BIOMARIN PHARMACEUTICAL INC Q4 FY2024 earnings call

• BioMarin's new strategic vision is being realized, with initiatives like prioritizing pipeline candidates, reorganizing the operating model, and realizing the $500 million cost transformation program.
• 2024 was a year of record growth and profitability with top line up 18%, non-GAAP operating margin expanding, and non-GAAP diluted earnings per share increasing.
• VOXZOGO grew 56% year-over-year in 2024 with global expansion, and enzyme therapies grew 9% in the fourth quarter and approached $2 billion annual revenues.
• Pipeline advancements include BMN 351 for Duchenne muscular dystrophy, BMN 333, and VOXZOGO indication expansion studies.
• Commercial performance: VOXZOGO had strong global growth, enzyme therapies saw double-digit revenue growth in PALYNZIQ and other products.

In 2024, VOXZOGO had total revenue of $735 million, with 24% from the U.S. and 76% from outside the U.S. The enzyme therapy business had full-year 2024 revenue over $1.9 billion, a 12% increase compared to 2023. Full year 2024 top line grew 18%, non-GAAP operating margin expanded over 900 basis points, and non-GAAP diluted earnings per share increased 69%.

• 2025 total revenue expected between $3.1 billion and $3.2 billion (10% growth compared to 2024).
• Non-GAAP operating margin guided to between 32% and 33%.
• Non-GAAP diluted earnings per share expected between $4.20 and $4.40 per share.

• Risks related to regulatory authorities' actions, progress of product programs, availability of capital, and competitive developments.

Q: Can you talk more on where VOXZOGO growth is expected to come from, including territories and reimbursement?

A: Brian Mueller and Cristin Hubbard discussed that VOXZOGO growth is from various geographies like the U.S., Germany, Brazil, etc., with focus on new patient starts in younger age cohorts, expanding prescriber base, and leveraging global footprint.

Q: What's the appetite for BioMarin in business development, particularly regarding pipeline assets?

A: Alexander Hardy said BioMarin is excited about business development, looking at a range of stages including preclinical and clinical assets to strengthen growth outlook.

Q: Thoughts on the durability and safety of the growth hormone and CNP combo with BMN 333?

A: Greg Friberg discussed the PK study for BMN 333 and the goal of developing a single agent therapy for achondroplasia that is convenient and efficacious.