Capricor reports new Phase 3 HOPE-3 data showing deramiocel improves cardiac and functional outcomes in Duchenne muscular dystrophy as FDA review continues.
Written By: Katherashala Dharan Kumar, PharmD
Reviewed By: Pharmacally Editorial Team
Capricor Therapeutics reported additional analyses and new functional outcomes from the Phase 3 HOPE-3 trial (NCT05126758) evaluating deramiocel (CAP-1002) in patients with Duchenne muscular dystrophy (DMD). The data were presented at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Orlando, Florida.
Deramiocel is currently under review by the U.S. Food and Drug Administration, with a PDUFA action date of August 22, 2026.
Cardiac MRI analyses showed that deramiocel may improve cardiac structure in patients with DMD. Investigators evaluated late gadolinium enhancement (LGE), a marker of myocardial fibrosis associated with worsening cardiac function.
Results demonstrated a significant reduction in fibrotic segments in patients treated with deramiocel compared with placebo, corresponding to a three-segment treatment difference at 12 months (p=0.022).
In patients with baseline cardiomyopathy, deramiocel produced a greater treatment effect on cardiac function. Treatment resulted in a 3.3 percentage-point improvement in left ventricular ejection fraction (LVEF) versus placebo, representing more than 100 percent attenuation of expected cardiac decline (p=0.017).
A Global Statistical Test (GST), a composite endpoint including Performance of Upper Limb (PUL v2.0), LVEF, and Patient Global Impression of Severity (PGI-S), demonstrated a statistically significant overall benefit favoring deramiocel (p=0.017). The composite integrates functional ability, cardiac performance, and patient-reported disease severity.
Additional functional outcomes also supported the treatment effect. Data from the Duchenne Video Assessment (DVA), which evaluates activities of daily living in patients with DMD, showed that performance in the “eat 10 bites” task slowed disease progression by approximately 83 percent compared with placebo (p=0.018).
The findings aligned with improvements in the mid-level elbow domain of the PUL v2.0 assessment (p=0.008), indicating preservation of arm and hand function.
“These newly released data provide additional clinically meaningful evidence supporting the potential impact of deramiocel on the daily lives and long-term outcomes of patients living with Duchenne,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor Therapeutics. She noted that the Duchenne Video Assessment captures real-world tasks important to patients, including eating independently, and that these results align with preserved arm and hand function observed in the HOPE-3 study. Marbán added that Capricor remains focused on working with the FDA toward potential approval.
Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs) with immunomodulatory and anti-fibrotic properties. These cells release extracellular vesicles known as exosomes that influence macrophages to adopt a reparative, anti-inflammatory phenotype, helping preserve cardiac and skeletal muscle function.
The HOPE-3 trial results form a key part of Capricor’s response to the Complete Response Letter (CRL) previously issued by the U.S. FDA for deramiocel. The company is using the full Phase 3 dataset, including the newly presented analyses, to address the agency’s questions and support its resubmission and ongoing review of the Biologics License Application.
The therapy has received Orphan Drug Designation from both the FDA and the European Medicines Agency, as well as Regenerative Medicine Advanced Therapy (RMAT), Advanced Therapy Medicinal Product (ATMP), and Rare Pediatric Disease designations.
Duchenne muscular dystrophy is a severe X-linked genetic disorder caused by the absence of dystrophin, a protein essential for maintaining muscle cell integrity. The disease leads to progressive degeneration of skeletal, respiratory, and cardiac muscles and primarily affects boys. Cardiac complications, including cardiomyopathy and heart failure, remain a leading cause of death in patients with DMD.
References
Capricor Therapeutics Announces Late-Breaking HOPE-3 Data at the 2026 MDA Conference Demonstrating Significant Functional Benefits of Deramiocel for Duchenne Muscular Dystrophy, 12 March 2026, Capricor Therapeutics Announces Late-Breaking HOPE-3 Data at the 2026 MDA Conference Demonstrating Significant Functional Benefits of Deramiocel for Duchenne Muscular Dystrophy :: Capricor Therapeutics, Inc. (CAPR)
Positive HOPE-3 Phase 3 Results Support Deramiocel as a Potential New Treatment for Duchenne Muscular Dystrophy, 05 December 2026, https://pharmacally.com/positive-hope-3-phase-3-results-support-deramiocel-as-a-potential-new-treatment-for-duchenne-muscular-dystrophy/
Capricor’s Deramiocel BLA Review Reinstated by FDA Following HOPE-3 Trial Data, 12 March 2026, https://pharmacally.com/capricors-deramiocel-bla-review-reinstated-by-fda-following-hope-3-trial-data/
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy (HOPE-3), ClinicalTrials.gov Identifier: NCT05126758, https://clinicaltrials.gov/study/NCT05126758
About the Writer
Katherashala Dharan Kumar, PharmD A dedicated Clinical Researcher with expertise in medical and scientific writing clinical trials, drug safety, and healthcare innovation. Actively contributes to research publications and clinical documentation focused on improving patient outcomes through evidence-based practices and ethical research standards. Believe that every complex data point has a human story waiting to be told. Focusing on providing actionable insights for clinical research.