Successful Launch of MIPLYFFA
Exceeded internal expectations for MIPLYFFA with over one-third of diagnosed Niemann-Pick disease type C (NPC) patients in the U.S. enrolled. Received 122 prescription enrollment forms by March 31, 2025, with 13 in Q1 2025.
Expansion Plans for MIPLYFFA
Plans to expand availability of MIPLYFFA outside the U.S. with a marketing authorization application in Europe in the second half of 2025, targeting approximately 1100 people living with NPC.
Financial Strength and Flexibility
Monetization of pediatric rare disease priority review voucher added $148.3 million of non-dilutive capital to the balance sheet, increasing total cash, cash equivalents, and investments to $217 million.
Recognition of Innovation
Earned a spot on Fast Company's top 10 most innovative companies list in the medicines, therapeutics, and pharmaceuticals category.
Pipeline Progress with Celiprolol
Phase 3 Discover trial for Celiprolol continues to progress with 32 patients enrolled for vascular Ehlers-Danlos syndrome (VEDS).