Deramiocel Meets Key Endpoints in Phase 3 HOPE-3 Duchenne Trial
Key Clinical Summary
- Phase 3 HOPE-3 trial reports statistically significant improvements in both skeletal muscle function and cardiac function in boys with Duchenne muscular dystrophy (DMD) treated with deramiocel.
- Deramiocel slowed skeletal muscle disease progression by 54% and reduced decline in left ventricular ejection fraction (LVEF) by 91% compared with placebo.
- The cardiosphere-derived cell therapy demonstrated benefit across all type 1 error–controlled secondary endpoints, including upper-limb function, cardiac structure, hand-to-mouth function, and CK-MB biomarkers.
Deramiocel, an investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD), met its primary and secondary endpoints in the phase 3 HOPE-3 trial (NCT05126758), according to Capricor Therapeutics.
“HOPE-3 delivered strong and definitive evidence that deramiocel can meaningfully improve the course of Duchenne muscular dystrophy, demonstrating statistically significant improvements in both skeletal and cardiac function,” said Linda Marbán, PhD, chief executive officer of Capricor.
Deramiocel consists of cardiosphere-derived cells (CDCs), a rare cell type that has shown “exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMDs,” according to clinical studies. CDCs act by emitting exosomes to target macrophages and shift them from pro-inflammatory to a healing phenotype.
The phase 3 multicenter, randomized, double-blind, placebo-controlled trial enrolled 106 participants across 20 clinical sites in the United States, including both ambulatory and nonambulatory boys. At baseline, 90% of the population received cardiac medications, and 75% had been diagnosed with cardiomyopathy.
Participants were randomized to receive deramiocel (150 million cells per infusion) or placebo every 3 months for a total of 4 doses during the first 12 months of the trial. The primary endpoint was change from baseline in upper-limb function (measured by Performance of the Upper Limb test, version 2.0 [PUL 2.0]). Secondary endpoints included changes in cardiac muscle function and structure, changes in hand-to-mouth function, quality of life assessments, and biomarker analysis for creatine kinase MB isoenzyme (CK-MB).
Compared to placebo, deramiocel exhibited a 54% slowing of skeletal muscle disease progression (P = 0.029) and a 91% slowing in cardiac function decline (measured by left ventricular ejection fraction [LVEF]; P = 0.041). Statistical significance was achieved in all type 1 error controlled secondary endpoints, Capricor reported in a news release.
“We believe the HOPE-3 PUL results show statistically and clinically meaningful and significant treatment effects on both upper limb function and cardiomyopathy,” said Craig McDonald, MD, distinguished professor of physical medicine & rehabilitation and pediatrics at UC Davis Health, and principal investigator of the HOPE-3 trial. “A nearly 54[%] slowing of skeletal muscle disease progression is extraordinary in [DMD] and directly linked to maintaining independence and quality of life in the most severely affected patients with [the] greatest unmet need.”
Capricor plans to submit the findings from HOPE-3 as part of its response to the complete response letter (CRL) it received from the US Food and Drug Administration (FDA) in July 2025. According to the FDA, the application had insufficient evidence supporting the efficacy of deramiocel for cardiomyopathy associated with DMD.
Detailed results from HOPE-3 are also anticipated to be presented at a “future scientific meeting” and published in a peer-reviewed journal, according to the company’s press release.
References
Capricor Therapeutics. Capricor Therapeutics announces positive topline results from pivotal phase 3 HOPE-3 study of deramiocel in Duchenne muscular dystrophy. News release. December 3, 2025. Accessed December 5, 2025. https://www.capricor.com/investors/news-events/press-releases/detail/331/capricor-therapeutics-announces-positive-topline-results
