Capricor Set to Launch Phase 2 Trial of Cell Therapy CAP-1002 in Advanced DMD Patients

Capricor Therapeutics has received a green light from the U.S. Food and Drug Administration to launch a Phase 2 study of CAP-1002 cell therapy in children and adults with advanced Duchenne muscular dystrophy.

Capricor expects to open the trial to patient enrollment in early 2018 and, if successful, to begin steps needed to receive FDA approval of CAP-1002 as a Duchenne treatment.

The study, called HOPE-2, will focus on non-ambulatory patients, aiming to preserve their arm strength and heart function.

“While there are many clinical initiatives in Duchenne muscular dystrophy, this is one of the very few to focus on non-ambulant patients,” Linda Marbán, PhD, Capricor’s president and CEO, said in a press release.

“These boys and young men are looking to maintain what function they have in their arms and hands and, based on our previous study, we think CAP-1002 may be able to do exactly that.”

Results of an earlier Phase 1-2 study (NCT02485938), called HOPE, show that a one-time infusion of the treatment — a type of cells derived from human hearts — improved heart function as well as arm and hand muscle function and strength.

In that study, the cells were infused through a catheter directly into the patient’s heart. In the HOPE 2 trial, the company wants to assess if it is possible to administer the treatment by repeat intravenous injections given every three months.

Researchers have shown that intravenous injections of CAP-1002 are beneficial in animal models of Duchenne.

The study will enroll about 84 boys and young men with Duchenne across 10 to 12 U.S. sites. Participants will be randomly assigned treatment with CAP-1002 or a placebo. After a year, researchers will explore whether the treatment improved patients’ arm strength and function.

The trial will be led by Craig M. McDonald, MD, a specialist in clinical management of muscular dystrophies and director of neuromuscular disease clinics at UC Davis Health.

“The FDA’s clearance of this IND upon its initial submission is a significant step forward in our development of CAP-1002,” said Marbán, referring to the FDA’s approval of Capricor’s investigational new drug application.

The company also plans to apply for a Regenerative Medicine Advanced Therapy (RMAT) Designation for CAP-1002. Such a designation could help speed up the regulatory review of the treatment.

While CAP-1002 is a cellular therapy, composed of so-called cardiosphere-derived cells from donated human hearts, it does not act as stem cells do. Instead, the cells release tiny vesicles containing factors that are beneficial for other cells.