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Scholar Rock Receives FDA Fast Track Designation for Apitegromab Facioscapulohumeral Muscular Dystrophy (FSHD) Program as Participant Dosing Commences in Phase 2 FORGE Trial

SRRK
  • FDA granted Fast Track & Orphan Drug designations to apitegromab for the treatment of Facioscapulohumeral Muscular Dystrophy (FSHD).
  • Dosing has commenced in the Phase 2 FORGE clinical trial evaluating apitegromab in adults with genetically confirmed FSHD.
  • The FORGE trial is a randomized, double-blind, placebo-controlled, multi-center study enrolling approximately 60 participants.
  • Participants will receive apitegromab at 10 mg/kg or placebo intravenously every four weeks for 52 weeks.
  • The primary endpoint is the percent change from baseline in total lean muscle volume measured by MRI at Week 52.
  • Secondary endpoints include total lean muscle volume at Week 24, muscle fat fraction changes, & safety/tolerability assessments.
  • Preclinical data from the FLExDUX4 FSHD mouse model demonstrated that apitegromab increases muscle mass, strength, & endurance.
  • Apitegromab is a fully human monoclonal antibody that inhibits myostatin activation & is the first muscle-targeted therapy to show clinical success in a Phase 3 SMA study.
  • FSHD is a rare, progressive neuromuscular disease with no currently approved therapies, affecting an estimated 40,000 individuals in the US & EU.
  • Scholar Rock aims to expand its global leadership in myostatin biology across rare neuromuscular diseases through this program.