Capricor Therapeutics will hold a phone meeting with the U.S. Food and Drug Administration (FDA) this month to discuss potential product registration strategies for CAP-1002, a cardiac cell therapy being developed to treat Duchenne muscular dystrophy (DMD).
CAP-1002 is produced from a healthy donor’s heart tissue, grown in a lab and stored until needed. Patients then receive the therapy,via infusion into one or more coronary arteries using regular cardiac catheterization methods.
The FDA meeting follows an April 25 presentation at which Capricor reported positive top-line six-month results from the randomized Phase 1/2 HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) clinical trial (NCT02485938).
That trial was an open-label study designed to evaluate the safety and effectiveness of CAP-1002 in 25 participants, randomized to receive either infusions of CAP-1002 in three coronary arteries (active group) or to standard of care (control group). It reportedly showed that CAP-1002 was safe and well-tolerated over the six-month study. Patients who received the active treatment showed statistically meaningful improvement compared to care controls in measures of cardiac and upper limb function.
Capricor will present its DMD therapeutic development program at the Parent Project Muscular Dystrophy (PPMD) 2017 Annual Connect Conference, set for June 29-July 2 in Chicago.
Linda Marbán, Capricor’s president and CEO, said her company’s meeting with the FDA will cover both the HOPE results and future plans for CAP-1002.
“In collaboration with a distinguished group of scientific and clinical leaders, we are currently designing a randomized, double-blind, placebo-controlled clinical trial of intravenous, repeat-dose CAP-1002 in DMD, in which we expect to begin patient enrollment in the latter half of this year, subject to regulatory approval,” she said in a press release.
Added Pat Furlong, PPMD’s president and CEO: “Each year, nearly 500 families from around the world gather at our Connect Conference to learn the latest progress in the fight to end Duchenne. I’m delighted that the six-month results from the HOPE trial, which provide indication of CAP-1002’s therapeutic potential in a population with advanced Duchenne, will be featured at this premier community event.”