Rare Daily Staff
Capricor Therapeutics said that the U.S. Food and Drug Administration has cleared its application to conduct a new clinical trial of its lead experimental therapy CAP-1002 in boys and young men in advanced stages of Duchenne muscular dystrophy, a rare progressive and fatal genetic muscle disorder.
CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a unique population of cells that contains cardiac progenitor cells. CAP-1002 has been shown to exert potent immunomodulatory activity and stimulate cellular regeneration, the company said.
Duchenne causes muscle degeneration and leads to death, often before the age of 30, most commonly from heart failure. It occurs in one in every 3,600 live male births across all races, cultures and countries. Duchenne muscular dystrophy afflicts approximately 200,000 boys and young men around the world. Treatment options are limited, and there is no cure.
“While there are many clinical initiatives in Duchenne muscular dystrophy, this is one of the very few to focus on non-ambulant patients,” said Linda Marbán, president and CEO. “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.”
The randomized, double-blind, placebo-controlled clinical trial called HOPE-2 is designed to evaluate the safety and efficacy of intravenous, repeat doses of Capricor’s experimental therapeutic CAP-1002 in boys and young men whose ability to walk has been seriously impaired by the loss of muscle function that occurs as Duchenne muscular dystrophy progresses.
The primary efficacy endpoint of the study will be the relative change in the mid-level dimension of the Performance of the Upper Limb test from baseline to month 12. The trial is expected to enroll approximately 84 patients and be conducted at up to 12 sites in the United States.
Capricor believes that if the primary endpoint is reached, the trial could serve as a registration trial, meaning that its results could support the submission of an application to obtain marketing approval of CAP-1002.
Capricor plans to apply for the Regenerative Medicine Advanced Therapy Designation for CAP-1002 based on updated guidance recently issued by the FDA. If granted, the RMAT Designation would be expected to facilitate CAP-1002’s path to potential registration.
Capricor previously reported significant and sustained improvements in cardiac structure and function, as well as skeletal muscle function, following a single dose of intracoronary CAP-1002. The HOPE-2 Trial will test the potential benefit of CAP-1002 as a repeated therapy delivered intravenously, with the goal of providing long-term benefit in a format that is compatible with repeat dosing over time.
November 29, 2017
Photo: Linda Marbán, president and CEO of Capricor