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Capricor Therapeutics Shared Topline Results From Its Phase 3 HOPE-3 Trial Evaluating Deramiocel Cell Therapy For Duchenne Muscular Dystrophy, The Trial Met The Primary Endpoint And The Key Secondary Cardiac Endpoint

Benzinga · 3d ago
  • Pivotal Phase 3 randomized, double-blind, placebo-controlled study (n=106) met the primary endpoint (PUL v2.0) and the key secondary cardiac endpoint (LVEF), both achieving statistical significance (p=0.03 and p=0.04, respectively)
  • Statistical significance was achieved in all type 1 error controlled secondary endpoints
  • Results demonstrate clinically meaningful and statistically significant skeletal and cardiac benefits, supporting Deramiocel as a potential first-in-class therapy designed to treat Duchenne cardiomyopathy, the leading cause of mortality in Duchenne
  • Deramiocel maintained a favorable safety and tolerability profile consistent with prior clinical experience
  • Company plans to submit its response to the Complete Response Letter incorporating HOPE-3 data, following prior alignment with FDA