Earlier this week Catabasis announced that a Phase 3 trial of edasalonexent in Duchenne has started, with trial centres to open shortly – first in the USA and then around the world (including Europe).

The company plans to enrol approximately 125 boys living with Duchenne and aged 4-7 years old to the trial. Only those not taking steroids will be eligible. The participants will be randomly assigned to groups, with one group receiving edasalonexent and the other group receiving placebo – an inactive version of the drug. The trial is designed so that 2 boys will receive edasalonexent for every boy who receives placebo.

The trial will use the North Star Ambulatory Assessment, as well as timed function tests and measures of muscle strength, growth, cardiac and bone health to determine the effectiveness of edasalonexent. After 12 months in the trial, the company plans to offer all participants the chance to enter an open-label extension.

We are so glad to bring edasalonexent into Phase 3 for boys affected by Duchenne that could benefit from this potential therapy - Dr Richard Finkel, a Principal Investigator of the trial.

Edasalonexent is designed to stop a protein called NF-kB working properly. The protein is activated in people with Duchenne and this may play a role in inflammation and muscle degradation as well as preventing of muscle regeneration. Early trial results of edasalonexent showed it could preserve muscle function and slow progression of Duchenne.

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