Axial Therapeutics Investigates Gut-Brain Connection in Autism

Privately-held biotechnology company, Axial Therapeutics, is investigating the gut-brain connection as it advances a treatment for Autism Spectrum Disorder (ASD). The company’s lead product candidate, AB-2004, is a first-in-class oral gut-targeted small molecule therapeutic designed to treat irritability associated with autism. The compound is formulated as a powder that can be mixed with food for convenient oral dosing.

Axial completed enrollment of 156 pediatric patients into its TAPESTRY Phase 2b clinical trial evaluating AB-2004 in September 2023. Data from the global, randomized study is expected to be released in the first quarter of 2024. 

Stewart Campbell, CEO of Axial Therapeutics, commented in a statement in September, “We are pleased to announce the successful completion of enrollment in our Phase 2b clinical trial – a critical milestone that brings us closer to our goal of addressing the significant unmet need for a safe and effective therapy to treat irritability in autism that works differently from existing medications.”

In a Phase 1b/2a clinical trial AB-2004 was safe and well-tolerated, and reduced several NMM® implicated in autism, including irritability and anxiety.

Roger J. Jou, MD, MPH, PhD, Child Psychiatrist, Yale School of Medicine, and Principal Investigator of the Tapestry Autism Study, said, “AB-2004 leverages the gut-brain connection; it works in the gut by trapping bacterial byproducts and removing them in the stool, which reduces their absorption into the body and ultimately their negative effect on the brain. I eagerly anticipate the results of the Phase 2 clinical trial of AB-2004 to assess the possibility this novel approach reduces irritability associated with autism.”

The AB-2004 Phase 2b study is being conducted at multiple clinical trial centers in the United States, Australia and New Zealand, and will evaluate a primary efficacy endpoint and key secondary outcome measures.

About Axial Therapeutics

Axial Therapeutics is a clinical-stage biotechnology company dedicated to improving the lives of people with neurological disorders and conditions. The company is a scientific leader in elucidating the biological role of the microbiome-gut-brain axis and its influence on the central nervous system. Harnessing the power of its Microbial Inspired Therapeutics® platform, Axial is developing small molecule drugs with defined mechanisms of action that act on new targets to mitigate the impact of metabolites and bacteria in the gut linked to disease pathology, progression, and symptoms. The company is advancing a pipeline of small molecule drug candidates for conditions with significant unmet patient need, including autism and Parkinson’s disease, and is also pursuing preclinical discovery of gut-targeted therapies in oncology and NASH/fibrosis. Axial’s lead product candidate is AB-2004, a small molecule therapeutic in Phase 2b clinical trials for the treatment of irritability in children with autism. For more information, visit

Share This Article


About the Author

Axial Therapeutics Investigates Gut-Brain Connection in Autism

Catie Corcoran

Biotech Editor