Capricor's DMD drug slows upper limb decline in 24-month study

Capricor Therapeutics (NASDAQ: CAPR) presented data from the HOPE-3 open-label extension study showing that patients with Duchenne muscular dystrophy (DMD) who switched from placebo to its experimental drug Deramiocel experienced a 76% reduction in the rate of upper limb decline during their first year on the treatment, compared with their prior year on placebo.

The data were presented at the 31st Annual Congress of the World Muscle Society in Hiroshima, Japan. The study enrolled 106 patients, with 98 entering the open-label extension phase. Among patients who received Deramiocel for both years of the study, the rate of upper limb decline remained similar between year one and year two, measuring -0.95 and -0.89, respectively, on the PUL 2.0 total score scale.

At 24 months, both patient groups showed less upper limb decline than natural history models predicted. The delayed-start group, which spent the first year on placebo, recorded an observed decline of -3.76 at month 24, compared with a predicted decline of -5.35. The early-start group, on Deramiocel for both years, showed an observed decline of -3.42 versus a predicted -5.88.

Capricor noted that the open-label extension was not statistically powered for month-24 comparisons, and no alpha was allocated to these analyses. Deramiocel remains investigational and has not received regulatory approval.

The data have been submitted to the U.S. Food and Drug Administration as part of a major amendment to the Deramiocel Biologics License Application. The FDA's PDUFA target action date is November 22, 2026.

Craig McDonald, MD, principal investigator at UC Davis Health, said the crossover design allowed clinicians to observe each patient's response before and after starting treatment, calling the within-patient comparison "a powerful way to see a treatment effect."

The one-year results from the randomized, placebo-controlled portion of HOPE-3 were previously published in The Lancet in July 2026.

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