Duchenne: Deramiocel Two-Year Extension Data, Company-Reported
In short: Capricor Therapeutics reported open-label extension data from its HOPE-3 trial of deramiocel in Duchenne muscular dystrophy. People who switched from placebo lost less arm function in their first treated year than in their placebo year. The numbers are company-reported, descriptive and not tested for statistical significance. The FDA is due to decide on November 22, 2026. This is not a cure.
What was reported
Capricor Therapeutics presented results from the open-label extension of HOPE-3, a trial of the cell therapy deramiocel in Duchenne muscular dystrophy. Of 106 people randomized, 98 joined the extension, 49 from each original group.
The arm-function numbers
Arm function was measured with the Performance of Upper Limb (PUL 2.0) scale. Capricor says people who switched from placebo to deramiocel lost 0.49 points in their first treated year, compared with 2.05 points during their year on placebo.
People treated with deramiocel for both years lost 0.95 points in year one and 0.89 in year two, which the company says suggests the effect held up over time.
What the data cannot show
The extension was open-label, so patients and doctors knew who received the drug. Capricor states the open-label analyses had no allocation for statistical significance testing and were not powered for Month 24 comparisons. Month 24 assessments covered 42 people from the original placebo group and 40 from the original deramiocel group.
The company also compared observed declines with predicted natural-history declines, but these are comparisons to a model, not to a control group.
What happens next
Capricor says the extension data were included in a major amendment to its biologics license application. The FDA's target action date is November 22, 2026. Deramiocel is not approved for Duchenne today, and no cure for Duchenne exists.
Frequently asked questions
Is deramiocel approved for Duchenne?
No. The FDA has a target action date of November 22, 2026, according to Capricor.
Do these results prove deramiocel works?
No. They are company-reported, open-label and not tested for statistical significance, so they are an early signal and not proof.
Read the full Duchenne muscular dystrophy (DMD) report: latest studies, treatments, trials near you, expert care centers and support groups, updated daily.
Sources
- Capricor Therapeutics presents positive HOPE-3 open-label extension data. Capricor press release via Stock Titan, October 5, 2026
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