Capricor Therapeutics Announces Treatment for Duchenne Muscular Dystrophy

Capricor Therapeutics, a biotechnology firm specializing in innovative cell and exosome-based treatments for rare diseases, has revealed that the U.S. Food and Drug Administration (FDA) will hold a Pre-BLA (Biologics License Application) meeting with the company in the third quarter of 2024. The focus of this meeting will be on deramiocel (CAP-1002), a therapy aimed at treating Duchenne Muscular Dystrophy (DMD). During this meeting, Capricor intends to finalize its BLA submission plans and collaborate with the FDA to define the rolling submission timeline.

Linda Marbán, Ph.D., CEO of Capricor, expressed her enthusiasm for the progress towards potentially gaining approval for deramiocel in treating DMD. Marbán noted the FDA's proactive stance in accelerating the availability of impactful therapies for DMD, emphasizing the substantial unmet needs of patients. She acknowledged the approval of gene therapy for DMD but highlighted the necessity for multiple therapeutic approaches to effectively combat the disease. Marbán suggested that deramiocel could serve as a cornerstone therapy for addressing the secondary consequences of DMD.

Capricor is also set to present the latest 36-month data from its HOPE-2 open-label extension (OLE) study at the upcoming Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference, scheduled from June 27-29, 2024. The data includes insights into both skeletal and cardiac aspects of the treatment.

Duchenne Muscular Dystrophy is a severe genetic condition characterized by progressive muscle weakness and chronic inflammation affecting skeletal, cardiac, and respiratory muscles. The disorder typically results in mortality around the median age of 30. DMD affects approximately one in every 3,500 male births, with an estimated 15,000 to 20,000 patients in the United States. The disease is caused by a deficiency in the production of functional dystrophin, a structural protein essential for muscle function. This deficiency leads to significant muscle cell damage, death, and fibrotic replacement. Currently, treatment options are limited, and there is no cure.

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is committed to pioneering the development of transformative cell and exosome-based therapies to redefine treatments for rare diseases. The company’s leading product candidate, deramiocel (CAP-1002), is an allogeneic cardiac-derived cell therapy. Preclinical and clinical studies have demonstrated its immunomodulatory, antifibrotic, and regenerative properties, particularly suited for dystrophinopathies and heart disease. Deramiocel is currently in Phase 3 clinical development for treating DMD.

Additionally, Capricor is leveraging its proprietary StealthX™ platform in preclinical development. This platform focuses on various applications, including vaccinology and targeted delivery of oligonucleotides, proteins, and small molecule therapeutics, aiming to treat and prevent a wide range of diseases. The company remains dedicated to exploring the potential of these therapies to bring transformative treatments to those in need.

Capricor has an exclusive agreement with Nippon Shinyaku Co., Ltd. for the commercialization and distribution of deramiocel in the United States and Japan, pending regulatory approval. Currently, deramiocel is an Investigational New Drug and has not been approved for any indications. Similarly, none of Capricor’s exosome-based candidates have received approval for clinical investigation.