Capricor Therapeutics Reports Positive Phase 3 Results for Cell Therapy in DMD

SAN DIEGO, December 03, 2025 (GLOBE NEWSWIRE) — Capricor Therapeutics announced today that its pivotal Phase 3 HOPE-3 trial evaluating Deramiocel, the company’s investigational cell therapy for Duchenne muscular dystrophy (DMD), achieved its primary and key secondary endpoints. The trial demonstrated statistically significant improvements in both skeletal muscle function and cardiac function.

The company stated that these results reinforce the durable benefits observed in previous trials, including HOPE-2 and its 48-month open-label extension, and position Deramiocel for potential regulatory approval following a Complete Response Letter received earlier this year.

Efficacy Highlights: Slowing Skeletal and Cardiac Progression

HOPE-3 was a randomized, double-blind, placebo-controlled study involving 106 boys and young men with DMD, most of whom were non-ambulatory and had a clinical diagnosis of cardiomyopathy. Participants received intravenous infusions of Deramiocel or placebo every three months for 12 months. The therapy maintained a favorable safety profile consistent with prior clinical experience.

Clinical Findings:

• Skeletal Muscle Function: Deramiocel demonstrated a 54% slowing of skeletal muscle disease progression compared to placebo, as measured by the Performance of Upper Limb (PUL v2.0) Total Score (p=0.029). This effect is considered “extraordinary” in DMD, particularly in the largely non-ambulatory population studied, and directly links to maintaining independence and quality of life.

• Cardiac Function: The cell therapy achieved 91% preservation of Left Ventricular Ejection Fraction (LVEF %) compared to placebo (p=0.041). Cardiomyopathy (heart muscle deterioration) is the leading cause of death in DMD, and stabilizing LVEF addresses a major unmet medical need.

Dr. Craig McDonald, National Principal Investigator of HOPE-3, emphasized that the study is the “first-ever Phase 3 trial in a largely non-ambulatory population with DMD to successfully meet its primary endpoint.”

Deramiocel: Mechanism of Cell Therapy Action

Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs). This cell therapy works by utilizing these rare cardiac cells to exert potent immunomodulatory and anti-fibrotic actions. The CDCs secrete extracellular vesicles (exosomes), which target immune cells (macrophages) to change their expression profile, promoting a healing phenotype rather than a pro-inflammatory one, thereby helping to preserve cardiac and skeletal muscle function.

Deramiocel holds several key regulatory designations, including Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation. Capricor Therapeutics expects to submit detailed HOPE-3 results for presentation at an upcoming scientific meeting.