Deramiocel improves arm function and cardiac measures in phase 3 trial as FDA advisory panel reviews Capricor's application

The Lancet on Wednesday published positive phase 3 results for deramiocel, an experimental cell therapy for advanced Duchenne muscular dystrophy, showing statistically significant benefits on upper-limb function and cardiac measures, with a safety profile comparable to placebo. The publication lands as U.S. regulators separately weigh whether the evidence is strong enough to support approval.

The therapy, also known as CAP-1002, is being developed by Capricor Therapeutics. It is an allogeneic heart-derived cell therapy, meaning it is made from donor cells, and it is gene-agnostic, so it is not limited to patients with a specific dystrophin mutation.

That matters in Duchenne muscular dystrophy, or DMD, an X-linked genetic disease caused by the absence of dystrophin. The disorder leads to progressive muscle weakness and cardiomyopathy, a weakening of the heart muscle, and cardiac disease is the leading cause of death in DMD. While patients may receive corticosteroids, supportive heart and respiratory care, and some mutation-specific medicines, treatment options are limited for people with advanced disease, especially therapies that might affect both skeletal muscle decline and heart damage.

The HOPE-3 study, published July 29, was a phase 3, multicenter, randomized, double-blind, placebo-controlled trial involving 106 participants. Boys and young men ages 10 and older were enrolled, including both ambulatory and non-ambulatory patients, and were randomized 1:1 to deramiocel or placebo. Treatment was given by outpatient intravenous infusion on Day 1 and again at months 3, 6 and 9.

Researchers assessed skeletal muscle function using Performance of the Upper Limb 2.0, or PUL 2.0, a scale commonly used in Duchenne to measure arm and hand function as mobility worsens. Cardiac function was evaluated with cardiac MRI, including left ventricular ejection fraction, or LVEF, a measure of how well the heart pumps blood, and late gadolinium enhancement, or LGE, an imaging marker associated with heart scarring. The trial also used a prespecified Global Statistical Test combining total PUL 2.0, LVEF and Patient Global Impression of Severity.

Across those measures, deramiocel outperformed placebo. On total PUL 2.0, the reported treatment difference was 4.55%, with a 95% confidence interval of 0.47 to 8.63 and a p-value of 0.029. On the mid-level PUL 2.0 measure, the difference was 8.12%, with a 95% confidence interval of 2.12 to 14.11 and a p-value of 0.008. Ranked change in LVEF also favored deramiocel, with a treatment difference of 11.65, a 95% confidence interval of 0.47 to 22.82 and a p-value of 0.041; the mean absolute LVEF difference was about 2.4 percentage points in favor of treatment. Change in LGE favored deramiocel by minus 3.00 affected segments, with a 95% confidence interval of minus 5.50 to minus 0.51 and a p-value of 0.022. The Global Statistical Test was also positive, with a z-score of 0.27, a 95% confidence interval of 0.05 to 0.49 and a p-value of 0.015. The study reported a safety profile comparable to placebo.

The publication comes the same day the Food and Drug Administration’s Cellular, Tissue, and Gene Therapies Advisory Committee is scheduled to review Capricor’s biologics license application for deramiocel. Capricor has said the FDA granted Priority Review and set an Aug. 22, 2026, target action date. According to the FDA’s advisory committee briefing materials, “The original BLA received a Complete Response due to lack of substantial evidence of effectiveness and an unfavorable benefit-risk assessment.”

That regulatory review is focused less on whether the trial produced encouraging signals than on whether HOPE-3 provides what the agency considers substantial evidence of effectiveness. In briefing documents prepared for the advisory committee, the FDA raised methodological questions, including whether the statistical analysis plan was properly finalized before unblinding and how sensitive the findings are to different analytical assumptions and to the handling of missing data.

Capricor has publicly disputed parts of that characterization, saying its final statistical analysis plan was completed before unblinding and arguing that the FDA relied on an earlier draft version. The company has also pointed to the peer-reviewed publication as support for the strength of the dataset.

HOPE-3 builds on the earlier HOPE-2 phase 2 trial, published in The Lancet in 2022, which showed signals on upper-limb and cardiac outcomes but was not enough on its own to secure approval. With HOPE-3 now published and the advisory committee meeting underway, the next major milestone is the FDA’s decision in August.