Capricor, Nippon Extend Partnership

LA JOLLA – Torrey Pines biotech innovator Capricor Therapeutics (Nasdaq: CAPR) signed a massive new deal with Japanese biopharma leader Nippon Shinyaku (TYO: 4516) for the European commercialization and distribution of Capricor’s leading drug – deramiocel – for the treatment of Duchenne muscular dystrophy (DMD), a debilitating genetic disorder that causes progressive muscle degeneration and weakness.

The deal – signed in a binding term sheet – has Nippon handing Capricor a $20 million upfront payment and a $15 million equity investment at a 20% premium. Capricor also has the potential to earn up to $715 million in potential milestone payments and a double-digit percentage of product revenue. The investment will extend Capricor’s cash runway into 2026.

“We plan on using the capital primarily for pre-commercial and commercial-related activities as we prepare to bring deramiocel to market for the treatment of patients with DMD, which is a rare neuromuscular disease with currently no cure available to patients,” Capricor CEO Dr. Linda Marbán told the Business Journal. “We have made tremendous progress over the last several years.”

The agreement is expected to close in Q4 of 2024. Under its terms, Capricor will be responsible for developing and manufacturing the drug for potential approval in the EU, U.K., and other regional countries. Nippon Shinyaku will oversee the sales and distribution of the drug in those territories. Capricor shared that it’s already in the process of applying for orphan and pediatric designations in Europe, paving the way for regulatory guidance.

It’s still working on establishing a timeline for approval but plans to meet with the European Medicines Agency (EMA) next year to determine next steps.

This deal further strengthens the partnership between the two companies, following an announcement in 2023 that Nippon agreed to pay more than $12 million, with additional potential milestones of up to $89 million, to Capricor for the exclusive commercialization and distribution of the drug in Japan. The year prior, the two companies inked a similar, but larger, deal for U.S. distribution, scoring Capricor a $30 million upfront payment with additional potential milestones of up to $705 million.

More Indications for Lead Asset

This September, the company revealed that it recently met with the FDA and intends to begin the process of filing a licensing application next month for the use of deramiocel in DMD patients with cardiomyopathy (a disorder of the heart muscle). A Phase 3 study is planned to seek approval for expanding the label use of the drug in patients with skeletal muscle myopathy.

In Nippon, Capricor has found an ideal partner for deramiocel. Nippon already launched its own DMD treatment in Japan, called Viltepso.

“We look forward to building deramiocel’s commercial footprint around the world and this partnership would allow us to continue to invest in Nippon Shinyaku’s DMD franchise and to potentially advance life-changing therapies for patients in need,” said Toru Nakai, president of Nippon Shinyaku.

DMD occurs in only one in every 3,600 live male births across all races, cultures and countries. It afflicts approximately 200,000 boys and young men around the world.

Treatment options are limited. 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.

Capricor’s DMD-fighting drug deramiocel is comprised of cardiosphere-derived cells (CDCs) that secrete vesicles known as exosomes that promote healing. “Deramiocel is a cell-based, off-the-shelf product that works by controlling inflammation, driving cellular repair of muscle and heart cells, supporting energy generation and promoting new blood vessel growth to support healthy muscle,” added Dr. Marbán. “We believe deramiocel can serve as the back-bone therapy for a large portion of patients with DMD due to its mechanism of action. There are other therapeutics that address portions of what we know of deramiocel’s mechanism of action, but there are currently no products out there that we are aware of that are immunomodulatory as well as anti-fibrotic.”

Capricor’s current enterprise value sits at $183.34 million (9/24/24). To date, the company has secured more than $200 million in funding, including collaborations, grants and awards from the National Institutes of Health, Department of Defense and California Institute for Regenerative Medicine.

“In five years, we believe Capricor can be a world-class biotechnology company with multiple products on the market for the treatment of a wide variety of diseases and a strong foothold in San Diego where we are based,” added Dr. Marbán.

Capricor Therapeutics
FOUNDED: 2006
CEO: Dr. Linda Marbán
HEADQUARTERS: La Jolla
STOCK: CAPR (NASDAQ)
ENTERPRISE VALUE: $183.34M (9/24/24)
EMPLOYEES: 125
WEBSITE: www.capricor.com
NOTABLE: Capricor studies have shown that its exosome technology is significantly active in several disease models.