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SAN DIEGO - Capricor Therapeutics (NASDAQ: NASDAQ:CAPR), a biotechnology firm with a market capitalization of $622 million, announced today that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025, for the potential approval of the therapy. According to InvestingPro data, the company maintains a strong financial position with a current ratio of 4.19, indicating robust liquidity to support its development pipeline.
Deramiocel, if approved, would be the first treatment specifically for DMD cardiomyopathy, a severe condition leading to heart failure and a major cause of death in patients with DMD. Currently, no therapies are approved for this aspect of the disorder. The BLA submission is backed by data from Capricor’s Phase 2 trials and a comparison with natural history data funded by the FDA. The market has responded positively to the company’s development progress, with the stock showing a remarkable 214% return over the past six months. InvestingPro analysis suggests the stock is currently trading near its Fair Value, with 12 additional ProTips available for subscribers.
The FDA has granted the BLA Priority Review status, which is designated for drugs that could significantly improve treatment for serious conditions. However, the agency has not yet decided if an advisory committee meeting is necessary.
Linda Marbán, Ph.D., CEO of Capricor, expressed optimism about the therapy’s potential to become a widely adopted treatment for DMD cardiomyopathy, administered quarterly throughout a patient’s life.
Dr. Craig McDonald, a leading researcher involved in the clinical trials, highlighted the therapy’s demonstrated ability to attenuate the effects of DMD cardiomyopathy.
Deramiocel has also received Orphan Drug Designation from the FDA and the European Medicines Agency (EMA), as well as Regenerative Medicine Advanced Therapy (RMAT) and Advanced Therapy Medicinal Product (ATMP) designations in the U.S. and Europe, respectively. Additionally, Capricor could be eligible for a Priority Review Voucher if the FDA approves deramiocel by September 30, 2026.
DMD is a genetic disorder affecting about 15,000-20,000 patients in the U.S., characterized by progressive muscle weakness and chronic inflammation. Deramiocel consists of allogeneic cardiosphere-derived cells, which have shown immunomodulatory and regenerative properties in clinical studies.
Capricor is also developing exosome-based therapies for various diseases through its StealthX™ platform. The company’s focus remains on advancing treatments for rare diseases, with deramiocel at the forefront of its late-stage development efforts.
This news is based on a press release statement from Capricor Therapeutics.
In other recent news, Capricor Therapeutics has completed its Biologics License Application (BLA) submission to the U.S. Food and Drug Administration for deramiocel, a cell therapy aimed at treating Duchenne muscular dystrophy cardiomyopathy. This submission, a significant milestone, could potentially lead to the first approved therapy for this condition if the FDA grants approval. The application is based on comprehensive clinical trials, including Phase 2 HOPE-2 and HOPE-2 Open Label Extension studies, which have shown promising results in managing cardiac complications associated with DMD. Capricor is optimistic about receiving a Priority Review designation, which could expedite the review process. As a result of the BLA submission, Capricor received a $10 million milestone payment from its partner, Nippon Shinyaku. H.C. Wainwright has reiterated its Buy rating for Capricor with a price target of $77, reflecting confidence in the company’s progress. Meanwhile, Jones Trading has maintained its Buy rating and $40 price target, despite an FDA warning letter sent to a researcher involved with the company’s deramiocel HOPE-3 study. The firm believes the market’s negative reaction to the letter was exaggerated and remains confident in Capricor’s prospects.
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