Having already scooped up the U.S. rights to Capricor Therapeutics’ late-stage Duchenne muscular dystrophy (DMD) therapy, Japan’s Nippon Shinyaku has signed off on $35 million in cash and a stock purchase to secure the same deal in Europe.
Capricor has been gearing up to make an approval filing to the FDA for the drug, called deramiocel, including holding a pre-BLA meeting with the regulator last month. The San Diego-based biotech also unveiled three-year data in June that showed a 3.7-point improvement in upper limb performance when compared to a data set of similar DMD patients, which the company said at the time “underscores the potential long-term benefits this therapy can offer” to patients with the muscle degeneration disorder.
Nippon has been on board the deramiocel train since 2022, when the Japanese pharma paid $30 million upfront for the rights to commercialize the drug in the U.S. Nippon also has the rights in Japan.
Now, the Kyoto-based company has agreed to a $20 million upfront payment for the rights across Europe, as well as buying around $15 million of Capricor’s stock at a 20% premium to the stock’s 60-day volume-weighted average price. Capricor could also be in line for up to $715 million in milestone payments as well as a double-digit share of regional revenues.
If the deal is finalized—which is expected to occur later this year—it would give Nippon the rights to sell and distribute deramiocel across the EU as well as in the U.K. and “several other countries in the region,” Capricor explained in a Sept. 17 release.
“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,” Capricor’s CEO Linda Marbán, Ph.D., said 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,” Marbán added.
Since August’s pre-BLA meeting with FDA, the biotech has held informal meetings with the regulator “to continue to refine our approval pathway” in the U.S., Marbán explained.
Pfizer axed its own DMD plans this summer after its gene therapy fordadistrogene movaparvovec failed a phase 3 trial. It left Sarepta Therapeutics as the only game in town—the biotech secured approval for a second DMD candidate last year in the form of the Roche-partnered gene therapy Elevidys.
Deramiocel is not a gene therapy. Instead, the asset consists of allogeneic cardiosphere-derived cells, a type of stromal cell that Capricor said has been shown to “exert potent immunomodulatory, antifibrotic and regenerative actions in dystrophinopathy and heart failure.”