Capricor Therapeutics Inc. (CAPR), on Wednesday, reported positive topline results from its pivotal Phase 3 HOPE-3 trial of Deramiocel, its investigational cell therapy for Duchenne Muscular Dystrophy (DMD).
DMD is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting skeletal, respiratory, and cardiac muscles. Cardiomyopathy is the leading cause of mortality in patients, underscoring the urgent needs for therapies that preserve both muscle and heart function.
The HOPE-3 study was a randomized, double-blind, placebo-controlled trial enrolling 106 boys and young men across 20 U.S. clinical sites. Participants received intravenous Deramiocel or placebo every three months for 12 months.
The trial met its primary end point, showing a 54% slowing of skeletal muscle disease progression as measured by the Performance of Upper Limb (PUL v2.0) score, with statistical significance at p=0.029.
It also achieved its key secondary endpoint, demonstrating a 91 percent slowing of decline in left ventricular ejection fraction (LVEF), with statistical significance at p=0.041.
In addition, statistical significance was achieved in type 1 error- controlled secondary endpoints, underscoring the consistency of Deramiocel's clinical benefits.
Deramiocel maintained a favorable safety and tolerability profile consistent with prior clinical experience.
Chief Executive Officer Linda Marbán, Ph.D., said the results reinforce the durable benefits seen in HOPE-2 and its open-label extension, which has continued for over 48 months, and position the company to address the clinical issues raised in the FDA's Complete Response Letter earlier this year.
Craig McDonald, M.D., National PI of the HOPE-3 trial, emphasized that a 54% slowing of skeletal muscle decline is extraordinary in Duchenne and directly linked to maintaining independence and quality of life.
Jonathan Soslow, M.D., of Vanderbilt University, added that the cardiac findings represent a significant advance in the management of Duchenne cardiomyopathy.
Deramiocel, also known as CAP-1002, consists of allogenic cardiosphere-derived cells that secrete exosomes to modulate immune responses and reduce fibrosis.
The therapy has received Orphan Drug Designation from both the FDA and EMA, as well as RMAT designation in the U.S. Capricor plans to submit HOPE-3 data in response to the FDA's Complete Response Letter, which was issued in July 2025 and publicly posted in September, aiming to advance Deramiocel toward regulatory approval. Detailed results are expected to be presented at a future scientific meeting and published in a peer-reviewed journal.
The company ended September 30, 2025, with cash, cash equivalents and marketable securities balance of approximately $98.6 million. Capricor expects this cash position to support operations into the fourth quarter of 2026.
When we alerted readers to CAPR on Sep.30, 2025, it was trading at $7.39.
CAPR shares soared as high as $40.37 during intraday trading yesterday, to close at $29.96, up 371%. In premarket trading today, the stock is down 2.10% at $29.33
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