RARE Daily

Capricor and Nippon Shinyaku Partner to Commercialize and Distribute Treatment for DMD in Japan

February 16, 2023

Rare Daily Staff

Capricor Therapeutics entered into a partnership with Nippon Shinyaku for the exclusive commercialization and distribution in Japan of Capricor’s lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy.

This follows the exclusive commercialization and distribution agreement between the two companies for the United States, signed in January 2022.

Under the terms of the agreement, Capricor will receive an upfront payment of $12 million and will potentially receive additional development and sales-based milestone payments of up to approximately $89 million and a double-digit share of product revenue. This agreement is similar to the terms of the U.S. agreement with Nippon Shinyaku, in that Capricor will be responsible for clinical development and Nippon Shinyaku will be responsible for the distribution of CAP-1002 in Japan, once approved. Capricor will sell commercial product to Nippon Shinyaku. In addition, Capricor will hold the marketing authorization in Japan, if the product is approved in that territory.

“With the addition of non-equity capital from this transaction, we are well positioned to advance and execute on our milestones including the execution of the HOPE-3 Phase 3 trial in the United States,” said Linda Marbán, CEO of Capricor. “CAP-1002 has shown clinical benefits for both the cardiac and skeletal muscle myopathy, which few therapies have demonstrated. The data from our recently announced 18-month HOPE-2 open label extension study showed evidence of disease modification and showed statistically significant differences in the Performance of the Upper Limb (PUL). This continues to build upon CAP-1002’s safety and efficacy profile and to potentially establish it as an anchor therapy for DMD patients.”

Duchenne muscular dystrophy is a genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart, and respiratory muscles. Patients suffering from DMD typically lose their ability to walk in their teenage years and generally die of cardiac or respiratory complications by age 30. It occurs in one in every 3,600 live male births across all races, cultures, and countries. DMD afflicts approximately 200,000 boys and young men around the world. Treatment options are limited and there is no cure.

CAP-1002 consists of allogeneic cardiosphere-derived cells, a type of progenitor cell that has been shown in pre-clinical and clinical studies to exert potent immuno-modulatory activity and is being investigated for its potential to modify the immune system’s activity to encourage cellular regeneration.

Photo: Linda Marbán, CEO of Capricor

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