The U.S. Food and Drug Administration has approved Capricor Therapeutics’ plan for further development of its CAP-1002 stem cell therapy for Duchenne muscular dystrophy, paving the way for another Phase 2 clinical trial of the treatment this year.
The thumbs-up came when Capricor received official minutes from a meeting with the FDA that included the company’s plan to file an investigational new drug application for CAP-1002. The treatment involves repeated intravenous injections of CAP-1002, which consists of heart stem cells that researchers believe can help regulate immune responses.
“The FDA’s response to our proposed clinical development plan supports our near-term objective of submitting an IND [investigational new drug application] for intravenous CAP-1002 as well as provides us with clarity on a path to potential product registration,” Dr. Linda Marbán, president and CEO of Capricor, said in a press release.
“We look forward to commencing a randomized, double-blind, placebo-controlled Phase 2 clinical trial of intravenous, repeat-dose CAP-1002 in boys and young men with DMD in the second half of 2017,” she added.
Capricor is already assessing the therapy in a Phase 1/2 trial (NCT02485938) of Duchenne patients with damaged heart muscles, also known as cardiomyopathy. But the study is delivering the cells directly to patients’ heart arteries as a one-time treatment, rather than intravenously. That’s why the new trial will cover intravenous administration.
The FDA has also agreed that Capricor can use a different main measure of CAP-1002’s effectiveness in the next trial. Called Performance of the Upper Limb, it assesses the arm function of Duchenne patients — both those who can and can’t walk.
The current trial, called HOPE, covers 25 children and adults with Duchenne MD who were randomly assigned to receive either CAP-1002 or a standard of care therapy.
Interim results, which Capricor shared in April, showed that patients improved both arm and heart function in the six months following treatment.
CAP-1002 is made up of donated cardiosphere-derived stem cells. Research has shown that they release substances that may slow the muscle degeneration in Duchenne.
“The cells in CAP-1002 release” substances that not only regulate immune response but also “exert anti-inflammatory, anti-fibrotic, and anti-apoptotic [anti-cell-death] effects,” Marbán said.
By reducing muscle cell damage “induced by dystrophin [gene] mutations, our product has been demonstrated to preserve and improve the structure and function” of weakened muscle, she said. “Its differentiated mechanism of action supports its potential to be a stand-alone therapy as well as an adjunct” to treatments that regulate the dystrophin protein.