Capricor presents 24‑month deramiocel data showing 76% slower upper‑limb decline
Capricor Therapeutics released results from the 24‑month HOPE‑3 open‑label extension study of its deramiocel cell therapy for Duchenne muscular dystrophy. Patients who moved from placebo to deramiocel after one year experienced roughly a 76% reduction in the rate of upper‑limb function loss. The company filed the data with the FDA in a major amendment to the deramiocel Biologics License Application, which has a PDUFA target action date of November 22, 2026. Shares fell about 10% on the day the data were announced.
Why it matters
The FDA’s upcoming decision will determine whether deramiocel can be marketed, affecting Capricor’s future revenue potential. The stock price reaction shows investors are uncertain about approval despite the positive trial results.
Key facts
- 1Patients who switched from placebo to deramiocel after one year showed a 76% reduction in upper‑limb decline rate. investing.com
- 2The study enrolled 106 randomized patients, of whom 98 entered the open‑label extension. investing.com
- 3At 24 months the placebo arm declined by –3.76 versus a predicted –5.35, while the deramiocel arm declined by –3.42 versus a predicted –5.88. investing.com
- 4The FDA has set a PDUFA target action date of November 22, 2026 for deramiocel. rttnews.com
- 5Capricor’s shares dropped roughly 10% on the day the data were released. seekingalpha.com
Summary written by AlphAI from 3 of 3 sources. Not investment advice. Figures are as stated by the linked sources.