BioMarin Pharmaceutical (BMRN) Canaccord Genuity's 46th Annual Growth Conference summary

Company overview and strategic direction

• Focused on rare, genetically defined diseases with nine approved therapies and six first-in-disease medicines.

• Operates globally in over 80 countries with in-house manufacturing and end-to-end R&D and commercial capabilities.

• Achieved nearly $1 billion in Q2 revenue, representing 20% year-over-year growth, and raised full-year revenue and EPS guidance.

• Recent transformation improved profitability and cash flow, supporting reinvestment in growth.

• Strategy centers on a virtuous cycle of growth and reinvestment in both internal innovation and external business development.

Amicus acquisition and integration

• Closed acquisition of Amicus Therapeutics, adding Galafold (Fabry disease) and Pombiliti/Opfolda (Pompe disease) to the portfolio.

• Peak revenue targets: $1.4 billion for Galafold and $1.2 billion for Pombiliti/Opfolda by the mid to late 2030s.

• Expect to realize 50% cost synergies from Amicus, mainly in G&A, while preserving and investing in sales and marketing.

• Accelerated de-leveraging target to less than 2.5x leverage by mid-2025, a year ahead of initial plan.

• Amicus business expected to reach 60% operating margin by 2030, enhancing overall profitability.

Growth drivers and commercial execution

• Global expansion of Galafold and Pombiliti is a key growth lever, leveraging broader BioMarin infrastructure.

• Plans to improve Fabry diagnosis through AI-enabled patient identification, newborn screening, and family genetic screening.

• For Pompe, strategy focuses on switching patients from existing therapies by highlighting real-world evidence and expanding geographically.

• Strong advocacy and regulatory partnerships support market development and patient access.