Phase 3 HOPE-3 Data Reinforce Skeletal and Cardiac Benefits of Deramiocel in Duchenne Muscular Dystrophy

New phase 3 findings presented at the 2026 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting further strengthened the case for deramiocel, an investigational allogeneic cell therapy from Capricor Therapeutics, as a potential treatment for Duchenne muscular dystrophy (DMD)-associated cardiomyopathy and progressive skeletal muscle decline.1 The data come at a pivotal regulatory moment for the therapy, with the FDA currently reviewing the biologics license application (BLA) and a Prescription Drug User Fee Act (PDUFA) date set for August 22, 2026.²

The newly presented HOPE-3 analysis demonstrated statistically significant improvements across both skeletal muscle and cardiac endpoints, while also showing reductions in myocardial scar progression measured by late gadolinium enhancement (LGE) on cardiac MRI. Investigators noted that the findings may provide the first clinical evidence that antifibrotic effects observed in preclinical DMD models can translate into measurable cardiac benefit in humans.1

Deramiocel is an investigational cell therapy composed of cardiosphere-derived cells isolated from human cardiac tissue. The therapy is designed to exert immunomodulatory, anti-inflammatory, and antifibrotic effects through extracellular vesicles and soluble signaling factors. Prior preclinical work in mdx mouse models suggested the therapy could preserve skeletal muscle function and attenuate cardiac fibrosis, helping establish the rationale for the phase 3 HOPE-3 trial.^3,4

“Deramiocel has successfully achieved primary muscle function and secondary cardiac endpoints in a second randomized placebo-controlled study with a favorable safety profile,” lead author Jonathon H. Soslow, MD, pediatric cardiologist at Vanderbilt University Medical Center, and colleagues wrote in the ASGCT presentation.¹

HOPE-3 was a multicenter, randomized, double-blind, placebo-controlled phase 3 study that enrolled 106 boys and young men aged 10 years and older with DMD. Participants were randomized to receive intravenous deramiocel or placebo every 3 months over 12 months. The primary endpoint evaluated change in Performance of Upper Limb (PUL 2.0) score, while key secondary endpoints included left ventricular ejection fraction (LVEF) and measures of myocardial fibrosis using cardiac MRI.

According to the presented findings, deramiocel met both the primary and key secondary endpoints. Treatment led to a statistically significant 4.55% advantage over placebo in total PUL 2.0 score (95% CI, 0.47-8.63; P = .029), with an even greater 8.12% benefit observed in mid-level PUL function (95% CI, 2.12-14.11; P = .008).¹

Cardiac outcomes also favored deramiocel. Investigators reported a statistically significant benefit in LVEF, corresponding to an absolute treatment difference of approximately 2.4% compared with placebo (P = .041). Among the subgroup of patients who underwent evaluable gadolinium-enhanced cardiac MRI scans, deramiocel reduced progression of myocardial fibrosis over 12 months, with a mean treatment difference of –3.00 affected cardiac segments (95% CI, –5.50 to –0.51; P = .022).¹

The cardiac fibrosis findings are particularly notable given that cardiomyopathy remains one of the leading causes of mortality in DMD. Progressive fibrofatty replacement of cardiac tissue contributes to declining ventricular function and eventual heart failure as patients age.

“These findings build on more than a decade of consistent clinical evidence and reinforce our confidence in Deramiocel’s potential,” Linda Marbán, PhD, chief executive officer of Capricor Therapeutics, previously said following release of topline HOPE-3 data.⁵

The HOPE-3 results also play a central role in the therapy’s ongoing FDA review. Capricor’s original BLA submission relied primarily on findings from the phase 2 HOPE-2 study and its open-label extension; however, the FDA issued a complete response letter (CRL) in July 2025, stating that the application did not yet meet the threshold for “substantial evidence of effectiveness.”⁶

Following the CRL, Capricor submitted topline HOPE-3 data and supporting analyses to the agency, prompting the FDA to resume review of the application earlier this year and assign the new August 2026 PDUFA date.² Importantly, the FDA reportedly did not request additional clinical trials, instead asking for the complete HOPE-3 clinical study report and supporting datasets as part of the ongoing review process.⁵

The current ASGCT presentation builds on prior HOPE-2 data published in The Lancet in 2022, where deramiocel demonstrated significant preservation of upper limb function and reductions in cardiac injury biomarkers over 12 months.⁷ Longer-term open-label extension data later suggested stabilization of cardiac function and slowing decline in upper limb performance through four years of follow-up.⁶

Across studies, deramiocel has generally maintained a favorable safety profile. In HOPE-3, investigators reported no imbalance in severe or serious adverse events between treatment and placebo groups.¹

If approved, deramiocel would become the first cell-based therapy approved for DMD cardiomyopathy and potentially the first therapy specifically targeting both cardiac and skeletal muscle dysfunction in later-stage DMD.

REFERENCES
1. Soslow JH, Villa C, Butterfield RJ, et al. Phase-3 efficacy in Duchenne muscular dystrophy in HOPE-3 confirms skeletal and cardiac muscle functional benefit and supports the clinical translation of anti-fibrotic activity for deramiocel, an allogeneic cardiac derived cell therapy. Presented at: 2026 American Society of Gene and Cell Therapy Annual Meeting; May 2026.
2. Capricor Therapeutics announces establishment of new PDUFA date for Deramiocel BLA. News release. Capricor Therapeutics. March 10, 2026. Accessed May 2026. https://www.capricor.com/investors/news-events/press-releases/detail/338/capricor-therapeutics-announces-establishment-of-new-pdufa
3. Rogers RG, et al. Disease-modifying bioactivity of intravenous cardiosphere-derived cells and exosomes in mdx mice. JCI Insight. 2019;4:e125754. doi:10.1172/jci.insight.125754
4. Li Y, et al. A Novel In Vitro Potency Assay Demonstrating the Anti-Fibrotic Mechanism of Action of CDCs in Deramiocel. Biomedicines. 2025;13:2652.
5. Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA Following FDA Review of HOPE-3 Topline Data. News release. Capricor Therapeutics. January 20, 2026. Accessed May 2026. https://www.capricor.com/investors/news-events/press-releases/detail/335/capricor-therapeutics-provides-regulatory-update-on
6. Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy. News release. Capricor Therapeutics. July 11, 2025. Accessed May 2026. https://www.capricor.com/investors/news-events/press-releases/detail/319/capricor-therapeutics-provides-regulatory-update-on
7. McDonald CM, Marban E, Hendrix S, et al. Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet. 2022;399(10329):1049-1058. doi:10.1016/S0140-6736(22)00012-5.