BioMarin Pharmaceutical (BMRN) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Strategic and financial highlights

• Outlined revamped near- and long-term strategy focused on innovation, growth, and value commitment, with three key pillars: innovation, growth (notably enzyme therapies and Voxzogo), and value commitment.

• Introduced a fifth R&D asset selection criterion to emphasize commercial potential and competitive landscape.

• Established new business units, including a growth conditions unit for Voxzogo, aiming for over $5 billion in revenue from Voxzogo across multiple indications.

• Announced first-ever long-term financial guidance, targeting $4 billion revenue and 40% non-GAAP operating margin by 2027, with mid-teens CAGR through 2034.

• Plans to more than double operating margin by 2026 and generate over $1.25 billion in operating cash flow in 2027.

Revenue growth drivers and pipeline strategy

• Revenue growth anchored by enzyme therapies (targeting high single-digit growth) and Voxzogo (expecting >25% growth, mainly from achondroplasia through 2027).

• Long-term growth to be driven by Voxzogo indication expansion into larger patient populations and early-stage pipeline assets, with modest penetration translating to significant revenue.

• Long-term guidance is not dependent on business development, but future BD could be additive.

• Revenue guidance is non-risk adjusted; risk-adjusted long-term CAGR would be approximately 10%.

• Voxzogo expansion considered de-risked due to consistent mechanism of action and supportive proof-of-concept data.

Market execution and competitive landscape

• U.S. market for Voxzogo is underpenetrated but offers the largest opportunity; execution focus is on building prescriber base and treatment home.

• Plans to expand Voxzogo into 20 additional markets, with execution and reimbursement as key levers for upside.

• Long-term guidance includes competition assumptions; Voxzogo expected to retain and grow share due to early treatment, safety, efficacy, and broad age label.

• Recent European patent court decisions upheld long-acting CNP portfolio, strengthening competitive position.

• Lifecycle management includes developing both daily and long-acting CNP (BMN 333), with the latter targeting similar or better safety and efficacy and potential for weekly dosing.