Capricor Therapeutics Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy

Core Insights - Capricor Therapeutics announced positive topline results from the Phase 3 HOPE-3 trial for Deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD) [1][2] - The trial demonstrated statistically significant improvements in both skeletal and cardiac function, reinforcing previous findings from the HOPE-2 trial [2][5] - The results are expected to support regulatory approval following a Complete Response Letter from the FDA earlier this year [2][5] Study Details - HOPE-3 was a randomized, double-blind, placebo-controlled trial involving 106 participants across 20 U.S. clinical sites, with an average age of approximately 15 years [2][11] - Participants received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for 12 months [2][12] - The study maintained a favorable safety and tolerability profile consistent with prior clinical experience [2][5] Efficacy Results - The primary endpoint, Performance of Upper Limb (PUL v2.0), showed a 54% slowing of progression compared to placebo (p=0.029) [3][5] - The key secondary endpoint, Left Ventricular Ejection Fraction (LVEF), demonstrated a 91% improvement (p=0.041) [3][5] - Statistically significant results were achieved across all controlled secondary endpoints [5] Clinical Implications - The findings indicate meaningful treatment effects on both upper limb function and cardiomyopathy, addressing critical aspects of DMD [5][6] - The preservation of cardiac function is particularly significant, as cardiomyopathy is the leading cause of mortality in DMD [6][8] - The results provide renewed confidence for families seeking therapies that maintain functional ability and protect heart health [6][10] Company Background - Capricor Therapeutics focuses on developing cell and exosome-based therapeutics for rare diseases, with Deramiocel as its lead candidate for DMD [13] - Deramiocel has received Orphan Drug Designation from the FDA and EMA, along with other designations that may facilitate regulatory approval [10][15] - The company is committed to advancing innovative therapies and has a proprietary platform for targeted delivery of therapeutics [13]