Key Takeaways
- Shares of Capricor Therapeutics plummeted up to 70% following critical FDA briefing documents on deramiocel
- FDA staff criticized post-trial modifications to primary endpoint measurements
- Concerns raised about whether study participants truly suffered from DMD-associated heart disease
- Regulators noted insufficient proof that adequate therapy amounts reached cardiac tissue
- The external advisory panel convenes Wednesday, July 29 to evaluate the treatment
Shares of Capricor Therapeutics (CAPR) collapsed by as much as 70% Monday following the release of FDA briefing materials in advance of the company’s advisory committee review scheduled for July 29.
Capricor Therapeutics, Inc., CAPR
The briefing documents from FDA staff highlighted significant concerns regarding efficacy data for deramiocel, the company’s experimental cell-based therapy designed to treat cardiomyopathy in male Duchenne muscular dystrophy (DMD) patients.
The biotechnology stock had already declined approximately 40% during morning trading before losses accelerated throughout the remainder of the session.
The Cellular, Tissue, and Gene Therapies Advisory Committee is scheduled to convene Wednesday to evaluate whether findings from the critical HOPE-3 clinical trial demonstrate substantial proof of deramiocel’s effectiveness.
A primary FDA concern centers on Capricor’s decision to modify the measurement methodology for the trial’s primary endpoint after study completion.
The biotech company converted its analysis from absolute scores on a 42-point upper extremity function assessment to calculating outcomes as percentage changes. The FDA stated this modification lacked scientific justification.
“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,” agency staff stated in the briefing documents.
Cardiac Measurement Methodology Modified Post-Study
The assessment method for cardiac function also underwent alteration. The initial protocol called for tracking absolute changes in left ventricular ejection fraction. However, Capricor modified this approach to a ranking-based system for patient outcomes.
FDA staff expressed skepticism about whether sufficient quantities of the intravenously administered therapy actually reached cardiac tissue to produce meaningful clinical benefit.
Concerns About Patient Population Selection
Regulatory reviewers raised questions about whether trial participants genuinely suffered from DMD-associated cardiomyopathy. They observed that patients demonstrated normal cardiac pumping function on average when entering the study.
Deramiocel represents an investigational allogeneic cell therapy product manufactured from cardiac tissue obtained from deceased donors.
The initial biologics license application relied on data from the phase 2 HOPE-2 study, which failed to demonstrate effectiveness on either skeletal muscle or cardiac function. That submission resulted in a Complete Response Letter citing inadequate evidence of effectiveness and an unfavorable benefit-risk profile.
Currently, no FDA-approved treatments exist specifically targeting cardiomyopathy associated with Duchenne muscular dystrophy.
Available DMD therapies, including Sarepta Therapeutics’ (SRPT) gene therapy Elevidys and various exon-skipping medications, address the underlying genetic disease rather than the progressive cardiac complications that emerge as the condition advances.
The advisory committee review is scheduled for Wednesday, July 29.
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