Business Wire·Jan 12, 2026, 7:00 AM ET

Scholar Rock Highlights 2026 Strategic Priorities

Scholar Rock anticipates apitegromab BLA resubmission & U.S. launch in 2026 for SMA treatment following FDA approval.
FDA scheduled meeting with Catalent Indiana (Novo Nordisk) in early 2026 to review remediation progress at the Bloomington fill-finish facility.
Apitegromab IND cleared for facioscapulohumeral muscular dystrophy (FSHD); Phase 2 FORGE trial initiation & patient dosing expected mid-2026.
Subcutaneous apitegromab Phase 1 study completed; data showed favorable bioavailability comparable to IV administration.
SRK-439 Phase 1 trial in healthy volunteers ongoing; topline data expected in H2 2026.
EMA regulatory review for apitegromab MAA ongoing with decision expected mid-2026.
Second U.S. fill-finish facility commercial capacity reserved starting Q1 2026; supplemental BLA submission planned later in 2026.
Phase 2 OPAL trial for apitegromab in SMA patients under two years of age is enrolling with dosing underway.
Company holds approximately $365 million in cash as of December 31, 2025, supporting operations into 2027.
Scholar Rock management to present updates at the 44th Annual J.P. Morgan Healthcare Conference on January 12, 2026.