Capricor Therapeutics Inc. (CAPR) has unveiled late-breaking results from its pivotal Phase 3 HOPE-3 trial of Deramiocel in Duchenne muscular dystrophy presented at the American Academy of Neurology (AAN) 2026 Annual Meeting in Chicago.
The findings mark a major moment for the Duchenne community, highlighting measurable preservation of upper-limb function- a milestone no prior therapy has achieved.
Duchenne muscular dystrophy (DMD) is a rare, progressive genetic disorder that weakens skeletal, cardiac, and respiratory muscles, primarily affecting boys. Over time, loss of muscle strength leads to severe disability and heart failure, the leading cause of death in DMD. Current treatments offer limited benefit, making functional preservation a critical goal.
In the HOPE-3 study, Deramiocel delivered statistically and clinically meaningful improvements in the Performance of the Upper Limb (PUL v2.0) scale- the trial's primary endpoint- compared with placebo. These results were reinforced by the Duchenne Video Assessment (DVA), which captured real-world tasks such as self-feeding, showing slowed disease progression and improved independence.
Dr. Aravindhan Veerapandiyan, Associate Professor at Arkansas Children's Hospital, who presented the data, noted that "no therapeutic has ever delivered a statistically and clinically meaningful difference in the upper limb functional endpoint."Capricor's CEO, Linda Marbán, Ph.D., said the results "tell a compelling story of preserved function, slowed decline, and real-world impact," adding that the company's Biologics License Application (BLA) for Deramiocel is currently under FDA review, with a target action of August 22, 2026.
Deramiocel (CAP-1002) is an allogeneic cardiosphere-derived cell therapy designed to modulate inflammation and fibrosis in muscle tissue. The cells secrete exosomes that reprogram macrophages toward a healing phenotype, helping preserve cardiac and skeletal muscle function. The therapy has received Orphan Drug, RMAT, and Rare Pediatric Disease designations from the FDA, and ATMP status in Europe.
Capricor has partnered with Nippon Shinyaku CO., Ltd (NS Pharma) for exclusive commercialization of Deramiocel as potential first-in-class therapy of alternating the course of Duchenne muscular dystrophy - a disease long defined by decline rather than preservation.
CAPR has traded between $4.30 and $40.37 over the past year. The stock closed Wednesday's trading at $35.00, down 0.96%. In pre-market trading, the stock is at $34.00, down 2.86%.
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