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Capricor Therapeutics Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy
Pivotal Phase 3 randomized, double-blind, placebo-controlled study (n=106) met the primary endpoi...
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[{"type":"text","content":"Capricor Therapeutics Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy\n\n\n\n\n\n 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)\n \n\n\n\n Statistical significance was achieved in all type 1 error controlled secondary endpoints\n \n\n\n\n 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\n \n\n\n\n Deramiocel maintained a favorable safety and tolerability profile consistent with prior clinical experience\n \n\n\n\n Company plans to submit its response to the Complete Response Letter incorporating HOPE-3 data, following prior alignment with FDA\n \n\n\n\n Conference call and webcast today at 8:00 a.m. ET\n \n\n\n\n\n\n SAN DIEGO, Dec. 03, 2025 (GLOBE NEWSWIRE) --\n \n Capricor Therapeutics\n \n (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced positive topline results from its pivotal Phase 3 HOPE-3 trial evaluating Deramiocel, the Company’s investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD).\n \n\n “HOPE-3 delivered strong and definitive evidence that Deramiocel can meaningfully improve the course of Duchenne muscular dystrophy, demonstrating statistically significant improvements in both skeletal and cardiac function,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “These results reinforce the durable benefits seen in HOPE-2 and its open-label extension, which has continued for over 48 months, and highlight the strength, consistency, and reproducibility of Deramiocel’s clinical profile after more than a decade of rigorous clinical development. We believe these pivotal study results, in addition to the evidence from the HOPE-2 and HOPE-2 OLE studies, position us to address the clinical issues in the Complete Response Letter received earlier this year, consistent with prior FDA guidance that HOPE-3...