Amicus Therapeutics, Inc. Q3 FY2024 earnings call

• Commercial Execution: Total revenue in Q3 was $142 million, a 37% year-over-year growth. The company raised full-year total revenue guidance to 30% to 32% from the previous 26% to 31%.
• Galafold Performance: Galafold continued strong performance with 19% growth in Q3. It holds over 60% of the global market share of treated Fabry patients with amenable mutations. The Fabry market is growing, with opportunities in diagnosing untreated patients and increasing market share.
• Pombiliti and Opfolda Launch: Pombiliti and Opfolda's global launch is progressing well, with $21 million in Q3 revenue. The company raised full-year sales guidance for these products to $69 million to $71 million. There is progress in reimbursement globally, with launches in multiple countries and ongoing discussions in additional European markets.
• Clinical and Regulatory: Submitted a Japan New Drug Application for Pombiliti and Opfolda. Ongoing clinical studies for pediatric Pompe disease populations to support label expansions.

Galafold: In Q3 2024, Galafold generated global revenue of $120 million, representing 19% growth year-over-year on a constant currency basis. Year-to-date, Galafold revenue grew 18% year-over-year at constant exchange rates, contributing significantly to the company's top line. Pombiliti and Opfolda: In Q3 2024, Pombiliti and Opfolda reported revenue of $21 million, a 33% increase from the second quarter. For the full year, the guidance for Pombiliti and Opfolda sales is raised to $69 million to $71 million.

• Total revenue guidance for full year 2024 is raised to 30% to 32% from 26% to 31%.
• Galafold full-year revenue growth guidance narrowed to 16% to 18% at constant exchange rates.
• Pombiliti and Opfolda sales guidance for the year is $69 million to $71 million.
• Non-GAAP operating expense guidance lowered to $340 million to $350 million from $345 million to $360 million.

• IP Litigation: Remaining IP litigation with other parties, though settlement with Teva is a milestone but still some risk remains.
• Regulatory and Reimbursement: Challenges in securing reimbursement and regulatory approvals in new markets for Pombiliti and Opfolda.
• Dependence on Execution: Continued need for strong commercial execution to maintain growth in both Galafold and Pombiliti/Opfolda.

Q: Asked about Pombiliti prescribing trends, breadth and depth of prescribers, patient switching experiences, and Japan market opportunity for Pompe.

A: Bradley Campbell and others discussed breadth/depth of prescribers, patient switching based on physician observations, and Japan having a smaller Pompe market with ~100 treated patients currently.

Q: Asked about previously untreated patients on Galafold in core vs emerging regions.

A: Bradley Campbell mentioned significant new patient finds in key markets like US, Europe, Japan, and ongoing switch opportunities in newer launch regions like Latin America, Southeast Asia, etc.

Q: Asked about remaining IP litigations for Galafold and dropout rates.

A: Bradley Campbell stated confidence in case against remaining litigants and compliance/adherence rates for Galafold have improved with lower discontinuation rates.

Q: Asked about pipeline focus, internal vs external development.

A: Bradley Campbell and Jeff Castelli discussed focus on later-stage products, internal R&D refining genetic constructs for Fabry/Pompe, and business development looking at specific modalities/stages.

Q: Asked about patient add trends in Q3 and 2025 expectations.

A: Bradley Campbell discussed Q3 revenue strength, patient treatment numbers, and ongoing reimbursement process impact on patient adds, with focus on 2025 launch sequence and reimbursement discussions.

Q: Asked about BD focus and pipeline constructs vs Caritas.

A: Bradley Campbell and Jeff Castelli talked about focusing on derisked products for top-line growth, internal constructs refined from prior work, and ongoing R&D efforts.

Q: Asked about Pombiliti Opfolda inventory and next gen manufacturing impact on gross margins.

A: Bradley Campbell and Simon Harford discussed previously expensed inventory impact in 2025 and next gen manufacturing expected to impact gross margins in back half of the decade.

Q: Asked about FDA feedback on genetic medicine for Fabry.

A: Bradley Campbell expressed excitement about potential regulatory flexibility but noted focus on Galafold for amenable patients and interest in addressing nonamenable population.

Q: Asked about competitive dynamics vs Nexviazyme in US and ex-US.

A: Sebastien Martel discussed switch dynamics in US reflecting Nexviazyme/Lumizyme shares and ex-US switch dynamics reflecting Myozyme/Nexviazyme shares, with naive patient uptake also seen.