Dosing Begins in HOPE-3 Study of Cell Therapy CAP-1002 for DMD

Previous trial shows investigational therapy can slow loss of function

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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The first patient has been dosed in a pivotal U.S. Phase 3 study evaluating the investigational cell therapy CAP-1002 in males with advanced Duchenne muscular dystrophy (DMD).

The trial, called HOPE-3 (NCT05126758), will evaluate the effect of CAP-1002 in upper limb function, among other assessments, in about 70 males, 10 and older, with DMD.

The study is currently enrolling participants at sites in Georgia and Arkansas, with one in California yet to begin recruiting.

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HOPE-3 comes on the heels of promising data from HOPE-2 (NCT03406780) and its open-label extension study. In HOPE-2, CAP-1002 improved arm and heart function compared to a placebo, meeting primary and secondary outcome measures. In the extension study, the cell therapy continued to improve arm function after patients resumed treatment after a break of about one year.

“We are delighted to begin dosing patients in HOPE-3,” Linda Marbán, PhD, CEO of Capricor, said in a press release. “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,” Marbán added.

CAP-1002 contains cardiosphere-derived cells, a type of heart cell progenitor, collected from healthy donors (allogeneic). These cells secrete exosomes, tiny vesicles filled with key information with the potential to modulate immune system activity and stimulate cellular regeneration.

In HOPE-3, participants will be randomly assigned to a placebo or CAP-1002 administered into the bloodstream every three months for a total of four doses. Each infusion of CAP-1002 contains 150 million cardiosphere-derived cells.

The primary goal is to evaluate the effect of CAP-1002 on functionality in the shoulders, elbows, wrists, and hands, using the Performance of the Upper Limb 1.2 test. Secondary goals including cardiac function assessments.

“Beginning this clinical trial is a significant milestone, not only for Capricor, but most importantly for those boys and young men with DMD,” said Marbán.

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CAP-1002 has been granted the designations of orphan drug, regenerative medicine advanced therapy, and rare pediatric disease in the U.S.

HOPE-3 is supported by funds from a recently established partnership between Capricor and Nippon Shinyaku, which holds the commercialization and distribution rights in the U.S.

The partnership also secures potential milestone-based payments to support the clinical development of CAP-1002 in DMD. Capricor holds the worldwide commercial rights to the therapy outside the U.S.