Application finalized for DMD cell therapy CAP-1002, now deramiocel
Capricor seeks priority review for heart muscle disease cell therapy
Capricor Therapeutics has completed its submission of a biologics license application (BLA) seeking U.S. approval of deramiocel, the company’s cell therapy for heart muscle disease in people with Duchenne muscular dystrophy (DMD).
“The submission of the BLA marks a pivotal step for Capricor and those impacted by DMD,” Linda Marbán, PhD, CEO of Capricor, said in a company press release.
Capricor started its rolling BLA a few months ago. The company is asking the U.S. Food and Drug Administration (FDA) to give the application priority review, which could reduce the review time from 10 months to six months.
“This BLA is the culmination of a body of work that has been focused on bringing this potentially transformational therapy to those patients in need,” Marbán said.
DMD is caused by mutations that lead to an absence of the protein dystrophin, which normally acts like a shock absorber in muscle cells to protect muscles from damage. The lack of dystrophin causes muscle cells to accumulate more damage over time, driving disease symptoms.
Along with affecting the skeletal muscles used to move the body around, DMD also impacts the heart, which can lead to cardiomyopathy, where the heart muscle weakens over time, impairing its ability to pump blood effectively.
Deramiocel, which was previously called CAP-1002, contains immature heart cells, called cardiosphere-derived cells, that are derived from a healthy donor. These cells secrete signaling molecules that help promote muscle regeneration, prevent scar tissue from forming, and modulate the immune system.
Results of HOPE-2 study
Capricor’s BLA is based primarily on data from the Phase 2 HOPE-2 trial (NCT03406780) and its open-label extension (NCT04428476).
HOPE-2 enrolled boys and young men with relatively advanced DMD, most of whom were unable to walk. The participants were randomly assigned to receive infusions of deramiocel or a placebo every three months for a year. The cell therapy led to improvements in heart function and arm function relative to the placebo, results showed.
After HOPE-2, all the participants stopped deramiocel treatment for more than a year and then most entered the ongoing open-label extension study where they continue to receive it. Three-year data from the extension suggest deramiocel led to improved heart and arm function relative to what would be expected without treatment.
“We believe that the strength of this application is that deramiocel has shown in multiple clinical trials attenuation of the cardiac implications of DMD,” Marbán said. “We look forward to working with the FDA throughout the review process to support this potential approval.”
Capricor is also conducting HOPE-3 (NCT05126758) to further test deramiocel. Participants 10 and older will be randomly assigned to receive the cell therapy or a placebo every three months for a year, with the main goal being to evaluate how the treatment affects arm function. HOPE-3 is taking place at multiple sites across the U.S.
The FDA has previously granted deramiocel regenerative medicine advanced therapy status, which is intended to accelerate the development of treatments for serious conditions. It has also been granted orphan drug and rare pediatric disease designations, which provide extra incentives for companies investing in rare disease treatments.
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