Amicus Therapeutics, Inc. Q4 FY2024 earnings call

• Commercial Execution: Amicus achieved total revenue of $528 million in 2024, a 32% year-over-year growth. Galafold continued its strong performance with growing new patient starts. Pombiliti and Opfolda had a successful global launch with 220 patients living with late-onset Pompe disease treated or scheduled for treatment by end of 2024.
• Diagnosis Initiatives: Worked with OM1 on machine learning AI diagnostics for Fabry, piloting technology at Penn to identify undiagnosed Fabry patients. Collaborated with NHS in UK on a pilot program to address diagnostic disparities in rare diseases.
• Clinical Studies: Ongoing open label studies for Pombiliti and Opfolda in children with Pompe disease (ZIP study for late-onset and Rossella study for infantile onset). Strong presence at medical conferences like WORLDSymposium, highlighting work in Fabry and Pompe.

Galafold: In 2024, Galafold generated revenue of $458 million, representing 18% year-over-year growth (19% at constant exchange rates). It had over 2,700 patients using the product, with 65%+ global market share of treated Fabry patients with amenable mutations. Pombiliti and Opfolda: In 2024, revenue for these products was over $70 million globally. For 2025, the company projects 65% to 85% revenue growth at constant exchange rates for these products.

• 2025 Financial Guidance: Total revenue growth guidance of 17% to 24% at constant exchange rates. Galafold revenue guidance of 10% to 15% at constant exchange rates. Pombiliti and Opfolda revenue guidance of 65% to 85% at constant exchange rates. Non-GAAP operating expense guidance of $350 million to $370 million, and GAAP net income expected to be positive during the second half of 2025.

• Foreign Exchange Volatility: Exposure to currency fluctuations, with a 1 percentage point move in the dollar up or down potentially resulting in a ~$4 million revenue impact.
• Regulatory and Commercial Execution Risks: Delays in approvals, reimbursement, or launch of products in new markets, such as potential issues with tariff exposure or challenges in expanding into certain regions.

Q: About Pom-Op, how long are doctors keeping patients on first or second gen ERTs before moving to Amicus' products?

A: Bradley Campbell stated that physicians want to see a year or two of experience once patients switch to Pombiliti and Opfolda, but there are clear target opportunities with patients in decline phase. Up to 40% of patients who switched to Nexviazym in the US will enter the two-year experience mark.

Q: Can you elaborate on FX and hedging programs?

A: Simon Harford mentioned Amicus hasn't traditionally hedged FX exposure as they're not experts at predicting FX, but a 1 percentage point move in the dollar has a ~$4 million revenue impact. They don't predict future FX but highlight the exposure.

Q: Update on second source manufacturing for Pom-Op and tariff exposure?

A: Bradley Campbell said they're making great progress on second source manufacturing in Dundalk, Ireland with WuXi, on track for commercial product in Europe back half of 2025 and US next year. Tariff exposure is minimal with no material impact on financials currently.

Q: Regarding compliance and adherence with Pom-Op, are physicians seeing patients stay on longer?

A: Jeffrey Castelli noted that over 90% compliance and adherence with Pombiliti and Opfolda, data from trials shows sustained improvement over four years, and there's potential to publish more data on long-term effects.

Q: For EU countries coming online, how many EAP patients transition to commercial therapy?

A: Sebastien Martel said roughly 30 late-onset Pompe disease patients on clinical studies would transition to commercial therapy in the 10 new EU markets launching in 2025.

Q: Strategy for future growth via R&D?

A: Bradley Campbell mentioned leveraging the global commercial infrastructure and late-stage development infrastructure, focusing on early-stage discovery efforts in Fabry and Pompe spaces over the next 12-24 months to build out programs and maintain leadership.

Q: AI aspect with OM1, regulatory and logistical burden?

A: Bradley Campbell said it's driven by proving technology, relying on electronic health records, and partnership with physicians. It's a tool for Fabry physicians to prove diagnosis, with work in US and UK, and potential to deploy in other markets.

Q: Follow up on AI collaboration with OM1, outcomes?

A: Bradley Campbell stated they're in the process of identifying high-risk undiagnosed Fabry patients, physicians are reaching out for confirmatory testing, and they expect to share outcomes of the research in the back half of 2025.

Q: Competitive dynamic for Pom-Op, label expansion, and business development?

A: Bradley Campbell noted Sanofi's lesser presence at World Congress, Jeff Castelli updated on label expansion timelines for Pompe disease in different patient cohorts, and Sebastien Martel said Amicus is carefully looking at adjacent spaces and late-stage assets for business development, leveraging its infrastructure for growth.