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Craig M. McDonald, MD, on Safety and Efficacy of DMD Cell Therapy

The principal investigator of the HOPE-2 trial discussed safety and efficacy of Capricor’s CAP-1002.

“We think that the therapy is going to preserve critical arm function that's important to maintain activities of daily living, such as feeding, dressing, scratching the nose, or even hugging a parent... those would be enhanced by this therapeutic.”

Capricor Therapeutics’ CAP-1002, an allogeneic cardiosphere-derived cell therapy, has yielded statistically significant clinical benefits in non-ambulatory patients with Duchenne muscular dystrophy (DMD).1 These newly released data, from the phase 2 HOPE-2 trial open-label extension (OLE; NCT04428476), showed that the trial met its primary endpoint of improvement on Performance of the Upper Limb 2.0 (P = .02) The OLE included 12 patients out of the original 20 from the HOPE-2 trial (NCT05126758). CAP-1002 is also being evaluated in the phase 3 HOPE-3 trial (NCT05126758), in which the first patient with late-stage DMD was recently dosed.2

CGTLive spoke with Craig M. McDonald, MD, chair, Department of Physical Medicine & Rehabilitation and professor, Department of Pediatrics and Physical Medicine & Rehabilitation, UC Davis Health, to learn more about the efficacy and safety data seen in the HOPE-2 trial. He also discussed nest steps for CAP-1002, including the current HOPE-3 trial.

1. Capricor Therapeutics announces statistically significant clinical benefits in skeletal muscle function in non-ambulant Duchenne muscular dystrophy patients treated with CAP-1002 in HOPE-2 open label extension study. News release. Capricor Therapeutics. June 27, 2022. Accessed July 20, 2022. https://feeds.issuerdirect.com/news-release.html?newsid=7075005202651835.
2. Capricor Therapeutics announces first patient dosed in pivotal phase 3 study of CAP-1002 for the treatment of Duchenne muscular dystrophy. News release. Capricor Therapeutics. July 19, 2022. Accessed July 20, 2022. https://feeds.issuerdirect.com/news-release.html?newsid=7011515935297837