Late-breaking data from the Phase 3 HOPE-3 trial of deramiocel in Duchenne muscular dystrophy (DMD), presented at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, suggest potential benefits across cardiac and functional outcomes. The investigational first-in-class therapy is currently under review by the FDA for DMD cardiomyopathy, with a Prescription Drug User Fee Act (“PDUFA”) target action date of 22 August, 2026.
The HOPE-3 trial is a randomized, double-blind, placebo-controlled Phase 3 study evaluating quarterly intravenous infusions of deramiocel over 12 months in boys and young men with DMD receiving standard-of-care corticosteroids. The study previously met its primary endpoint of upper limb function, assessed by Performance of Upper Limb (PUL v2.0), as well as a key secondary cardiac endpoint (left ventricular ejection fraction [LVEF]), with a favourable safety profile reported to be consistent with earlier studies.
About deramiocel
Deramiocel is an investigational allogeneic cell therapy composed of cardiosphere-derived cells, a population of cardiac-derived cells with immunomodulatory and anti-fibrotic properties. These cells act by secreting extracellular vesicles (exosomes), which target macrophages and alter their expression profile towards a reparative, rather than pro-inflammatory phenotype. Through these effects, deramiocel is proposed to support preservation of cardiac and skeletal muscle function in DMD.
Late-breaking data
Cardiac magnetic resonance imaging demonstrated reduced progression of myocardial fibrosis in patients treated with deramiocel compared with placebo, corresponding to a statistically significant reduction in fibrotic segments at 12 months. Myocardial fibrosis was assessed using late gadolinium enhancement (LGE), a marker associated with progressive cardiac dysfunction and heart failure risk in DMD cardiomyopathy.
Among patients with baseline cardiomyopathy, deramiocel was associated with a 3.3 percentage-point improvement in LVEF relative to placeb. Furthermore, a Global Statistical Test combining PUL v2.0, LVEF and patient-reported disease severity (Patient Global Impression of Severity) showed a statistically significant overall benefit favouring deramiocel.
Functional outcomes related to hand-to-mouth activity, a measure of patient independence, were assessed using the Duchenne Video Assessment. In the “eat 10 bites” task, treatment was associated with an approximately 83% slowing of disease progression compared with placebo. These findings were consistent with improvements observed in mid-level upper limb function, suggesting concordance between clinical assessments and real-world measures of patient independence.
About Duchenne muscular dystrophy cardiomyopathy
Duchenne muscular dystrophy (DMD) is a severe, X-linked genetic disorder caused by the absence of functional dystrophin, leading to progressive degeneration of skeletal, respiratory and cardiac muscle. While initially characterised by skeletal muscle weakness, cardiac involvement becomes increasingly prominent over time, with deterioration of the myocardium resulting in cardiomyopathy and heart failure, the leading cause of death in DMD. Treatment options remain limited, particularly for addressing cardiac disease progression.
Regulatory review
Separately, regulatory review of deramiocel for DMD-associated cardiomyopathy has resumed following a prior Complete Response Letter issued in 2025. The Biologics License Application has been classified as a Class 2 resubmission, and a target action date of August 2026 has been set under the PDUFA.
The resubmission incorporated additional data from the HOPE-3 trial. If approved, deramiocel may represent a novel therapeutic approach targeting both cardiac and functional decline in DMD. The therapy may also be eligible for a Priority Review Voucher if approved.
References
- Capricor Therapeutics™. Capricor Therapeutics Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy. Available at: https://www.capricor.com/investors/news-events/press-releases/detail/331/capricor-therapeutics-announces-positive-topline-results (accessed on 17 March 2026).
- Capricor Therapeutics™. Capricor Therapeutics Announces Late-Breaking HOPE-3 Data at the 2026 MDA Conference Demonstrating Significant Functional Benefits of Deramiocel for Duchenne Muscular Dystrophy. Available at: https://www.capricor.com/investors/news-events/press-releases/detail/339/capricor-therapeutics-announces-late-breaking-hope-3-data (accessed on 17 March 2026).
- Capricor Therapeutics™. Capricor Therapeutics Announces Establishment of New PDUFA Date for Deramiocel BLA. Available at: https://www.capricor.com/investors/news-events/press-releases/detail/338/capricor-therapeutics-announces-establishment-of-new-pdufa (accessed on 17 March 2026).
More content in congenital conditions.
Cite: Late-breaking data support DMD cardiomyopathy therapy under regulatory review. touchCARDIO. 17 March 2026.
Editor: Victoria Smith, Senior Content Editor.
Disclosures: This article was created by the touchCARDIO team utilizing AI as an editorial tool (ChatGPT (GPT-4o) [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.
Related content:
SIGN UP to touchCARDIOLOGY!
Join our global community today for access to thousands of peer-reviewed articles, expert insights and learn-on-the-go education across 150+ specialties, plus concise email updates and newsletters so you never miss out.
