Deramiocel (CAP-1002) - (HOPE-3)

October 5, 2026: Capricor Therapeutics (NASDAQ: CAPR) reported late-breaking World Muscle Society 2026 data from the HOPE-3 open-label extension of Deramiocel (CAP-1002) in Duchenne muscular dystrophy patients with upper limb impairment. In the delayed-start group, patients who switched from placebo to Deramiocel had a 76% reduction in rate of upper limb decline versus their own prior placebo year, with PUL 2.0 LS mean change improving from -2.05 in Year 1 (n=52) to -0.49 in Year 2 on Deramiocel (n=49), a difference of 1.56 points (95% CI 0.47 to 2.64). In the early-start group, decline was similar across two years on Deramiocel (-0.95 then -0.89; difference 0.05, 95% CI -1.03 to 1.14).

At Month 24, observed decline was less than predicted by natural history in both groups: -3.76 vs -5.35 for the delayed-start arm after 12 months on Deramiocel, and -3.42 vs -5.88 for the early-start arm after 24 months on treatment. The company said these data were included in a major amendment to Deramiocel’s BLA. FDA PDUFA target action date is November 22, 2026. A webinar with PPMD is scheduled for October 7, 2026.