BioMarin Pharmaceutical (BMRN) UBS Global Healthcare Conference 2024 summary

Strategic review and organizational changes

• Completed a comprehensive strategic review, leading to a new long-term growth strategy and improved profitability guidance.

• Established new business units focused on enzyme therapies, skeletal conditions, and gene therapy to enhance operational effectiveness.

• Added commercial competitiveness as a fifth core R&D pillar, resulting in the termination of five programs and prioritization of three.

• Significant management changes, including new Chief Commercial Officer, Chief Business Officer, and Head of R&D.

• Now targeting non-GAAP operating margins of 40% by 2026 and $4 billion in revenue by 2027, with a mid-teen CAGR through 2034.

Enzyme replacement therapy (ERT) business outlook

• ERT business expected to grow at mid- to high single digits, supported by chronic dosing, high barriers to competition, and ongoing patient identification.

• Focused investments in high-yield diagnostics and patient support programs to find new patients and improve adherence.

• Expanding into select new markets, leveraging a commercial footprint in 80 countries.

• Palynziq growth driven by targeting adolescent PKU patients, with a trial in 12–17-year-olds expected to yield results next year.

Voxogo and skeletal conditions franchise

• Voxogo projected as a $5 billion+ opportunity over the next decade, with six potential indications including achondroplasia and hypochondroplasia.

• Achondroplasia label expansion in the U.S. to ages 0–5 has driven new patient starts; further growth expected from increased awareness and earlier treatment.

• Geographic expansion ongoing, with launches in 44 countries and plans for 20+ more by 2027.

• BMN 333, a long-acting CNP, prioritized as a successor to Voxogo, with clinical trials starting early next year.

• Confident in defending intellectual property and maintaining leadership despite competition.