Capricor plans FDA amendment after advisory committee rejects cardiomyopathy endpoint, stock jumps

Capricor Therapeutics received a negative vote from an FDA advisory committee on the efficacy of its cell therapy, deramiocel, for treating cardiomyopathy in Duchenne muscular dystrophy. The nine‑to‑three vote was followed by a sharp rise in the company’s share price.

The committee’s concern centered on a secondary endpoint in the Hope‑3 trial, which measured heart function. The trial’s primary goal was to assess improvements in upper‑limb function, a result that was not the focus of the advisory discussion.

In response, Capricor announced it will submit an amendment to its approval application that narrows the indication to the primary upper‑limb endpoint. The filing will include 24‑month data from the open‑label extension of Hope‑3 and additional analyses of existing results. The company expects the FDA to extend the PDUFA action date to accommodate the amendment.