Capricor Therapeutics Issues Progress Report on Its Cardiac Cell Therapy CAP-1002 for DMD

Capricor Therapeutics recently shared an update of the company’s quarterly financial results and operational highlights, including positive results for the ongoing HOPE study with Duchenne muscular dystrophy (DMD) patients.

The Phase I/II HOPE study (NCT02485938) is assessing the safety and tolerability of investigational cardiac cell therapy CAP-1002 in boys and young men with DMD. Results from the trial’s first six months showed that patients treated with CAP-1002 experienced significant improvements in cardiac and upper limb functions compared with patients under standard care.

Researchers are assessing patient outcomes with lab tests, physical exams, and electrocardiograms, and by measuring coronary blood flow and major cardiac events. The study also will measure patient responses on the Performance of Upper Limb (PUL) scale, a tool developed by experts to assess upper limb function in DMD patients; with spirometry; and in a test that measures how far patients can walk in six minutes.

“In April, we reported positive interim results from our ongoing HOPE-Duchenne clinical trial which have ignited the interest of patients, advocates and thought leaders who constitute the greater Duchenne muscular dystrophy community. These results are particularly notable given the patients’ advanced disease as approximately 70% of trial participants were wheelchair-dependent at entry,” Linda Marbán, PhD, president and CEO of Capricor, said in a press release. 

“A key finding in HOPE-Duchenne was that, as compared to usual care controls, patients treated with CAP-1002 demonstrated improvement in skeletal muscle function according to the Performance of the Upper Limb (PUL) test, a validated instrument,” she added.

Trial data for the primary outcome is expected in November 2017, but the study will continue through November 2021.

Capricor also announced that the U.S. Food and Drug Administration (FDA) is reviewing a biologics license application (BLA) for CAP-1002 to treat DMD, and the PUL test is being evaluated for use in assessing patient outcomes. Earlier this year, the FDA granted CAP-1002 rare pediatric disease designation, which allows it to obtain priority review.

In the meantime, Capricor is planning a placebo-controlled Phase 2 clinical trial for intravenous, repeat-dose CAP-1002 in boys and young men with DMD, which is expected in the second half of 2017 following FDA approval earlier this year.

“Having now obtained clarity from the U.S. Food and Drug Administration (FDA) on a development plan that could potentially support a Biologics License Application (BLA) for CAP-1002, we are preparing to conduct our next clinical trial in DMD, which will evaluate the ability of intravenous CAP-1002 to improve skeletal muscle function as measured by the PUL,” Marbán said. “We expect this to commence in the fourth quarter of this year, subject to regulatory approval.”