Capricor Therapeutics (NASDAQ: CAPR) has announced positive topline results from its pivotal Phase 3 HOPE-3 trial evaluating Deramiocel for the treatment of Duchenne muscular dystrophy (DMD).
HOPE-3 is a randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating Deramiocel in boys and young men with DMD. The study randomized 106 participants across 20 leading U.S. clinical sites. Participants received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for a 12-month period.
In a statement, Linda Marbán, PhD, CEO of Capricor, commented, “HOPE-3 delivered strong and definitive evidence that Deramiocel can meaningfully improve the course of DMD, demonstrating statistically significant improvements in both skeletal and cardiac function. 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 results should be sufficient to support regulatory approval.”
Dr. Marbán added, “For families living with Duchenne who are looking for therapies that preserve functional ability, protect the heart and maintain independence, today’s results provide real momentum and meaningful progress, offering renewed confidence as we work to advance Deramiocel toward potential regulatory approval.”
