Capricor Therapeutics Completes BLA Submission for Deramiocel Treatment of Duchenne Muscular Dystrophy

Capricor Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology firm specializing in innovative cell and exosome-based therapies for rare diseases, finalized the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for deramiocel. This investigational cell therapy aims to address Duchenne muscular dystrophy (DMD) cardiomyopathy, a condition affecting patients with DMD.

Notably, the Company disclosed that the BLA submission has led to the activation of a $10.0 million milestone payment from Nippon Shinyaku under their U.S. Exclusive Commercialization and Distribution Agreement, dated January 24, 2022. Capricor has marked this achievement in an official press release, confirming the successful completion of the BLA submission process.

Linda Marbán, Ph.D., the Chief Executive Officer of Capricor, emphasized the significance of this milestone, highlighting the potentially transformative impact of deramiocel on individuals grappling with DMD. Dr. Marbán underscored the robust clinical data supporting deramiocel’s effectiveness in mitigating the cardiac complications associated with DMD. The Company aims to collaborate closely with the FDA during the review phase to facilitate potential approval for this therapy.

The BLA submission incorporates data from Capricor’s Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials, bolstered by comparative data from an FDA-referenced dataset regarding DMD cardiomyopathy and potential disease progression markers. Capricor has petitioned for a priority review to expedite the standard 10-month review period to a six-month review post-acceptance by the FDA.

Concurrent with this achievement, Capricor stands to benefit from a $10 million milestone payment facilitated by its distribution partner, Nippon Shinyaku Co., Ltd., and stands to receive additional support from Orphan Drug Designation by the FDA and European Medicines Agency (EMA).

Deramiocel represents a significant advancement in treating DMD, offering promising immunomodulatory and regenerative effects for affected patients. Capricor remains committed to pioneering groundbreaking therapeutic approaches for rare diseases and continues to explore the potential of its revolutionary exosome technology.

Investors and stakeholders keen on following Capricor’s progress are urged to monitor future FDA proceedings and clinical developments closely. The Company’s dedication to reshaping treatment paradigms for rare diseases like DMD underscores its commitment to transformative healthcare solutions.

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read Capricor Therapeutics’s 8K filing here.

About Capricor Therapeutics

Capricor Therapeutics, Inc (NASDAQ:CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor’s lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy.