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

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.

8 comments

  1. Michael Smith says:

    Once again a treatment that may benefit many types of MD is blatantly restricted to only DMD patients. This type of research bias is ignorant and immoral. It’s obviously all about the dollars.

    • Johnny Smith says:

      Have you considered DMD is the most common, fatal, X-linked disease worldwide. Thus, in the realm of MD this is the easiest “type” to recruit and do clinical trials due to shear numbers. Therefore, the most logical and fastest route to approval is through DMD subjects.

      Then if approved the chances of approval for other “types” of MDs is very likely and could rapidly.

      Also, if it was “all about the dollars” they would have included many dystrophinopathies in this trial because it would increased the number of patients they could sell the drug to.

      In conclusion: You are the ignorant one. Get out of the way and let people a lot smarter than you help these kids.

      • Michael Smith says:

        Well then it places a higher value on one life over another. There are other forms of MD that are just as fatal. My point is that these research labs, pharmaceutical companies, and universities won’t even consider trying new drugs and therapies on the forms of MD even when they admit that it would applicable. Sometimes even more so than what they’re focused on. You can call me ignorant Johnny but I will continue to speak out about this problem.

    • Magdalena Kegel says:

      Hi Vicky,
      I’m sorry to hear about your son.
      Unfortunately, we currently have no information if the company plans to have trials of CAP 1002 in India.

    • Magdalena Kegel says:

      Hi Tracy,
      The trial has not launched yet and we don’t have information about trial sites or enrollment at the moment. We will cover the progress of this trial, so please sign up for our newsletter to receive the information as it becomes available.

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