Amicus Therapeutics (FOLD) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Key product performance and growth drivers

• Galafold sales reached $111 million last quarter, with annual sales guidance raised to 14%-18% growth.

• Galafold now holds about 60% global market share among treated amenable Fabry patients, with 80%-90% share in mature markets.

• Growth is fueled by newly diagnosed and previously untreated patients, especially in the U.S., U.K., and Europe.

• AI and machine learning are expected to further improve Fabry patient identification and market expansion.

• Pombiliti and Opfolda generated $16 million in Q2 revenue, targeting $62-67 million for the year.

Market dynamics and expansion strategies

• Fabry market growth is driven by increased diagnosis and higher rates of amenable mutations among late-onset patients.

• In Japan and other newer markets, market share is growing but lags mature regions; Japan is expected to become a top-three market.

• Pompe market growth is supported by newborn screening in the U.S. and targeted screening in neuromuscular clinics globally.

• Spain's early launch ahead of competitors led to faster uptake, while Germany and the U.S. are on track for 12%-15% adult market share, aiming for 25%-30% in two years.

• The company targets 30%-35% market share within three years of launch in each market, with potential to exceed 50% as standard of care adoption increases.

Operational improvements and financial discipline

• Time from prescription to infusion for Pompe therapies is improving, targeting 30-45 days by year-end.

• Growth is balanced between new and repeat prescriptions, with a 50% increase in prescribing physicians in Q2.

• Real-world efficacy and patient word-of-mouth are key drivers for physician and patient adoption.

• Achieved non-GAAP profitability in Q2, with tightened expense guidance and continued focus on cost control.

• Manufacturing transition from China to Ireland is on track, with commercial supply expected by late 2025 or early 2026; second-generation manufacturing aims to reduce costs by 25%-30% over 3-5 years.