BIOMARIN PHARMACEUTICAL INC Q3 FY2024 earnings call

• Implemented new corporate strategy focused on innovation, growth, and value commitment, structuring the company into business units for skeletal conditions, enzyme therapies, and ROCTAVIAN.
• VOXZOGO showed 50% revenue growth in Q3, driven by new patient starts, expanded indication into infants, and evidence of benefits beyond height; confident in long-term growth due to global approvals, commercial capabilities, BMN 333 development, and IP defense.
• Enzyme Therapies portfolio had 27% Q3 growth, 13% YTD growth, with ALDURAZYME, NAGLAZYME, and PALYNZIQ contributing to growth.
• Record revenues, raised full-year 2024 guidance, improved profitability, and operating margin expansion.

Skeletal Conditions: VOXZOGO saw 50% revenue growth in Q3, reaching $190 million. This growth was driven by new patient starts, expanded indication into infants, and evidence of benefits beyond height. Enzyme Therapies: The portfolio had 27% revenue growth in Q3, with combined net product revenues of $509 million. ALDURAZYME contributed due to order timing, NAGLAZYME grew 21%, and PALYNZIQ had 15% growth in the U.S. ROCTAVIAN: Generated $7 million in revenue during the quarter with new patient starts in Italy and the U.S.

• Raised full-year 2024 revenue guidance to between $2.79 billion and $2.825 billion, midpoint ~16% Y/Y growth.
• Reaffirmed long-term outlook: ~$4 billion in total revenues by 2027, 40% non-GAAP operating margin by 2026, >$1.25 billion operating cash flow by 2027, mid-teen CAGR through 2034, and >$5 billion revenue opportunity in skeletal conditions.

• Regulatory risks related to actions of regulatory authorities.
• Competitive risks, especially in markets outside the U.S. for VOXZOGO.
• IP risks, including the need to vigorously defend intellectual property.
• Operational risks such as quarter-to-quarter fluctuations and timing impacts on revenues (e.g., ALDURAZYME).

Q: Philip Nadeau asked about the phase of development ideal for BioMarin in business development.

A: Alexander Hardy responded that BioMarin will focus on transactions less than $1.5 billion, leveraging its scientific footprint and early pipeline.

Q: Salveen Richter inquired about the development strategy for BMN 333 and IP strategy.

A: Greg Friberg discussed BMN 333 entering the clinic early 2025 and potential for increased pharmacodynamic effect, while Alexander Hardy stated confidence in defending intellectual property.

Q: Jessica Fye asked about COGS and margin impact and BMN 333 data timeline.

A: Brian Mueller explained COGS factors including ALDURAZYME timing and inventory reserves, and Greg Friberg noted BMN 333 single ascending dose study data expected in 2025.

Q: Paul Matteis asked about guidance reiteration and initial guidance conservatism.

A: Brian Mueller and Alexander Hardy explained that long-term guidance was reaffirmed after reworking assumptions to account for competition, not conservative.

Q: Eliana Merle asked about ERT business growth drivers.

A: Cristin Hubbard discussed PALYNZIQ growth in U.S. and initiatives like high-yield diagnostic efforts and geographic expansion in MPS CLN2 areas.

Q: Cory Kasimov asked about VOXZOGO patients at full adult height and DMD program data.

A: Greg Friberg stated tracking VOXZOGO patients' final adult height and DMD program data expected mid-2025.

Q: Kostas Biliouris asked about VOXZOGO regulatory transition and PALYNZIQ competitive positioning.

A: Greg Friberg discussed VOXZOGO's regulatory commitment and PALYNZIQ's position with multiple treatment options for patients.

Q: Joseph Schwartz asked about BMN 333 patent timeline and BMN 390 development.

A: Alexander Hardy and Greg Friberg discussed BMN 333 patent longevity and BMN 390 early development with IND filing target next year.

Q: Akash Tewari asked about VOXZOGO patient adds and IP aggression.

A: Cristin Hubbard discussed VOXZOGO patient growth and Alexander Hardy stated focus on IP defense without elaborating on specific actions.

Q: Christopher Raymond asked about BMN 333 vs TransCon CNP differences.

A: Greg Friberg discussed BMN 333's potential for higher free CNP exposure and better efficacy in preclinical models.

Q: Mohit Bansal asked about BMN 333 trial length and NAGLAZYME strategy.

A: Greg Friberg discussed potential acceleration of BMN 333 trial and Brian Mueller explained NAGLAZYME's growth due to durable enzyme therapy portfolio.

Q: Gena Wang asked about BMN 333 efficacy ceiling and dosing frequency.

A: Greg Friberg discussed BMN 333's potential for higher exposure and different PK curve shape compared to VOXZOGO