Clinical Trial Highlights

Rare Disease Key ongoing clinical-trial highlights TRIAL POPULATION PATIENTS DESIGN Palovarotene MOVE Phase III NCT03312634 Fidrisertib FALKON Phase II NCT05039515 IPSEN Innovation for patient care FOP (chronic) 107 PRIMARY ENDPOINT(S) STATUS Palovarotene - 5mg QD and upon flare-up, 20mg QD for 28 days, followed by 10mg for 56 days Annualized change in new HO volume U.S. regulatory approval August 2023 Placebo or FOP (chronic) 00 90 two dosing regimens Annualized change in new HO volume and safety of fidrisertib First patient commenced dosing Q1 2022 FOP: fibrodysplasia ossificans progressiva; QD: once a day; HO: heterotopic ossification; P DUFA: Prescription Drug User Fee Act. 23

View entire presentation