Capricor cell therapy improves cardiac and upper limb function in phase 2 Duchenne muscular dystrophy victory lap

Capricor Therapeutics is taking a victory lap for their phase 2 Duchenne muscular dystrophy (DMD) trial. At three years, the San Diego-based company’s cell therapy deramiocel improved patients’ left ventricular ejection fraction and ability to use their upper limbs.

“These results are extremely impactful for patients living with DMD as they showed sustained cardiac and skeletal muscle benefits after three years of continuous treatment with deramiocel,” Capricor CEO Linda Marbán, Ph.D., said in an Oct. 11 release. “This dataset will be one of the key elements of our biologics license application submission to the FDA for approval of deramiocel to treat patients with DMD cardiomyopathy.”

The extended data drop comes a few days after the biotech started a rolling submission process with the FDA seeking full approval for deramiocel in all patients with DMD cardiomyopathy. Capricor expects the submission to be complete by the end of this year. 

The new results were presented at the 29th Annual Congress of the World Muscle Society in Prague. The phase 2 HOPE-2-OLE trial enrolled 13 patients with a deramiocel infusion given every three months. Capricor had previously reported that the treatment met the trial’s main goal in 2021.

In a subgroup of patients without possible heart failure, deramiocel improved the volume of blood in the ventricle by 11.1 ml/m2 at two years compared to an external group of patients who didn’t receive the treatment. The cell therapy also slowed muscle deterioration, with patients receiving it showing a decline in an index of upper arm function of four points after three years compared to 7.7 in the external group, as measured by a 22-item scale evaluating several functional skills in people with DMD.

All 13 patients experienced a mild to moderate adverse event, with five also experiencing a severe or life-threatening event. Nine of the 13 events were related to the treatment, Capricor reported in the presentation.

Deramiocel is an allogeneic cell therapy of cardiosphere-derived cells, which are connective tissue cells from the heart. The cells secrete tiny cargo packets called exosomes, which target macrophages and alter their behavior so that they become anti-inflammatory and pro-tissue regeneration, the company said.

Capricor is now testing deramiocel in a phase 3 trial, HOPE-3, which plans to enroll up to 102 patients and is set to wrap up in December 2026. The firm had been working on an exosome-based COVID vaccine, using the technique as an mRNA-delivery vehicle, but scrapped those plans to focus on deramiocel in 2022.

In Jan. 2024, the jab made a comeback after it was selected by the U.S. Department of Health and Human Services for Project NextGen, an initiative to advance new COVID vaccines. As part of Project NextGen, the National Institute of Allergy and Infectious Diseases will conduct a phase 1 trial of Capricor’s vaccine, the company said in a release.