Capricor Therapeutics has dosed the first subject in the Phase III HOPE-3 clinical trial of cell therapy, CAP-1002, to treat late-stage Duchenne muscular dystrophy (DMD).

CAP-1002 comprises allogeneic cardiosphere-derived cells (CDCs), a kind of progenitor cell. 

The double-blind, randomised, placebo-controlled trial will enrol nearly 70 subjects in the US. 

Trial subjects will be categorised into a 1:1 ratio to receive either CAP-1002 or placebo. 

Patients in the active arm will be given 150 million CDCs (CAP-1002) intravenously every three months for a total of four doses.

The Performance of the Upper Limb (PUL) 2.0, with a conceptual framework indicating weakness progression in upper limb function will be the trial’s primary outcome measure. 

An established tool, PUL is intended to evaluate high (shoulder), mid (elbow) and distal (wrist and hand) function.

Various other secondary endpoints including cardiac function analyses will also be measured in the trial.

Recently, the company collaborated with Nippon Shinyaku which holds the marketing and distribution rights in the US. 

Outside the US, Capricor holds global commercial rights to the cell therapy.

This collaboration offers funding for HOPE-3 and other potential milestone-based payments to back CAP-1002’s clinical development for DMD.

Capricor Therapeutics CEO Linda Marbán said: “The data from our Phase II clinical trial suggest that CAP-1002 can slow loss of function by as much as 70% in terms of upper limb skeletal muscle function. 

“Since there are very limited therapeutic options for these patients and CAP-1002 has been shown to be safe and effective, we are pleased to begin this pivotal trial with the goal of achieving regulatory approval as quickly as possible.”

A genetic disorder, DMD is characterised by progressive weakness and chronic skeletal, heart and respiratory muscle inflammation.

In March this year, the company reported topline data from the Phase II INSPIRE trial of CAP-1002 to potentially treat severe Covid-19 in hospitalised patients.

Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.

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