Capricor Therapeutics and Nippon Shinyaku Form Partnership for CAP-1002 Distribution in Japan, Treating Duchenne Muscular Dystrophy

Capricor Therapeutics, a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, announced that it has entered into a partnership with Nippon Shinyaku Co., Ltd., a Japanese pharmaceutical company listed on the TYO, for the exclusive commercialization and distribution in Japan of Capricor’s lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD), a rare neuromuscular disease with limited treatment options. This follows the exclusive Commercialization and Distribution Agreement entered into with Nippon Shinyaku in the United States in January 2022.

“Nippon Shinyaku is a proven leader in developing therapeutics for rare diseases, specifically DMD, and an ideal partner for us to maximize the opportunity with CAP-1002 in Japan and the U.S.,” said Dr. Linda Marbán, CEO of Capricor. “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. 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. Furthermore, we remain committed to securing strategic partners that will further strengthen our balance sheet and help us achieve our goal of bringing our therapies to the global patient community as quickly as possible.”

Under the terms of the agreement, Capricor will receive an upfront payment of $12 million and in addition, Capricor will potentially receive additional development and sales-based milestone payments of up to approximately $89 million and a meaningful, 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.

Toru Nakai, President of Nippon Shinyaku, commented, “We look forward to further strengthening our partnership with Capricor as CAP-1002 moves towards potential commercialization. This agreement provides an opportunity to expand Nippon Shinyaku’s DMD franchise and to advance potentially life-changing therapies for patients with Duchenne Muscular Dystrophy. In Japan, we already have launched Viltepso, an exon skipping agent for the treatment of DMD and will leverage our established commercial infrastructure to deliver on our shared mission of bringing hope to more patients with the addition of CAP-1002 to our pipeline.”