FDA Paves Way for Capricor to Hold Phase 2 Trial of CAP-1002 for Duchenne MD

FDA Paves Way for Capricor to Hold Phase 2 Trial of CAP-1002 for Duchenne MD

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.

Magdalena is a writer with a passion for bridging the gap between the people performing research, and those who want or need to understand it. She writes about medical science and drug discovery. She holds an MS in Pharmaceutical Bioscience and a PhD — spanning the fields of psychiatry, immunology, and neuropharmacology — from Karolinska Institutet in Sweden.
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Magdalena is a writer with a passion for bridging the gap between the people performing research, and those who want or need to understand it. She writes about medical science and drug discovery. She holds an MS in Pharmaceutical Bioscience and a PhD — spanning the fields of psychiatry, immunology, and neuropharmacology — from Karolinska Institutet in Sweden.

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