San Diego – October 19, 2026 -- Capricor Therapeutics (NASDAQ: CAPR) reported that Duchenne muscular dystrophy patients who switched from placebo to Deramiocel after one year slowed their upper limb decline by 76% compared with their own prior year, according to open-label extension data from the Phase 3 HOPE-3 trial. The U.S. FDA has set a PDUFA target action date of November 22, 2026, for the therapy.
Crossover analysis shows 76% slower decline on PUL 2.0
The late-breaking poster was presented at the 31st Annual Congress of the World Muscle Society in Hiroshima, Japan. It measured upper limb function with the Performance of Upper Limb (PUL 2.0) scale. Of 106 randomized patients, 98 entered the open-label extension, 49 per arm. At Month 24, 42 patients in the placebo-origin arm and 40 in the Deramiocel arm were assessed.
In the placebo-origin arm, the observed decline in the untreated first year was 2.7 points, against 2.9 predicted by a natural history model. That matched a published non-ambulant cohort that declined about 2.7 points per year.
Both arms declined more slowly than natural history predicts
At 24 months, both groups showed slower upper limb decline than the natural history model predicts. Patients on Deramiocel for both years declined at a similar rate in years 1 and 2. Craig McDonald, MD, principal investigator of HOPE-3 at UC Davis Health, said this indicates the slowing was triggered by starting treatment rather than by the passage of time.
The analysis carries limits. No alpha was allocated to the open-label analyses, and the extension was not powered for Month 24 comparisons. The natural history comparison is descriptive only, with no matching or statistical testing.
FDA review includes the extension data ahead of November 22 decision date
The extension data are part of the major amendment to the Deramiocel Biologics License Application. The amendment also contains sensitivity and robustness analyses supporting the HOPE-3 randomized trial results. CEO Linda Marbán said Capricor will continue working with the FDA through the remainder of the review.
HOPE-3 randomized 106 patients to intravenous Deramiocel or placebo every three months over 12 months. The one-year results were published in The Lancet in July 2026. In the U.S., Deramiocel holds Orphan Drug, RMAT and Rare Pediatric Disease designations, and the Rare Pediatric Disease designation may qualify Capricor for a Priority Review Voucher upon approval.
Capricor and Parent Project Muscular Dystrophy will host a webinar on the data on October 7, 2026, at 1:00 p.m. ET.