Capricor Initiates Late-Stage Neuropsychiatric Disorder Study

Capricor Therapeutics CAPR initiated dosing of first patient in HOPE-3, a Phase 3 clinical trial investigating CAP-1002 for treating late-stage Duchenne muscular dystrophy (DMD).

HOPE-3 study is designed to enroll approximately 70 patients in the United States. The participants will be randomized to either CAP-1002 or placebo in a 1:1 ratio.

The primary outcome measure of this Phase 3 study will be the Performance of the Upper Limb (PUL) 2.0, a validated tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist and hand) function, with a conceptual framework reflecting weakness progression in upper limb function.

Linda Marbán, Ph.D., CEO, said, “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.”

The FDA has previously granted Regenerative Medicine Advanced Therapy Designation (RMAT) as well as Orphan Drug Designation (ODD) to CAP-1002.

The company is eligible to receive a Priority Review Voucher in its regulatory pathway for CAP-1002 based on the designation as a rare pediatric disease.

Price Action : Capricor shares are trading around 5 percent higher at $4.58 on Tuesday at the time of publication.