Written By: Pharmacally Medical News Desk
Capricor Therapeutics has reported positive topline results from the Phase 3 HOPE-3 study of Deramiocel (CAP-1002), showing meaningful preservation of skeletal muscle function and heart health in boys and young men with Duchenne muscular dystrophy (DMD). The trial reached its primary endpoint with a 54 percent slowing of upper-limb functional decline compared with placebo. These data will support the company’s response to the FDA’s earlier Complete Response Letter as it prepares a renewed approval submission.
Trial Design and Outcomes
HOPE-3 (NCT05126758) enrolled 106 participants across 20 US sites in a randomized, double-blind, placebo-controlled design. Patients received intravenous Deramiocel, 150 million cells every three months for 12 months, or placebo.
Primary Endpoint- Performance of Upper Limb (PUL v2.0) total score:
Deramiocel slowed decline by 54 percent versus placebo.
This benefit was seen across ambulatory and non-ambulatory patients, indicating preservation of functional abilities important for daily independence.
Key Secondary Endpoint-Left Ventricular Ejection Fraction (LVEF)
The treatment group showed 91 percent preservation of cardiac function compared with placebo.
Protecting heart function is especially important in DMD, where cardiomyopathy is the leading cause of death.
The company reports that both primary and key secondary endpoints reached statistical significance.
Endpoint | Deramiocel Benefit | Clinical Relevance |
Primary- Performance of Upper Limb (PUL v2.0) Total Score | 54% slowing | Upper limb function |
Key Secondary- Left Ventricular Ejection Fraction (LVEF %)² | 91% preservation | Heart function in DMD |
Deramiocel Mechanism and Regulatory Path
Deramiocel contains allogeneic cardiosphere-derived cells, which act through paracrine signaling rather than long-term engraftment. These cells secrete bioactive exosomes that influence immune activity within damaged cardiac and skeletal muscle.
The exosomes reprogram macrophages toward a more regenerative and less pro-inflammatory state, reducing chronic inflammation and slowing fibrotic tissue replacement. Through these immunomodulatory and anti-fibrotic actions, Deramiocel helps preserve muscle integrity and supports better functional outcomes in conditions such as Duchenne muscular dystrophy.
The Deramiocel holds: Orphan Drug designation, Rare Pediatric Disease designation, RMAT designation and ATMP classification in Europe. If approved, the Rare Pediatric Disease designation may qualify Capricor for a Priority Review Voucher.
Linda Marbán, PhD, CEO of Capricor, said the HOPE-3 results provide strong evidence that Deramiocel can meaningfully change the course of DMD, building on more than four years of data from prior studies. She noted that the new findings support the company’s planned response to the FDA’s earlier Complete Response Letter and offer new hope for families seeking therapies that maintain function and protect the heart.
Craig McDonald, MD, Distinguished Professor at UC Davis Health and HOPE-3 National PI, called the 54 percent slowing of upper-limb decline “extraordinary,” especially for non-ambulatory patients where few therapies show measurable benefit. He highlighted the importance of preserving daily functional abilities and maintaining quality of life.
Jonathan Soslow, M.D., MSCI, Professor of Pediatrics (Cardiology) at Vanderbilt University Medical Center added HOPE-3’s cardiac findings mark a significant advance in DMD cardiomyopathy management, the leading cause of mortality. Deramiocel preservation of left ventricular ejection fraction addresses a major unmet need.
Capricor will include the HOPE-3 data in its response to the FDA’s Complete Response Letter from earlier in 2025. The company already aligned with the FDA on using these data to support approval in DMD. The FDA had previously accepted a BLA for DMD cardiomyopathy with priority review, supported by HOPE-2 results, but later issued a CRL. This led Capricor to prepare a new submission strategy. Full HOPE-3 results will be presented soon.
References
Capricor Therapeutics Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy, 03 December 2025, Capricor, https://www.capricor.com/investors/news-events/press-releases/detail/331/capricor-therapeutics-announces-positive-topline-results
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3), ClinicalTrials.gov ID NCT05126758, https://clinicaltrials.gov/study/NCT05126758