Amicus Therapeutics(FOLD) - 2021 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - For the full year 2021, Galafold revenue was $306 million, reflecting a 17% increase over 2020, with operational revenue growth at constant currency rates of 14% and a positive currency impact of 3% [7][42] - The net loss for 2021 was $250.5 million or $0.92 per share, compared to a net loss of $276.9 million or $1.07 per share for the prior year [42] - Cash, cash equivalents, and marketable securities were $482.5 million at December 31, 2021, compared to $483.3 million at the end of 2020 [46] Business Line Data and Key Metrics Changes - Galafold's total product revenue was $306 million globally, with $210 million (69%) generated outside the U.S. and $95 million (31%) from within the U.S. [23][24] - The company ended 2021 with over 1,750 patients on Galafold globally, achieving nearly half of the global market share of treated amenable patients [28] - Compliance and adherence rates for Galafold exceeded 90%, indicating strong patient retention [30] Market Data and Key Metrics Changes - In Europe, the company achieved an 80% to 90% market share of switch patients, while in the U.S. and Japan, the market remains approximately 50/50 between switch and naïve patients [29] - New markets in Latin America and Asia Pacific are showing greater traction, contributing to growth expectations for the coming years [29] Company Strategy and Development Direction - The company has decided not to spin off its gene therapy programs due to unfavorable market conditions and will focus on core franchises in Fabry and Pompe diseases [11][12] - Strategic investments will be made in the global growth of Galafold and the launch of AT-GAA, with a target of achieving profitability in 2023 [10][12] - The company anticipates approximately $400 million in net cost savings through 2026 by streamlining its R&D operations [13][45] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory approval of AT-GAA, with a PDUFA action date set for May 29, 2022, and July 29, 2022, for the NDA and BLA, respectively [8][36] - The company remains committed to maintaining a strong financial position and achieving profitability without the need for dilutive financing [10][45] - Management highlighted the importance of patient access to Galafold and the anticipated demand for AT-GAA, indicating a positive outlook for future growth [30][40] Other Important Information - The company is preparing for the global launch of AT-GAA, leveraging its existing commercial organization that has experience in rare disease markets [32][40] - The leadership transition is set for August 1, 2022, with Bradley Campbell succeeding John Crowley as CEO [20] Q&A Session Summary Question: Inquiry about mid-cycle review and inspections - Management indicated a positive mid-cycle review with no surprises and confirmed that inspections of the WuXi facility are planned [49][50] Question: Inventory position and supply chain management - The company confirmed a strong inventory position to support launches in the U.S. and Europe, with effective supply chain management in place [51][53] Question: Trends in patient initiation for Galafold - Management noted that patient initiation trends are improving, with a return to normalcy in interactions between patients and physicians [56][58] Question: Uptake expectations for AT-GAA compared to Neo-GAA - Management expressed confidence in the uptake of AT-GAA, particularly among switch patients, based on compelling clinical data [60][63] Question: Gene therapy strategies and differentiation - Management clarified that gene therapy programs will not move into the clinic in the near term, focusing instead on core science and technology [67][71]