After showing its cell-based therapy slowed disease progression in Duchenne muscular dystrophy last September, Capricor Therapeutics is now ready to take the drug into a phase 3 trial with $30 million upfront from Nippon Shinyaku to help out.
San Diego-based Capricor will receive up to $705 million in milestones in exchange for Nippon distributing and commercializing the drug in the U.S., should it gain approval. Capricor is also actively seeking out partners for the treatment in Europe and Asia, said Linda Marbán, Ph.D., CEO and president, in an interview.
The news sent Capricor's shares up about 10% to $3.12 apiece as of 9:39 a.m. Eastern time Tuesday.
The Japanese pharma is no stranger to the rare muscle degeneration disease having gained FDA approval for Viltepso in August 2020, setting it up to compete with Sarepta's Vyondys 53 in the same patient population.
That market experience alongside a rare disease focus and appetite for new pipeline assets were three key attributes Capricor looked for when shopping around for partners, Marbán said.
RELATED: Capricor's DMD therapy slows disease progression in older patients, securing move to phase 3
Capricor will handle the pivotal trial for the cell therapy, dubbed CAP-1002, in which the biotech will look at improving upper limb strength and cardiac function in approximately 70 patients, Marbán said. The company is initiating sites and will begin enrolling patients "as soon as we can" for the HOPE-3 study, the CEO promised. The treatment will be given every three months for a total of four doses, according to ClinicalTrials.gov.
Marbán is hoping that CAP-1002 can help tame potentially dangerous immune responses that occur in DMD patients, perhaps in conjunction with some of the new gene therapies being developed by the likes of Pfizer and Sarepta someday.
CAP-1002 takes from donated heart muscle with the goal of regenerating skeletal and cardiac muscle cells in boys and young men.
"Even if [the gene therapies] were successful, you would need a drug like CAP-1002 to modulate and mediate the profound inflammatory responses to Duchenne muscular dystrophy," the CEO said. "So, we knew this was going to be a product that would have longevity in the space that was highly anticipated by the community and that payers would be interested in reimbursing for because of this strong bioactivity and also our good clinical trial data.”
RELATED: FDA approves NS Pharma's Duchenne drug on biomarker data, just like rival Sarepta
Gene therapies for DMD have raised a lot of hopes in the patient community, but the road through the clinic has been bumpy. Pfizer announced late last year that a patient in an early-stage trial of the gene therapy PF-06939926 had died. Sarepta is also working on gene therapies for DMD.
Pfizer delayed a data readout on its gene therapy from 2022 to the first quarter of 2023. Meanwhile, Sarepta could ask the FDA as soon as next year to approve its fourth DMD treatment, a gene transfer therapy.
Back at Capricor, the biotech is also developing CAP-1002 for treatment of hospitalized patients with severe COVID-19 who are on supplemental oxygen. The company will report data from the 60-patient phase 2 Inspire trial by the end of this quarter, the CEO said.
After that, Capricor will work with the FDA on a development path, whether that's a pivotal trial or filing for emergency use authorization, Marbán said.