Capricor Therapeutics Initiates FDA Submission for DMD Treatment

By Bobby Samwell • Wednesday, October 09 10:17 • Permanent Link

Capricor Therapeutics (NASDAQ: CAPR) has announced the initiation of the rolling submission process with the U.S. Food and Drug Administration (FDA) for a Biologics License Application (BLA) seeking full approval for Deramiocel to treat all patients diagnosed with Duchenne Muscular Dystrophy (DMD) cardiomyopathy.

The company plans to complete its rolling BLA submission by the end of 2024. If approved, Deramiocel could potentially provide significant improvements in the safety and/or effectiveness of the treatment for DMD cardiomyopathy, a serious condition for which there are currently no approved treatment options available.

Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a population of stromal cells that have been shown to exert potent immunomodulatory, antifibrotic, and regenerative actions in dystrophinopathy and heart failure. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials.

DMD is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart, and respiratory muscles, with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births, with the patient population estimated to be approximately 15,000-20,000 in the United States.

Capricor Therapeutics is dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. Its lead product candidate, Deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy, is currently advancing through phase 3 clinical development for the treatment of Duchenne Muscular Dystrophy. Additionally, Capricor is harnessing the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins, and small molecule therapeutics. As a result of these announcements, the company's shares have moved -6.5% on the market, and are now trading at a price of $18.08. If you want to know more, read the company's complete 8-K report here.

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