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Approximately 20,000 children globally are diagnosed with DMD each year, the majority of which are male. The disease results in muscle degeneration and eventually leads to premature death, with the majority of patient lifespans lasting less than three decades. The cause of DMD is a gene mutation that results in a lack of functional dystrophin protein, an essential structural component of healthy muscle cells. The lack of this dystrophin protein causes muscle cells to progressively die as they are highly susceptible to damage. This is especially problematic in the muscle cells of the human heart. DMD patients accumulate scar tissue in the heart as their muscle cells progressively die off, which eventually leads to heart failure, the leading cause of death among DMD patients.
There is currently no cure for DMD and patients are subject to substantial healthcare costs. In fact, annual healthcare costs for DMD patients are approximately 10 times higher than for people without DMD in the United States. The standard line of treatment for DMD patients includes disease management therapies like mobility and breathing assistance, creatine supplements, and steroids, according to the United Kingdom National Health Service. Capricor’s late-stage product candidate is a cellular medicine that aims to provide a superior therapy option for DMD patients. The company also has a range of innovative cell-targeting therapies in preclinical development.
CAP-1002 is a cell therapy that consists of cardiosphere-derived cells (CDCs), which are stromal cells that are isolated from the healthy human hearts of donors. Stromal cells are connective tissues that make up the infrastructure of organs and support healthy bodily functions. Administering CDCs into the human body results in exosomes that contain bioactive cargo to be secreted. Included in those bioactive cargoes are microRNAs. These RNAs alter gene expression in targeted cells, which serves to alleviate inflammation and stimulate tissue regeneration in inflammatory diseases, including DMD.
CAP-1002 is currently undergoing phase 3 clinical trials. The product candidate has received the Regenerative Medicine Advanced Therapy (“RMAT”), orphan drug, and Rare Pediatric Disease designations from the FDA, which help to support the therapy’s pathway toward commercial approval. CAP-1002 clinical trials have generated positive results so far and the company appears on track to submit a BLA application within the next few years.
CAP-2003 is a therapeutic product candidate derived from exosomes found in CDCs. Exosomes mediate intercellular communication by carrying essential cellular materials like nucleic acids and proteins. Preclinical testing of CAP-2003 within Capricor labs and those of collaborating entities have generated promising results. After filing an IND with the FDA in 2020 to test the application of CAP-2003 in DMD patients, the FDA requested additional information on the manufacturing procedures of the product candidate. However, Capricor has yet to submit this information.
Capricor has developed a precision-engineered exosome platform technology, StealthX. This proprietary platform has rapidly produced two Covid 19 vaccine candidates that each target a different protein. When tested in animal modeling systems, the combination of the two vaccines showed strong results, like persistent antibody production. Capricor believes StealthX has the potential to rapidly develop a range of vaccines, similar to mRNA vaccine production, and is planning to expand the testing of vaccines within the platform.
Capricor has no FDA products approved for commercial sale and so has yet to generate any product revenue. Furthermore, the company is still several years away from potentially commercializing any product candidates currently in development. Thus far, Capricor has primarily financed operations through public equity sales, government grants, and payments from collaboration agreements with a Japanese company, Nippon Shinyaku. These collaboration agreements give Nippon Shinyaku the distribution rights to CAP-1002 in both the United States and Japan. Despite some revenue-generating functions, Capricor will require substantial additional funding in order to complete the development of product candidates and may seek that funding in the form of further equity financing, debt, or partnership agreements.
Capricor Therapeutics is on track to potentially provide an improved treatment option for the many children and young adults suffering from DMD. The company has its own manufacturing capabilities in place and a strong distribution partner in Nippon Shinyaku to support this initiative in two major markets. Furthermore, Capricor’s CDCs and exosome-based therapies are cutting-edge technologies that could positively affect human longevity beyond just the treatment of DMD, which is why leading institutions like John Hopkins University, US Department of Defence, US Army Institute of Surgical Research, and the National Institutes of Health are collaborating with the company to research the potential applications of this technology. Although the company has substantial hurdles to overcome before any of its products reach the market, Capricor has enough positive elements in place to intrigue longevity enthusiasts.