BioMarin Pharmaceutical (BMRN) investor relations material

Strategic and operational updates

• Significant changes to strategy, structure, pipeline, and portfolio since December 2023, resulting in strong execution and 16% revenue growth in the last quarter.

• Positive Phase 1 results for BMN 333, a long-acting CNP, led to advancement into a Phase 2/3 program.

• Completed and integrated a business development deal within one quarter, reflecting a new focus on external innovation.

• InnoSign acquisition secured a first-in-disease ENPP1 asset with peak sales potential of $500–600 million and first pivotal data expected in 2026.

• Ongoing comprehensive review of 2027 revenue guidance, with plans to provide more transparency at the product level.

Market and competitive landscape

• U.S. maintains leadership in genetically defined conditions, with China less active in this area but closely monitored for future competition.

• Geographic footprint in 80 countries is a key competitive advantage, with 75% of Voxzogo revenue generated outside the U.S.

• Voxzogo's growth driven by further U.S. penetration and expansion into new markets, aiming for presence in 60 countries by 2027.

• Competitive risk to Voxzogo considered limited due to early diagnosis, established presence, and unique infant indication.

Innovation and technology

• AI strategy follows a fast-follower approach until 2027, with ambitions for leadership in genetically defined conditions thereafter.

• AI leveraged across R&D, regulatory, manufacturing, and commercial functions, including patient identification and supply chain optimization.

• AI use in hypochondroplasia enabled faster study recruitment by clarifying genetic variants.