Edasalonexent (formerly CAT-1004) is an oral and small molecule experimental therapy being developed by Catabasis Pharmaceuticals to treat all forms of Duchenne muscular dystrophy (DMD).

How edasalonexent works

DMD is one of the most common types of muscular dystrophy. It primarily affects boys and leads to the progressive loss of muscle fibers. The condition is caused by a mutation that results in a lack of dystrophin protein production. This protein provides structure to muscle fibers and protects them against injury. Without dystrophin, muscle fibers get damaged at each contraction.

A signaling pathway called the NF-kappa B pathway is activated in DMD patients from infancy. This pathway is responsible for muscle fiber breakdown, and the failure to repair muscle injuries that are seen in DMD patients.

Edasalonexent inhibits NF-kappa B, blocking the pathway. Through this mechanism, it is thought that edasalonexent may be able to preserve muscle function in DMD patients.

Edasalonexent in clinical trials

A combined Phase 1/2 clinical trial (NCT02439216) called MoveDMD is largely completed. It evaluated the safety, efficacy, and pharmacokinetics (how the treatment moves in the body) of edasalonexent in children with a genetically confirmed diagnosis of DMD. A total of 31 boys, ages 4 to 8, were enrolled.

Edasalonexent preserved muscle function and substantially slowed DMD disease progression through 60 weeks of treatment compared to placebo. Consistent improvements in all assessments of muscle function were observed after more than a year of treatment. Edasalonexent was well-tolerated with no safety signals observed in the trial.

A Phase 3 clinical trial to further test edasalonexent is now beginning. This 52-week trial, PolarisDMD (NCT03703882), will be a multi-center trial with test sites in the U.S., Canada, Asia, and Australia.

PolarisDMD aims to recruit 125 boys, ages 4 to 7, who have not been treated with corticosteroids for at least six months. Patients will be randomized, 2:1, to receive either edasalonexent (100 mg/kg capsule) or placebo daily. Patients will have clinic visits every three months, and its primary goal is changes in North Star Ambulatory Assessment scores at one year. This includes tests that measure the time it takes the children to stand, the four-stair climb test, and the 10-meter walk/run test. The boys’ muscle strength and growth, as well as their heart and bone health, will also be assessed. No muscle biopsies or magnetic resonance imaging (MRI) will be required for this study.

After 12 months, patients will be invited to continue receiving edasalonexent, or begin treatment, in an open-label extension study. PolarisDMD is now recruiting eligible patients, and information is available by clicking here. The trial is expected to conclude in June 2020.

Other information

The U.S. Food and Drug Administration (FDA) granted edasalonexent orphan drug and fast track designations. The European Commission (EC) also granted the treatment orphan medicinal product designation for the treatment of DMD.

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