Capricor Therapeutics (NASDA: CAPR) and Nippon Shinyaku Co. Ltd. have signed an exclusive distribution deal for CAP-1002 for Duchenne muscular dystrophy (DMD) treatment in the US.
CAP-1002 can enhance muscular and cardiac performance
DMD is degenerative muscular dystrophy induced by a dystrophin protein shortage, which causes cardiac, pulmonary, and skeletal muscle weakening. The disease has a wide range of genetic variations, and there are several medicines advanced that help meets the unmet medical requirements. Interestingly, the treatment for DMD with different types of genetic mutations and treatment for older, non-ambulant DMD individuals remains inadequate, and more effective medicines are needed.
Human allogeneic cardiosphere-derived cells make up CAP-1002.CAP-1002’s mechanism of action is thought to be exosomes, which have been found to lower oxidative stress, fibrosis, inflammation, and boost myocyte production, as well as enhance muscular and cardiac performance. The product candidate is likely to be helpful in a wide spectrum of DMD patients, irrespective of the kind of gene mutation, and a second Phase clinical trial found that non-ambulatory DMD subjects had better upper limb mobility and heart function. The FDA has designated CAP-1002 as an orphan medication and an RMAT, and Capricor is currently preparing to begin the third phase clinical trial. CAP-1002 will be commercialized by the company’s wholly-owned subsidiary NS Pharma Inc., after BLA approval in the United States.
Collaboration to enable Nippon Shinyaku to bring Viltepso to patients
Nippon Shinyaku focuses on intractable, rare diseases, and its in-house created DMD therapy, Viltepso® (an antisense exon-skipping drug), has been marketed in the United States. Most importantly, Nippon Shinyaku hopes to contribute even more to the treatment of DMD patients by completing this collaboration with Capricor.
VILTEPSO is used to treat DMD in people who have a verified DMD genetic mutation that can be treated by exon 53 skipping. Depending on an increase in dystrophin generation in skeletal muscle reported in patients receiving VILTEPSO, the indication has been granted under accelerated approval. However, ongoing authorization for the indication could be conditional on a confirmatory trial demonstrating and documenting clinical benefit.
