BioMarin Pharmaceutical (BMRN) investor relations material

Strategic direction and recent acquisitions

• Integration of Amicus added two significant growth drivers, strengthening long-term growth profile and cash flow.

• Recent acquisition of Alesta (ALE1) expands the pipeline, with ongoing plans for further deals to support growth.

• Amicus deal focused on top- and bottom-line value creation, with projected $1.2B peak for POMBILITI + OPFOLDA and $1.4B for GALAFOLD.

• $200M in non-GAAP expense savings expected from Amicus integration, mainly from G&A and R&D overlaps.

• Integration execution included rapid employee decisions, retaining 192 of 505 Amicus staff, mainly in commercial and medical affairs.

Financial outlook and synergy realization

• Most synergies from Amicus integration will be realized by 2027, with full-year impact in 2028.

• Deal expected to be accretive within the first year, with substantial margin and cash flow growth.

• Deleveraging target of less than 2.5x leverage pulled forward to mid-2025, restoring pre-Amicus acquisition firepower.

• By 2028, balance sheet capacity could reach approximately $6B, up from $4-5B.

• Ongoing ability to pursue acquisitions up to $5B as integration progresses.

Product performance and growth drivers

• GALAFOLD and POMBILITI + OPFOLDA targeted for $2.6B combined peak sales by mid-2030s, driven by diagnosis rate improvements and geographic expansion.

• VOXZOGO achieved 20% quarterly growth, with exclusive indication in infants and strong global presence in 55 countries.

• Settlement with Ascendis brings royalty revenue from TransCon, and pending hypochondroplasia approval could further boost VOXZOGO.

• PALYNZIQ adolescent label adds 10% patient potential, with strong uptake among motivated families.

• VIMIZIM expected to exceed $1B in revenue, maintaining 7% annual growth despite quarterly fluctuations.