Capricor Inks $100M Deal with Nippon Shinyaku

Capricor Therapeutics is strengthening its partnership with Japanese pharmaceutical company Nippon Shinyaku Co., Ltd.

The San Diego-based Capricor (NASDAQ: CAPR) announced Feb. 16 it had entered into an agreement with Nippon Shinyaku, who is listed on the TYO, for the exclusive commercialization and distribution in Japan of Capricor’s lead asset CAP-1002 – a treatment for Duchenne muscular dystrophy (DMD), a rare neuromuscular disease with few treatment options.

The Japan distribution deal comes just over one year after the two companies inked a similar, but larger, deal for U.S. distribution.

“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 Linda Marbán, CEO of Capricor.

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 double-digit share of product revenue.

In both deals, 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.

Last year, the two companies entered a similar deal for U.S. distribution, where Capricor received a $30 million upfront payment with the potential to earn an additional $700 million based on milestones.

Ideal Partnership

In Nippon, Capricor has found an ideal partner for its CAP-1002 treatment for DMD. Nippon has already launched its own DMD treatment in Japan, an exon skipping agent named Viltepso. For CAP-1002, the company will be able to leverage its commercial infrastructure to deliver on a “shared mission of bringing hope to more patients,” said Nippon Shinyaku President Toru Nakai.

“We look forward to further strengthening our partnership with Capricor as CAP-1002 moves towards potential commercialization,” he said. “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.”

Positive Clinical Results

DMD is a rare disease, occurring 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.

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.

CAP-1002 is being investigated in clinical trials for its potential to modify the immune system’s activity to encourage cellular regeneration – specifically in heart and upper limb muscles, which are weakened as DMD progresses.

Data from Capricor’s recently announced 18-month HOPE-2 clinical trial and open label extension study “showed evidence of disease modification and showed statistically significant differences in the performance of the upper limb,” Marbán said. “This continues to build upon CAP-1002’s safety and efficacy profile and to potentially establish it as an anchor therapy for DMD patients.”

With the additional non-equity capital from the latest deal with Nippon, Capricor is “well positioned to execute” on its milestones, “including the execution of the HOPE-3 Phase 3 trial in the United States,” Marbán said. “CAP-1002 has shown clinical benefits for both the cardiac and skeletal muscle myopathy, which few therapies have demonstrated. 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.”

Capricor Therapeutics

Founded: 2006
CEO: Dr. Linda Marbán
Stock: CAPR (on NASDAQ)
Headquarters: La Jolla
Stock: CAPR (NASDAQ)
Employees: 40
Website:  www.capricor.com
Notable: Capricor studies have shown that its exosome technology is significantly active in several disease models.