Capricor Therapeutics Makes Progress in DMD Treatment

By Bobby Samwell • Thursday, January 02 15:18 • Permanent Link

Capricor Therapeutics (NASDAQ: CAPR) has made significant strides in the development of its investigational cell therapy, Deramiocel, for the treatment of Duchenne Muscular Dystrophy (DMD) cardiomyopathy. The company has successfully completed the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking full approval for Deramiocel.

This milestone has triggered a $10 million milestone payment to Capricor from Nippon Shinyaku, the company's distribution partner. Deramiocel has received orphan drug designation from both the FDA and the European Medicines Agency (EMA).

The BLA submission is supported by Capricor's existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials, which have demonstrated attenuation of the cardiac implications of DMD. Capricor has also requested a priority review, which, if granted, would reduce the review timeline to six months from the date the submission is accepted by the FDA.

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

DMD is a devastating genetic disorder with an estimated occurrence in approximately one in every 3,500 male births and a patient population of 15,000-20,000 in the United States. The pathophysiology of DMD is driven by the impaired production of functional dystrophin, leading to progressive muscle weakness and chronic inflammation, with mortality at a median age of approximately 30 years. The leading cause of death among those with DMD is heart failure due to cardiomyopathy.

Capricor Therapeutics, Inc. is dedicated to advancing transformative cell and exosome-based therapeutics for rare diseases, with Deramiocel at the forefront of its innovation. The company is also utilizing its proprietary StealthX™ platform in preclinical development focused on vaccinology, targeted delivery of oligonucleotides, proteins, and small molecule therapeutics to potentially treat and prevent various diseases. Today the company's shares have moved 4.9% to a price of $14.48. Check out the company's full 8-K submission here.

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