Gut-Brain Axis Targeted by New Autism Treatment

A treatment by Axial Therapeutics now entering a phase 2 trial indirectly affects brain cells by working on metabolites in the gut.

What to know:

  • Biotech company Axial Therapeutics recently raised $37.25 million to fund a phase 2 trial of AB-2004, their molecular therapy intended to treat irritability in children with autism.

  • Axial is exploring how metabolites in the gut can alter the development of certain brain cells, including those that produce myelin, and that potentially affect psychological states, such as danger sensing and fear conditioning.

  • AB-2004 is an oral medication that works by passing through the gut and picking up metabolites that are then passed out of the body in stool.

  • Data from the phase 1a/2b trial showed that AB-2004 was safe and that it reduced plasma and urine levels of key gastrointestinal neuroactive microbial metabolites.

  • Results from the phase 2 trial, which is beginning soon, are expected in 2023.

This is a summary of the article, “Treating Autism Through the Gut? Axial Therapeutics Refuels on Its Exploration of the Gut-Brain Axis,” published by Endpoints News on October 13. The full article can be found on

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