Amicus Therapeutics, Inc. Q2 FY2025 earnings call

Management Statement and Operational Highlights

• Delivered 17th consecutive quarter of double-digit revenue growth in core Pompe and Fabry disease business.
• Galafold had 13% year-over-year patient growth and is on track for highest new patient starts in 2025. U.S. contributed significantly to growth with over 1,000 PRS since launch.
• Pombiliti and Opfolda had strongest quarter since launch, with growth in the U.S. and 5 new European launch countries. Launched in 11 countries in Q2, including Italy, Switzerland, etc., and recently approved in Japan.
• Built evidence for differentiation of Pombiliti and Opfolda in Pompe disease, including a publication in Muscle and Nerve demonstrating benefits for switching ERT-experienced patients.
• Strategic partnership with Dimerix for DMX-200, with the ACTION3 pivotal study on track for full enrollment by end of 2025.
• Reiterated confidence that Pombiliti and Opfolda will reach combined sales of $1 billion by end of 2028. Galafold has long runway for growth with unmet need in Fabry disease.
• Aim for GAAP profitability in the second half of 2025.

Segment Performance

• Galafold: Revenue reached $128.9 million in Q2, up 12% at constant exchange rates and 16% in reported terms. Contributed significantly to growth with the U.S. being a major driver. Global market share for treated Fabry patients with amenable mutations is over 69%. Underlying growth driven by new patient starts, with 13% year-over-year patient growth this quarter and on track for record new patient starts in 2025.
• Pombiliti and Opfolda: Q2 revenue was $25.8 million, up 58% at constant exchange rates. U.S. represented approximately 42% of revenue, ex-U.S. 58%. Strong commercial demand in the U.S. and 5 new European launch countries. Launched in 11 countries in Q2, including Italy, Switzerland, Portugal, etc., and recently approved in Japan with a label indicated for ages 15 and older.

Guidance

• Total revenue growth for 2025 is expected to be 15% to 22% at constant exchange rates.
• Galafold revenue growth is projected to be 10% to 15%.
• Pombiliti and Opfolda revenue growth is expected to be 50% to 65%.
• Gross margin is anticipated to be in the mid-80s.
• Non-GAAP operating expense guidance is $380 million to $400 million.
• Anticipate positive GAAP net income during the second half of 2025.

Risks

• Potential impact of tariffs and manufacturing supply chain challenges on production and costs.
• Litigation risks related to intellectual property, though confident in the strength of IP; majority of litigations typically settle.
• Uncertainties in regulatory approval timelines for DMX-200 and other pipeline assets, which could affect product launch and market entry.

Question and Answer

Q: About real-world evidence for switching from Nexviazyme to PomOp and differences between U.S. and OUS.

A: Jeff discussed that physicians look at biomarkers, muscle strength, 6-minute walk, FVC, and quality of life when switching; no major differences between U.S. and other geographies.

Q: Update on additional manufacturing facility for PomOp in Ireland.

A: Material to enter commercial supply chain in Europe back half of 2025 and U.S. next year; diverse supply chain in place to navigate challenges.

Q: Confidence in IP position and settlement with Aurobindo.

A: Highly confident in IP strength; majority of litigations settle, and settlement with Teva reinforces the case against remaining litigants.

Q: Timelines for new U.S. manufacturing process, impact on COGS, pipeline expansion, PomOp launch, and reimbursement.

A: Manufacturing strategy advanced with multiple sites in progress; reimbursement going well in all geographies; Nexviazyme switches continuing; focus on late-stage derisked assets for pipeline expansion.

Q: Ex-U.S. starts for Pombiliti and Opfolda and reasons for switching at 2 years.

A: Strong growth in ex-U.S. markets; switches due to decline, physician/patient initiative, and proactive switch as data supports continued improvement.

Q: FDA regulatory alignment on FSGS and Phase III data timeline.

A: FDA alignment on proteinuria as primary endpoint; Phase III study on track for full enrollment by end of 2025, top line data by end of 2027; clinical relevance through responder analysis on proteinuria thresholds.

Q: Phase III data timeline and clinical relevance for DMX-200.

A: Phase III study on track for last patient in by end of 2025, top line data end of 2027; clinically relevant through responder analysis on proteinuria thresholds to show improvement in FSGS patients.