Apitegromab - (FORGE)

September 2, 2026: Scholar Rock (NASDAQ: SRRK) said the FDA granted Fast Track and Orphan Drug designations to apitegromab for facioscapulohumeral muscular dystrophy (FSHD), and that dosing has begun in the Phase 2 FORGE trial.

FORGE (NCT07435129) is a randomized, double-blind, placebo-controlled, multicenter study of apitegromab monotherapy in adults with genetically confirmed FSHD. About 60 participants will be randomized 1:1 to apitegromab 10 mg/kg or placebo IV every four weeks for 52 weeks. The primary endpoint is percent change from baseline in total lean muscle volume by MRI at Week 52. Secondary endpoints include lean muscle volume at Week 24, changes in other muscle parameters including muscle fat fraction at Weeks 24 and 52, and safety/tolerability; exploratory functional endpoints will also be assessed.

The company said the FSHD program is supported by translational preclinical data in the FLExDUX4 mouse model, where a murine form of apitegromab increased muscle mass, strength, and endurance. The release also notes apitegromab remains investigational and is not approved for any use.