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Investing.com -- Capricor Therapeutics (NASDAQ: NASDAQ:CAPR) shares climbed 12% following the announcement that the U.S. Food and Drug Administration (FDA) has granted Priority Review to its Biologics License Application (BLA) for deramiocel, a treatment for Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA’s acceptance of the BLA signifies a critical step towards potentially bringing the first therapy for DMD cardiomyopathy to market, with a target action date set for August 31, 2025.
The BLA submission for deramiocel, an investigational cell therapy, is bolstered by cardiac data from Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials. This data is compared against natural history data from an FDA-funded dataset on DMD cardiomyopathy and potential biomarkers of disease progression. The FDA has not identified any potential review issues at this stage and has not yet decided if an Advisory Committee meeting will be necessary.
Capricor’s CEO, Linda Marbán, Ph.D., expressed enthusiasm about the advancement towards offering this treatment for a condition with no approved therapies. If successful, deramiocel would be a quarterly-administered lifelong treatment with the potential for broad adoption in the treatment landscape for DMD-cardiomyopathy. The company also acknowledged the contributions of patients, families, and advocates, as well as the FDA’s commitment to expediting treatments for DMD.
Dr. Craig McDonald, national PI and Chair of the Department of Physical Medicine and Rehabilitation at the University of California, Davis, emphasized the clinical trials’ findings that deramiocel attenuates DMD-cardiomyopathy, a leading cause of death among those with DMD. The safety and efficacy data suggest that the approval of deramiocel could introduce a first-in-class therapeutic option for the condition.
In addition to the Priority Review, deramiocel has received Orphan Drug Designation from the FDA and EMA, as well as RMAT and ATMP Designations in the U.S. and Europe, respectively. Furthermore, Capricor could be eligible for a Priority Review Voucher if deramiocel is approved by September 30, 2026, due to its rare pediatric disease designation.
In response to the news, Oppenheimer analyst Leland Gershell commented, "With shares trading at an EV of just $680M at last night’s close despite $1B+ sales potential in the US alone and attractive terms with commercial partner NS Pharma, we expect the stock to trade up sharply as the market interprets today’s news as meaningfully derisking." The positive analyst sentiment reflects the market’s reaction to the potential derisking of Capricor’s stock with the FDA’s latest move.
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