fidrisertib (IPN60130) / Blueprint Medicines, Ipsen - LARVOL DELTA

FALKON: A Study to Assess the Effectiveness and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP). (clinicaltrials.gov) - P2 | N=113 | Active, not recruiting | Sponsor: Clementia Pharmaceuticals Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Aug 2029 ➔ Mar 2029

Enrollment closed • Trial completion date • Pediatrics • ACVR1

An ALK2 inhibitor, BLU-782, prevents heterotopic ossification in a mouse model of fibrodysplasia ossificans progressiva. (PubMed, Sci Transl Med) - "Delayed dosing revealed a short 2-day window after injury when BLU-782 treatment prevented HO in ALK2R206H mice, but dosing delays of 4 days or longer abrogated HO prevention. Together, these data suggest that BLU-782 may be a candidate for prevention of HO in FOP."

Journal • Preclinical • Genetic Disorders • ACVR1 • ALK1

Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva: Methodology of the Randomized, Double-Blind, Placebo-Controlled Phase II FALKON Trial (ACMG 2024) - P2 | "Results from FALKON will allow evaluation of fidrisertib in FOP."

Clinical • P2 data • Genetic Disorders • Pain • ACVR1

FALKON: A Study to Assess the Effectiveness and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP). (clinicaltrials.gov) - P2 | N=98 | Recruiting | Sponsor: Clementia Pharmaceuticals Inc. | Trial completion date: Aug 2025 ➔ Aug 2029

Trial completion date • Pediatrics • ACVR1

Safety, Tolerability, Pharmacokinetics, and Food Effect of BLU-782 in Healthy Adults (clinicaltrials.gov) - P1; N=108; Completed; Sponsor: Blueprint Medicines Corporation; Recruiting ➔ Completed; N=80 ➔ 108; Trial primary completion date: Jun 2019 ➔ Sep 2019

Clinical • Enrollment change • Trial completion • Trial primary completion date

Safety, Tolerability, Pharmacokinetics, and Food Effect of BLU-782 in Healthy Adults (clinicaltrials.gov) - P1; N=80; Recruiting; Sponsor: Blueprint Medicines Corporation

Clinical • New P1 trial