TLDR
- Capricor Therapeutics stock fell as much as 70% after FDA staff raised concerns about its cell therapy deramiocel
- The FDA flagged that Capricor changed how it measured results after its late-stage trial ended
- Reviewers questioned whether trial patients actually had DMD-related cardiomyopathy
- The FDA also said there was limited evidence the therapy reached the heart in sufficient amounts
- An external advisory committee meeting is scheduled for Wednesday, July 29
Capricor Therapeutics (CAPR) stock cratered as much as 70% on Monday after the FDA released briefing documents ahead of the company’s July 29 advisory committee meeting.
Capricor Therapeutics, Inc., CAPR
The FDA staff documents raised concerns about the effectiveness data for deramiocel, Capricor’s cell therapy targeting cardiomyopathy in male patients with Duchenne muscular dystrophy (DMD).
The stock had already fallen around 40% in early trading before extending losses further through the session.
The Cellular, Tissue, and Gene Therapies Advisory Committee will meet Wednesday to decide whether results from the pivotal HOPE-3 study provide substantial evidence that deramiocel works.
One of the FDA’s central concerns is that Capricor changed how it measured the trial’s main results after the study ended.
The company switched from analyzing scores on a 42-point arm-function test to calculating results as a percentage change. The FDA said this was not scientifically justified.
“FDA does not consider the conversion of raw change to percent change and then back to raw change to have been scientifically justified, as it adds complexity and reduces accuracy,” the agency’s staff wrote.
Heart Function Measurements Also Changed
The way heart function was measured was also altered. Originally, the plan was to track changes in left ventricular ejection fraction. Instead, Capricor shifted to ranking patients’ results.
The FDA reviewers said evidence was limited that enough of the intravenously delivered therapy actually reached the heart to provide a benefit.
Questions Over Trial Population
Reviewers also questioned whether the patients in the trial actually had DMD-related cardiomyopathy at all. They noted that patients had normal heart-pumping function on average at the start of the trial.
Deramiocel is an investigational allogeneic cellular product derived from cadaver donor hearts.
The original BLA submission was based on the earlier phase 2 HOPE-2 study, which showed no evidence of effectiveness on skeletal or cardiac function. That application received a Complete Response Letter due to lack of substantial evidence of effectiveness and an unfavorable benefit-risk assessment.
There are currently no FDA-approved therapies specifically for DMD-associated cardiomyopathy.
Existing DMD treatments, including Sarepta Therapeutics’ (SRPT) gene therapy Elevidys and exon-skipping drugs, target the underlying disease rather than the heart complications that develop over time.
The advisory committee meeting is set for Wednesday, July 29.
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