US-based biotechnology firm Capricor Therapeutics has obtained clearance for its investigational new drug (IND) application of CAP-1002 from the US Food and Drug Administration (FDA) to treat patients in advanced stages of Duchenne muscular dystrophy.

CAP-1002 is an investigational therapy made of allogeneic cardiosphere-derived cells (CDCs) that contain cardiac progenitor cells, while Duchenne muscular dystrophy is a fatal genetic disorder with limited treatment options.

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The new randomised, double-blind, placebo-controlled HOPE-2 clinical trial will assess the safety and efficacy of intravenous and repeat doses of CAP-1002 in around 84 boys and young men at 10-12 sites in the US.

Planned to be launched in the first quarter of next year, the trial will enrol subjects whose walking ability has been seriously impaired by the loss of muscle function due to the progression of the disease.

Capricor Therapeutics president and CEO Linda Marbán said: “The FDA’s clearance of this IND upon its initial submission is a significant step forward in our development of CAP-1002.

“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.”

“While there are many clinical initiatives in Duchenne muscular dystrophy, this is one of the very few to focus on non-ambulant patients.

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“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 primary efficacy endpoint of the HOPE-2 trial is a relative change from baseline in the mid-level dimension of the Performance of the Upper Limb test to month 12.

Capricor expects that achieving primary endpoint in this trial could support its Biologics License Application (BLA) submission for marketing approval of CAP-1002.

The firm also intends to apply for the regenerative medicine advanced therapy (RMAT) designation for CAP-1002.