Capricor Therapeutics and Nippon Shinyaku (US subsidiary: NS Pharma) have announced that they have entered into an agreement for the exclusive commercialization and distribution in the United States of CAP-1002, Capricor’s novel cell therapy candidate for Duchenne.
Under the terms of the agreement, Capricor will be responsible for the conduct of HOPE-3, a Phase 3 clinical trial of CAP-1002 in Duchenne, as well as the manufacturing of CAP-1002. Nippon Shinyaku will be responsible for the distribution of CAP-1002 in the United States.
We look forward to learning more as this partnership moves forward.
Read the Announcement from Capricor and Nippon Shinyaku
Capricor Therapeutics and Nippon Shinyaku Enter Partnership for Exclusive Commercialization and Distribution of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy in the U.S.
SAN DIEGO, Jan. 25, 2022 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ: CAPR) (“Capricor” or “the Company”), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases, announced today that it has entered into a partnership with Nippon Shinyaku Co., Ltd., a Japanese pharmaceutical company listed on the TYO (US subsidiary: NS Pharma), for the exclusive commercialization and distribution in the United States of Capricor’s lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD), a rare neuromuscular disease with limited treatment options.
Capricor’s proprietary cell therapy, CAP-1002, is comprised of human allogeneic cardiosphere-derived cells, which have demonstrated positive results in patients with DMD. CAP-1002’s mechanism of action is immunomodulatory and regenerative and its broad applicability makes it suitable for patients regardless of genetic mutation. HOPE-Duchenne and HOPE-2, the Phase 1 and Phase 2 clinical trials using CAP-1002 to treat late-stage DMD patients, showed statistically significant improvements in upper limb and/or cardiac function in the treatment groups. HOPE-3, the Phase 3 clinical trial that will be supported by this partnership, will commence shortly and is expected to be the pivotal trial for CAP-1002 in DMD. The regulatory pathway for CAP-1002 is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) as well as Orphan Drug Designation. If Capricor were to receive market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher based on its designation as a rare pediatric disease.
Under the terms of the agreement, Capricor will be responsible for the conduct of HOPE-3 as well as the manufacturing of CAP-1002. Nippon Shinyaku will be responsible for the distribution of CAP-1002 in the United States. Capricor will sell commercial product to Nippon Shinyaku and in addition will receive a meaningful, double-digit share of product revenue and additional development and sales-based milestone payments. Capricor will receive an upfront payment of $30 million with potential additional milestone payments of up to $705 million.
“The partnership with Nippon Shinyaku aligns us with a larger, seasoned pharmaceutical company experienced in rare disease with specific expertise in DMD. Nippon Shinyaku recently launched Viltepso, an exon skipping agent for the treatment of DMD and has a fully assembled U.S. team to support a broad commercialization effort. The addition of non-equity capital from this transaction provides the requisite funding for the execution of HOPE-3 without dilution to Capricor shareholders. Further, the structure of the agreement allows us to leverage our expertise in manufacturing CAP-1002 and to have a very meaningful share of future product revenues. Our initial planned indication is for late-stage DMD patients with more advanced disease. Presently, this comprises approximately half of all DMD patients. Indication expansion to younger boys is something we hope to look at in the future as well as potential synergies with other developing therapies including gene therapy and oligonucleotides” said Dr. Linda Marbán, CEO of Capricor.
“At Nippon Shinyaku, we believe in the potential of CAP-1002 to address the severe unmet need for boys and young men with Duchenne muscular dystrophy,” said Toru Nakai, President of Nippon Shinyaku. “As we have an established commercial program for DMD in the USA, working with Capricor to bring CAP-1002 to patients presents the opportunity for a strong partnership for both companies. As CAP-1002 moves towards potential commercialization, the overarching goal of both companies is to develop novel, life changing therapies for DMD and, most importantly, give hope to patients in need.”