Capricor sells European rights to DMD therapy for $35M as FDA application nears

Nippon Shinyaku has committed to a $20 million upfront payment for the rights to Capricor Therapeutics’ advanced Duchenne muscular dystrophy (DMD) therapy across Europe. In addition, Nippon Shinyaku will acquire approximately $15 million worth of Capricor’s stock.

Having already secured the U.S. rights to Capricor Therapeutics’ late-stage DMD therapy, Nippon Shinyaku, a Japanese pharmaceutical company, has agreed to a total of $35 million in cash and stock purchases to secure the European rights. Capricor is preparing to file for approval with the FDA for the drug, named deramiocel, including holding a pre-BLA (Biologics License Application) meeting with the regulator recently. Furthermore, the San Diego-based biotech revealed three-year data in June showing a 3.7-point improvement in upper limb performance compared to a similar patient group. This data highlighted the potential long-term benefits of the therapy for patients with this muscle degeneration disorder.

Nippon Shinyaku has been involved with deramiocel since 2022, when the company paid $30 million upfront to commercialize the drug in the U.S. Nippon Shinyaku also holds the rights to the drug in Japan. Now, the company based in Kyoto has agreed to a $20 million upfront payment for the rights in Europe and will purchase around $15 million of Capricor’s stock at a 20% premium to the stock’s 60-day volume-weighted average price. Furthermore, Capricor could receive up to $715 million in milestone payments and a double-digit share of regional revenues.

If the agreement is finalized, which is anticipated to occur later this year, Nippon Shinyaku will obtain the rights to sell and distribute deramiocel across the European Union, the U.K., and several other countries in the region, as detailed by Capricor in a release dated September 17.

“With the addition of the upfront payment and equity investment, we will be able to extend our runway into 2026 and be well positioned to advance toward potential approval of deramiocel in the United States and beyond,” said Capricor’s CEO, Linda Marbán, Ph.D., in the release. “Furthermore, these funds will provide necessary capital for commercial launch preparations, manufacturing scale-up and product development for Europe, as we envision high global demand for deramiocel,” she added.

Since the pre-BLA meeting with the FDA in August, Capricor has held informal discussions with the regulator to continue refining their approval pathway in the U.S., Marbán explained.

Earlier this summer, Pfizer abandoned its own DMD plans after its gene therapy fordadistrogene movaparvovec failed a phase 3 trial. This left Sarepta Therapeutics as the primary player in the field, with the biotech securing approval for a second DMD candidate last year, a gene therapy called Elevidys, in partnership with Roche.

Deramiocel is distinct from gene therapies. It consists of allogeneic cardiosphere-derived cells, a type of stromal cell that, according to Capricor, has demonstrated potent immunomodulatory, antifibrotic, and regenerative effects in dystrophinopathy and heart failure.