Capricor Announces Major Advances in Duchenne Muscular Dystrophy Treatment

SAN DIEGO, Dec. 3, 2025 — Capricor Therapeutics has reported significant findings from its pivotal Phase 3 HOPE-3 trial for Deramiocel, a cell therapy targeting Duchenne muscular dystrophy (DMD). The trial showed that Deramiocel improved both skeletal and cardiac functions in patients, meeting critical endpoints necessary for potential regulatory approval.

The trial involved 106 participants and demonstrated a 54% slowing of disease progression according to the Performance of Upper Limb (PUL v2.0) assessment. Additionally, the therapy resulted in a statistically significant improvement in left ventricular ejection fraction (LVEF), a key measure of heart function.

Linda Marban, CEO of Capricor, expressed confidence that the compelling results from this larger, placebo-controlled study could influence the FDA to reassess its earlier decision. In July, the FDA had rejected the treatment due to concerns over its effectiveness based on previous studies.

Dr. Marban referenced the long-term benefits seen in the prior HOPE-2 study, highlighting Deramiocel’s positive clinical profile over more than a decade of rigorous testing. Experts like Craig McDonald, the trial’s principal investigator, also praised the findings, emphasizing their potential to improve patients’ quality of life.

According to Dr. Jonathan Soslow of Vanderbilt University Medical Center, the cardiac results from HOPE-3 signal progress in DMD management, particularly addressing cardiomyopathy, a leading cause of mortality in affected individuals.

Capricor plans to respond to the FDA’s Complete Response Letter with the new data, hopeful for approval that could change treatment for DMD, a severe genetic disorder primarily affecting boys. The company anticipates presenting detailed results at upcoming scientific meetings and publishing findings in a peer-reviewed journal.