RARE Daily

Capricor and Nippon Shinyaku Partner to Commercialize and Distribute CAP-1002 to Treat DMD in U.S.

January 25, 2022

Capricor Therapeutics has granted Japanese biopharma Nippon Shinyaku giving it exclusive rights to commercialize and distribute its lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD), a rare neuromuscular disease with limited treatment options.

Photo: Linda Marbán, CEO of Capricor

Under the terms of their agreement, Capricor will be responsible for the conduct of the phase 3 clinical trial, 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 Duchenne muscular dystrophy,” said Linda Marbán, CEO of Capricor. “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.”

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.

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 Regenerative Medicine Advanced Therapy as well as Orphan Drug designations. If Capricor were to receive U.S. regulatory approval for CAP-1002, Capricor would be eligible to receive a Priority Review voucher based on its designation as a rare pediatric disease.

Marbán said that the addition of non-equity capital from this transaction provides the requisite funding for the execution of HOPE-3 without dilution to Capricor shareholders. The structure of the agreement allows Capricor to leverage its expertise in manufacturing CAP-1002 and to have a meaningful share of future product revenues.

The initial planned indication for CAP-1002 is late-stage DMD patients with more advanced disease. This comprises approximately half of all DMD patients. The company hopes to expand indications to include younger boys in the future, as well as exploring potential synergies with other developing therapies including gene therapy and oligonucleotides.

Author: Rare Daily Staff

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