Capricor Therapeutics (NASDAQ:CAPR) shares dropped 40% on Monday after the U.S. Food and Drug Administration published briefing documents ahead of an upcoming advisory committee meeting to review the company’s lead cell therapy candidate.
The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee is scheduled to meet on July 29 to evaluate Capricor’s resubmitted Biologics License Application (BLA) for deramiocel, which is being developed to treat cardiomyopathy in male patients with Duchenne muscular dystrophy (DMD).
FDA highlights evidence required for approval
The advisory committee will assess whether findings from the pivotal Phase 3 HOPE-3 trial provide sufficient evidence to support approval of deramiocel for the proposed indication.
In its briefing materials, the FDA reiterated that substantial evidence of effectiveness generally requires at least two adequate and well-controlled clinical studies, with each trial independently demonstrating convincing results.
Previous application received Complete Response Letter
Deramiocel is an investigational allogeneic cell therapy derived from donor heart tissue.
Capricor’s original BLA relied on data from the Phase 2 HOPE-2 trial and its open-label extension. According to the FDA, those studies did not demonstrate evidence of effectiveness in improving skeletal or cardiac function, including measurements based on left ventricular ejection fraction and other cardiac endpoints.
The agency previously issued a Complete Response Letter, citing insufficient evidence of efficacy and determining that the therapy’s overall benefit-risk profile did not support approval.
Advisory committee review in focus
Duchenne muscular dystrophy is a rare inherited disorder that causes progressive muscle degeneration beginning in childhood.
Cardiomyopathy is one of the leading causes of death among patients with DMD, yet currently approved treatments for the disease have not been systematically evaluated for their impact on heart-related complications.
Investors will now closely watch the July 29 advisory committee meeting, which could provide important insight into the regulatory outlook for deramiocel ahead of any future FDA decision.
Capricor Therapeutics stock price
