Key Takeaways
- Shares of Capricor Therapeutics plummeted up to 70% following the release of FDA staff briefing documents
- FDA staff expressed concerns that the company modified trial endpoint measurements after completing its late-stage study
- Federal regulators questioned whether enrolled trial participants genuinely had cardiomyopathy related to Duchenne muscular dystrophy
- The agency noted insufficient evidence demonstrating adequate delivery of the therapeutic agent to cardiac tissue
- A meeting with an external advisory panel is set for Wednesday, July 29
Shares of Capricor Therapeutics (CAPR) experienced a devastating collapse of up to 70% on Monday following the Food and Drug Administration’s publication of staff briefing materials in advance of the company’s scheduled advisory committee review on July 29.
Capricor Therapeutics, Inc., CAPR
The briefing documents prepared by FDA staff highlighted multiple concerns regarding the efficacy evidence supporting deramiocel, the company’s investigational cell-based treatment designed for cardiomyopathy in male Duchenne muscular dystrophy (DMD) patients.
The biotechnology company’s shares had already declined approximately 40% during early market hours before accelerating losses throughout the trading day.
The upcoming Cellular, Tissue, and Gene Therapies Advisory Committee session on Wednesday will evaluate whether data from the critical HOPE-3 clinical trial offers substantial proof of deramiocel’s therapeutic efficacy.
Among the FDA’s primary objections is Capricor’s decision to modify the methodology for calculating the trial’s primary endpoint results following study completion.
The biotech firm transitioned from evaluating absolute score changes on a 42-point upper limb functionality assessment to computing outcomes as percentage-based modifications. Federal reviewers indicated this methodological shift lacked scientific rationale.
“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.
Cardiac Assessment Methodology Also Modified
The protocol for evaluating cardiac performance underwent changes as well. The initial design called for monitoring modifications in left ventricular ejection fraction. However, Capricor pivoted to a ranking-based analysis of patient outcomes.
FDA staff members indicated that evidence demonstrating sufficient concentrations of the intravenously administered treatment reaching cardiac tissue to confer clinical benefit was inadequate.
Concerns Regarding Patient Selection
Federal reviewers raised doubts about whether trial participants genuinely suffered from DMD-associated cardiomyopathy. They observed that enrolled patients demonstrated normal cardiac pumping capacity on average at baseline.
Deramiocel represents an experimental allogeneic cell-based product sourced from donated cardiac tissue obtained from deceased donors.
The initial Biologics License Application submission relied on findings from the earlier-stage HOPE-2 investigation, which failed to demonstrate efficacy improvements in either skeletal or cardiac parameters. That regulatory filing resulted in a Complete Response Letter citing insufficient effectiveness evidence and an unfavorable benefit-risk profile.
Currently, no FDA-approved therapeutic interventions exist specifically targeting cardiomyopathy associated with Duchenne muscular dystrophy.
Available DMD treatments, such as Sarepta Therapeutics’ (SRPT) gene therapy Elevidys and various exon-skipping medications, address the primary genetic disorder rather than the progressive cardiac complications that emerge as the disease advances.
The advisory committee review is scheduled to convene Wednesday, July 29.
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