Capricor Drug Slows Duchenne Upper Limb Decline 76% in Trial
New open-label extension data show deramiocel sharply slowed upper limb deterioration in Duchenne muscular dystrophy patients after crossover from placebo.
Capricor Therapeutics presented new clinical data at the 2026 World Muscle Society Congress showing its experimental therapy deramiocel significantly slowed upper limb decline in patients with Duchenne muscular dystrophy, according to results from the HOPE-3 open-label extension study.
The most striking finding came from a 24-month crossover analysis. Patients who had spent their first year on placebo and then switched to deramiocel saw their rate of upper limb decline slow by 76% compared with the deterioration they had experienced during that prior placebo year — using each patient as their own historical control.
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Duchenne muscular dystrophy is a rare, progressive genetic disease that primarily affects boys and leads to steadily worsening muscle weakness. Upper limb function is a critical measure of daily independence for patients as the disease advances, making preservation of arm and hand strength a key clinical target.
The crossover design of the analysis strengthens the signal by removing between-patient variability, since the same individuals serve as the reference point for comparison. Analysts and patient advocates are likely to scrutinize whether the effect size holds in broader or longer-term datasets as Capricor advances the program.
Capricor did not announce regulatory submission timelines or commercial availability details alongside the congress presentation. Continue reading at GlobeNewswire - Industry News on Financial Services.