BioMarin Pharmaceutical (BMRN) Wells Fargo 21st Annual Healthcare Conference summary

Business performance and growth

• Metabolic conditions business achieved 9% CAGR over three years, reaching $2.1 billion in annual revenue, driven by established products and the integration of Amicus.

• GALAFOLD and Fabry expected to reach $1.4 billion, and POMBILITI and OPFOLDA in Pompe projected at $1.2 billion peak revenue in the mid to late 2030s.

• VOXZOGO saw 14% revenue growth and 20% patient growth year-over-year, with global expansion into 55 countries and strong retention rates.

• Settlement with Ascendis provides retroactive royalties and clears competitive overhang, supporting continued commercial momentum.

• International markets contribute 75% of VOXZOGO revenue, with country-specific strategies to defend and expand market share.

Pipeline and R&D updates

• Hypochondroplasia data for VOXZOGO exceeded expectations in efficacy and safety, with FDA review underway.

• BMN 333, a next-generation CNP product, is in phase II with plans for a phase III superiority trial versus VOXZOGO; results expected next year.

• BMN 351 for Duchenne muscular dystrophy will have higher dose data later this year, focusing on benefit-risk profile and functional outcomes.

• Oral HPP therapy acquired from Alesta targets upstream disease mechanisms, aiming for broader tissue impact and commercial differentiation.

• BMN 820 (formerly DMX-200) for FSGS is in phase III, targeting a differentiated mechanism with readout expected in 2028.

Commercial and strategic execution

• Growth driven by a three-pillar commercialization platform: finding patients, starting therapy, and ensuring adherence, leveraging genetic and AI-driven screening.

• Amicus acquisition expands reach for GALAFOLD and Pompe, with plans to enter 10 new countries for GALAFOLD and 20+ for Pompe.

• Guidance for VOXZOGO sales raised to $1 billion midpoint, factoring in competition, global growth, and favorable market access renegotiations.

• Settlement with Ascendis ensures immediate value capture and does not impact current commercial strategy.

• Ongoing focus on both organic and inorganic pipeline growth, with recent deals and continued business development activities.