenue given revenue trends and H2 being larger than H1?

A: Brian Mueller stated that VOXZOGO revenue trends show order dynamics with global markets, leading to revenue appearing relatively similar in Q2 but expecting stronger growth in H2.

Q: How could financials be impacted by US imposing 25% tariff on EU pharmaceuticals and EU retaliating with 25% tariff on US?

A: Brian Mueller said BioMarin has immaterial exposure to China, Mexico, and Canada tariffs, with one-third of sales in US and two-thirds outside US providing insulation. They are analyzing scenarios but not speculating on details at this time.

Q: Update on business development interest and timing, and detail of BMN 333 data before full presentation next year?

A: Alexander Hardy said BioMarin is excited about business development, focusing on aligned opportunities, with plans to do at least one deal this year. Greg Friberg mentioned BMN 333 Phase 1 study in healthy volunteers, with top-line update expected later in the year and detailed data in first half of next year.

Q: Strategies to drive VOXZOGO adoption, especially in US?

A: Cristin Hubbard said strategies include expanding prescriber base, investing in initiatives to drive awareness, increasing field personnel, and leveraging business unit structure for focused execution.

Q: Safety signals in BMN 351 mouse models and ability to dose chronically?

A: Greg Friberg said class effects for ASO categories are observed, watching toxicities closely, and data from six milligram per kilogram cohort in second half will include safety data.

Q: Addressing market perception of VOXZOGO's long-term value beyond 2030?

A: Cristin Hubbard and Greg Friberg mentioned start and stay treatment paradigm, growing body of evidence beyond height, life cycle management, and development of BMN 333 as a next-generation agent.

Q: VOXZOGO net price change and 333 pivotal trial design?

A: Brian Mueller said no pricing dynamics observed for VOXZOGO. Greg Friberg said 333 pivotal trial is a comparative effectiveness study vs VOXZOGO, looking for superiority.

Q: VOXZOGO US vs ex-US sales split and manufacturing sites?

A: Cristin Hubbard said ex-US revenue is approximately 75% of VOXZOGO revenues, expected to fluctuate but mirror portfolio split. Brian Mueller said VOXZOGO drug substance is sourced from US.

Q: Defining BMN 333 top-line data for registration trial, competitor CNP plus growth hormone studies, and BMN 351 muscle dystrophin data goals?

A: Greg Friberg said 333 study will have PK data unlocking next steps, growth hormone combination has challenges, BMN 351 muscle dystrophin data at 26 weeks will include safety and PK data, with 10% dystrophin level a key goal but other parameters also considered.

Q: BMN 333 pivotal program plan, approval bar, and ex-US plans?

A: Greg Friberg said 2026 pivotal study is a combined Phase 2/3 study looking for superiority vs controller, and working with regulators globally on ex-US plans.