Capricor Therapeutics Stock Down 70% as FDA Raises Concerns Over Deramiocel

Capricor Therapeutics' (CAPR) stock plummeted as much as 70% on Monday, following the release of FDA briefing documents ahead of an upcoming advisory committee meeting.

The FDA raised concerns about the effectiveness data for deramiocel, Capricor's cell therapy targeting cardiomyopathy in male patients with Duchenne muscular dystrophy (DMD).

The agency staff flagged that Capricor changed how it measured trial results after its late-stage HOPE-3 study ended. Specifically, the company switched from analyzing scores on a 42-point arm-function test to calculating results as a percentage change.

The FDA reviewers questioned whether patients in the trial actually had DMD-related cardiomyopathy, noting that they had normal heart-pumping function on average at the start of the trial. Additionally, there was limited evidence that enough of the intravenously delivered therapy reached the heart to provide a benefit.