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 significant global expansion and strong retention rates.

• Commercial strategy focuses on patient identification, therapy initiation, and adherence, leveraging genetic screening, AI, and digital campaigns across 80 countries.

• Amicus acquisition enables expansion into new geographies for GALAFOLD and Pompe, with plans to enter 10 more countries for GALAFOLD and 20+ for Pompe.

R&D and pipeline updates

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

• Pipeline expanded through internal innovation and external deals, including Alesta Therapeutics for HPP oral therapy and acquisition of BMN 820 for FSGS, with phase III readout expected in 2028.

• BMN 333, a next-generation CNP product, aims to demonstrate superiority over VOXZOGO in achondroplasia, with phase II 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.

• HPP oral therapy targets upstream disease mechanisms, aiming for broader tissue reach and commercial differentiation from existing therapies.

Competitive landscape and strategy

• Recent settlement with Ascendis provides retroactive royalties and clears competitive overhang, with 20% royalties in the U.S. and 18% in select international markets.

• VOXZOGO maintains a strong position with 90% patient retention and exclusive label for the zero to two age group for the next few years.

• International markets contribute 75% of VOXZOGO revenue, with country-specific strategies and longer timelines for competitive entry.

• BMN 333 phase III is powered to detect a 50% improvement in AGV, aiming for meaningful increases in growth and overall health outcomes.

• Oral competitors and growth hormone combinations are monitored, but safety, efficacy, and long-term data are emphasized as key differentiators.