Phase 3 Cardiosphere-Derived Cell Therapy Trial Doses First Patient With DMD


Recent long-term data from the phase 2 extension has also demonstrated CAP-1002's efficacy in Duchenne.

Capricor Therapeutics has dosed the first patient with Duchenne muscular dystrophy (DMD) in its phase 3 HOPE-3 study (NCT05126758) of CAP-1002, an allogeneic cardiosphere-derived cell therapy.1

“We are delighted to begin dosing patients in HOPE-3. The data from our phase 2 clinical trial suggest that CAP-1002 can slow loss of function by as much as 70% in terms of upper limb skeletal muscle function. Since there are very limited therapeutic options for these patients and CAP-1002 has been shown to be safe and effective, we are pleased to begin this pivotal trial with the goal of achieving regulatory approval as quickly as possible,” Linda Marbán, PhD, chief executvie officer, Capricor, said in a statement.1 “Beginning this clinical trial is a significant milestone, not only for Capricor, but most importantly for those boys and young men with DMD.”

HOPE-3 plans to enroll up to 58 participants with DMD randomized to receive either 150 million CAP-1002 cells in 4 doses every 3 months or placebo in a 1:1 ratio. The study is primarily evaluating Performance of the Upper Limb (PUL) 2.0. Secondary endpoints include cardiac function assessments. CAP-1002 has previously been granted regenerative medicine advanced therapy and orphan drug designations.

Capricor also recently released data from the phase 2 HOPE-2 study open-label extension (OLE; NCT04428476) that showed statistically significant clinical benefits in non-ambulatory patients with DMD. The trial met its primary endpoint of improvement on PUL 2.0 (P = .02) The OLE evaluated 12 patients out of the original 20 from the HOPE-2 trial (NCT05126758).

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“For patients with DMD, time is associated with loss of function. The progressive nature of the disease puts these patients on a slow, steady decline. Anything that we can do to delay the progression is an essential step in the right direction, allowing for patients to preserve key everyday activities requiring upper limb function,” national principal investigator Craig McDonald, chair, Department of Physical Medicine & Rehabilitation and professor, Department of Pediatrics and Physical Medicine & Rehabilitation, UC Davis Health, said in a previous statement.2 “The initial HOPE-2 data, augmented with this new body of evidence showing slowing in the rate of decline with re-introduction of systemic cell therapy, indicate that CAP-1002, when used over time, is slowing DMD’s devastating effects and may be disease modifying.”

“In the HOPE-2 trial we focused on the non-ambulatory population with Duchenne, which has largely been ignored in previous clinical trials. Most clinical trials in Duchenne are focused on the ambulatory population and these patients have largely been at the back of the bus as far as some of the more innovative therapeutics go,” McDonald told CGTLive in a recent interview.

CAP-1002 is also being assessed in the phase 2 INSPIRE trial (NCT04623671) in hospitalized patients with severe COVID-19.3 Data released from the trial in April 2022 showed that the therapy was well-tolerated in this population, but Capricor stated that they would evaluate next steps for the therapy considering the decline of COVID-19 hospitalizations and mortality and the widespread availability of vaccines.

1. 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. Assessed July 21, 2022.
2. 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.
3. Capricor Therapeutics reports topline results from phase 2 exploratory INSPIRE trial in severe hospitalized COVID-19 patients. News release. Capricor Therapeutics. March 28, 2022. Assessed July 21, 2022.
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