AMICUS THERAPEUTICS, INC. Q4 FY2023 earnings call

Management Statement and Operational Highlights

• Galafold Performance: Strong global uptake with over 2,400 patients on Galafold at year-end 2023. 2023 was the strongest year for net new Galafold patients since 2019. Underlying market dynamics, including improved diagnosis and medical education, provide opportunities for continued growth.
• Pombiliti and Opfolda Launch: Global launch off to a great start in key markets. Teams made progress in Germany, UK, and US. Rate of new commercial patients increasing, with patients switching from competitor therapies and naive populations. Focus on expanding access and reimbursement through engagement with payers.
• Financials: Achieved non-GAAP profitability in Q4 2023 with $2.6 million. Full year 2023 revenue was $399 million, up 21% from 2022. Cash, cash equivalents, and marketable securities were $286 million as of Dec 31, 2023.

Segment Performance

• Galafold: In 2023, Galafold revenue was $388 million, representing an 18% year-over-year growth. It had over 2,400 patients on the drug at the end of 2023, capturing 60%-65% of the global market share of treated amenable patients. Growth was driven by patient demand, including net new patients and switches. The treated Fabry patient market has more than doubled since 2015, with over 11,000 treated patients and nearly 6,000 untreated patients.
• Pombiliti and Opfolda: Global commercial launch underway, with full year 2023 revenue of $11.6 million. As of early 2024, 120 patients were on treatment or scheduled, with the rate of new commercial patients increasing across key markets like Germany, UK, and US. Patients are switching from competitor therapies and naive populations, with strong patient and physician demand.

Guidance

• Galafold: Full year 2024 growth expected to be 11%-16% at constant exchange rates.
• Profitability: Anticipate full year 2024 non-GAAP profitability driven by over $0.5 billion in combined revenue and prudent expense management. Q1 2024 expected to be non-GAAP profitable similar to Q4 2023, with sequential growth.

Risks

• Forward-Looking Statements: Forward-looking statements are subject to risks and uncertainties, including those detailed in the Form 10-K. Impact from inaccurate assumptions, known or unknown risks, and potential deviations from plans.

Question and Answer

• Q: In the US with regard to pump pay, are you seeing switches from patients that are clearly progressing or patients who are stable but just not gaining a benefit from their current treatment? A: Yes, a little bit of both. Some patients eager to start regardless of status, others in declining phase hoping for different outcomes, as per label indication for patients not improving on current therapies.
• Q: How are you guys seeing clinicians in the field define the decliners and the two symposiums that world, both yours and your competitors, having spent a lot of time on defining decliners, defining stable. What do you see in your conversations during the conference on the tools that clinicians are using to define that in their own head? A: Clinicians use holistic assessment including quantitative endpoints (6 minute walk distance, forced vital capacity) and qualitative patient reports. About 50% stable, 25% clearly declining, 25% improving. Holistic approach varies by site/country.
• Q: If you can just remind us on the 15 that were new and not part of the expanded access or clinical trial. Did you guys ever break them down in terms of geographies? A: Those 15 new patients came roughly equally from each launch country (Germany, UK, US). In US, majority from Myozyme; outside US, proportionally from Lumizyme, Nexviazyme, and naive patients. Rate of new patient starts increasing.