Amicus Therapeutics(FOLD)2025-02-19 19:04Financial Data and Key Metrics Changes - Total revenue for Amicus Therapeutics in 2024 reached 458 million, reflecting an 18% growth year-on-year, or 19% at constant exchange rates [11][25] - Non-GAAP net income for 2024 was 39 million in 2023 [65] Business Line Data and Key Metrics Changes - Galafold continued to show strong performance with over 2,700 patients living with Fabry disease on the therapy by the end of 2024 [10] - Pombiliti and Opfolda generated over 12 million in 2023 [40][20] - The company anticipates Galafold revenue growth of 10% to 15% in 2025, while Pombiliti and Opfolda are projected to grow by 65% to 85% at constant exchange rates [22][66] Market Data and Key Metrics Changes - The global market for Fabry disease has expanded, with the number of diagnosed patients increasing from 10,000 at launch to 18,000 by 2024 [12] - The treated market for Fabry disease has more than doubled to 12,000 patients globally [13] - In the Netherlands, approximately 125 patients with late-onset Pompe disease are expected to be treated, highlighting the market's significance [91] Company Strategy and Development Direction - Amicus aims to achieve total revenue growth of 17% to 24% in 2025, with a focus on expanding its portfolio in rare diseases [21][66] - The company is committed to advancing ongoing studies to broaden labels and strengthen scientific leadership in Fabry and Pompe diseases [22] - Amicus plans to leverage its global infrastructure to support the launch of Pombiliti and Opfolda in up to 10 new countries in 2025 [54] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in Amicus's position to achieve its vision of becoming a leading rare disease biotechnology company [9] - The company highlighted the importance of continued commercial execution and financial discipline to support long-term growth [72] - Management noted that the Fabry market is expected to grow robustly, driven by improved diagnosis and medical education [30] Other Important Information - The company achieved full-year non-GAAP profitability in 2024 and aims for positive GAAP net income in the second half of 2025 [21][68] - Amicus is focused on maintaining high compliance and adherence rates, exceeding 90% for its therapies [14][114] - The company is exploring opportunities for additional assets to build upon its existing portfolio [130] Q&A Session Summary Question: Insights on the switch patient population for Pom-Op - Management noted that physicians are excited about Pombiliti and Opfolda, but some prefer to wait one to two years before switching patients from first-generation therapies [76][80] Question: Details on the prevalence of Pompe disease in the Netherlands - Management indicated that the Netherlands has a higher diagnosed population for Pompe disease, with expectations to start dosing patients by the end of Q1 or early Q2 [84][87] Question: FX exposure and hedging strategies - Management clarified that they do not traditionally hedge FX exposure due to unpredictability, but noted that a 1% change in exchange rates could impact revenues by approximately $4 million [97][100] Question: Update on second source manufacturing for Pom-Op - Progress is being made on establishing a second manufacturing site in Ireland, with commercial product expected to enter the supply chain in Europe by the second half of 2025 [106][108] Question: Transition of EAP patients to commercial therapy - Approximately 30 patients on clinical studies are expected to transition to commercial treatments in the first half of 2025 [122][124] Question: Future growth strategy via R&D - Management highlighted plans to leverage existing infrastructure for future growth and explore early-stage discovery efforts in the Fabry and Pompe space [128][130]