Capricor Reports Five-Year Deramiocel Data Showing Sustained Muscle and Cardiac Benefits in Duchenne Muscular Dystrophy

Capricor Therapeutics has reported positive five-year follow-up data from the ongoing HOPE-2 Open-Label Extension (OLE) study (NCT04428476) of deramiocel (CAP-1002), demonstrating sustained benefits in both skeletal and cardiac muscle function among patients with Duchenne muscular dystrophy (DMD).

The findings, presented at the Parent Project Muscular Dystrophy (PPMD) 2026 Annual Conference, complement previously reported positive results from the pivotal Phase 3 HOPE-3 trial (NCT05126758), which met both its primary and key secondary endpoints.

The long-term data strengthen the clinical evidence supporting deramiocel as the therapy remains under U.S. FDA review, with a Prescription Drug User Fee Act (PDUFA) target action date of August 22, 2026.

Scientific and Clinical Context

Deramiocel is an investigational allogeneic cell therapy composed of cardiosphere-derived cells (CDCs). These cells release exosomes that modulate immune responses and reduce fibrosis by shifting macrophages toward a tissue-repair phenotype. The therapy is intended to preserve both skeletal and cardiac muscle function in DMD, a progressive X-linked neuromuscular disorder caused by the absence of functional dystrophin.

DMD primarily affects boys and leads to progressive muscle weakness, loss of mobility, respiratory decline, and cardiomyopathy, which remains the leading cause of death. Despite advances in treatment, effective therapies that provide long-term protection of cardiac and upper limb function remain limited.

 Five-Year HOPE-2 OLE Findings

The ongoing HOPE-2 OLE study evaluated nine patients who continued receiving deramiocel for five years. Upper limb function, assessed using the Performance of the Upper Limb (PUL 2.0) scale, declined by fewer than five points over the study period. This compares favorably with external natural-history data, where standard-of-care patients typically experience an estimated decline of approximately 2.4 points annually, translating to roughly 12 points over five years. A separate published natural-history analysis also reported an average decline of 8.1 points over three years in non-ambulatory patients.

Cardiac function also remained stable throughout the five-year follow-up. Left ventricular ejection fraction (LVEF), measured by cardiac magnetic resonance imaging, showed no meaningful deterioration, contrasting with a modeled annual decline of approximately 3.2% observed in propensity-matched external controls.

Safety remained consistent with previous experience. Across the overall clinical development program, more than 800 intravenous infusions have been administered without identifying new safety signals.

HOPE-3 Phase 3 Results Reinforce Long-Term Benefit

The long-term extension data were presented alongside previously disclosed results from the randomized, double-blind, placebo-controlled HOPE-3 Phase 3 trial, which enrolled 106 patients with DMD. The study achieved statistical significance for its primary endpoint measuring upper limb function (PUL 2.0; p=0.03) and its key secondary endpoint evaluating cardiac function through LVEF (p=0.04), while also meeting all Type I error-controlled secondary endpoints.

According to Chief Executive Officer Linda Marbán, Ph.D., the combined HOPE-2 and HOPE-3 datasets provide consistent evidence that deramiocel delivers durable improvements in both skeletal and cardiac muscle function while maintaining a favorable long-term safety profile.

Regulatory Status and FDA Review

Deramiocel has received multiple regulatory designations, including Orphan Drug Designation from both the U.S. FDA and European Medicines Agency, Regenerative Medicine Advanced Therapy (RMAT) designation in the United States, Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA.

Capricor’s Biologics License Application (BLA) is currently under FDA review, with a regulatory decision expected by August 22, 2026. If approved, deramiocel could become an important new treatment option that addresses both skeletal and cardiac disease progression in patients with Duchenne muscular dystrophy.

What This Means for Patients

For people living with Duchenne muscular dystrophy, progressive loss of upper limb function and declining heart function eventually affect independence, daily activities, and survival. The five-year HOPE-2 extension data suggest that deramiocel may slow disease progression over the long term while preserving both skeletal and cardiac muscle function, with no new safety concerns identified.

If the therapy receives FDA approval, it could become one of the few treatments to demonstrate durable benefits across both muscle and heart health, potentially helping patients maintain mobility, arm function, and cardiac performance for longer. The upcoming FDA decision on August 22, 2026, could mark an important milestone for the Duchenne community, where effective long-term treatment options remain limited.

Reference

Capricor to Present Positive Five-Year HOPE-2 OLE Data and HOPE-3 Phase 3 Results for Deramiocel in Duchenne Muscular Dystrophy at PPMD 2026 Annual Conference :: Capricor Therapeutics, Inc. (CAPR)