Approval and Launch of MIPLYFFA
The first FDA-approved therapy for Niemann-Pick disease type C was launched, with 90 prescription enrollment forms submitted as of October 31.
Rare Pediatric Disease Priority Review Voucher
Received a priority review voucher with the approval of MIPLYFFA, intended to be monetized for future growth.
Clinical Pipeline Progress
Achieved end of Phase II meeting with the FDA for KP1077 in idiopathic hypersomnia and dosed new patients in Phase III program for celiprolol.
Strong Financial Position
Completed an underwritten public offering with net proceeds of approximately $64.5 million, extending cash runway into 2027.