Catabasis has announced plans to start a global phase 3 trial to test the safety and effectiveness of a potential drug called edasalonexent in people living with Duchenne muscular dystrophy. The trial is scheduled to start later in 2018.

The researchers aim to recruit 125 participants aged between 4 and 7 to the trial, regardless of their mutation type. Participants will receive either edasalonexent or a placebo (an inactive form of the drug) for 12 months – with two people receiving the potential drug for every person receiving placebo. Following the trial, Catabasis currently plans to offer all participants edasalonexent in an open-label extension.

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.

The trial will also consider participants who are receiving a stable dose of Eteplirsen – an exon-skipping drug that is licensed in the US. This important inclusion shows that drug developers are starting to consider licensed treatments in their trial designs.   

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