BioMarin Pharmaceutical (BMRN) TD Cowen 46th Annual Health Care Conference summary

Strategic overview and business development

• Recent strategic refresh emphasized business development, leading to acquisitions of Inozyme and Amicus, with the latter expected to close in Q2 2024.

• Both acquisitions will integrate into the enzyme therapy business, expanding global reach to 80 countries.

• Business development remains a core strategy, with ongoing efforts to strengthen the pipeline and plans for further early-stage deals while deleveraging post-Amicus.

• The company is positioned as a leading rare disease biotech, aiming for continued value creation through active business development.

Financial guidance and growth outlook

• 2026 revenue guidance reflects a 3% headwind due to lower royalty and Kuvan revenues, and removal of ROCTAVIAN expectations.

• Enzyme therapies are projected to grow at 7% and skeletal conditions at 8% at the midpoint, despite competitive pressures.

• Operating margin is expected to reach 40% in 2026, supported by a completed $0.5 billion cost transformation program and disciplined expense management.

• Amicus acquisition is anticipated to be slightly dilutive in 2026 but accretive over the first 12 months, with substantial accretion from 2027 onward.

Key risks and performance drivers

• Risks to guidance include order timing variability, geopolitical and economic instability, and competitive dynamics in skeletal conditions.

• Outperformance could be driven by growth in the under-two population for Voxzogo and deeper penetration in ex-US markets, which represent 75% of Voxzogo revenue.