Potential Autism Drug Improves Quality of Life in Children

Autism Spectrum Disorder
Source: Freepik

New drug significantly reduces irritability and anxiety among children with autism spectrum disorder in a recent phase 1b/2a trial.

Autism spectrum disorder (ASD) affects one in 160 children worldwide. Along with behavioural and communication problems, affected individuals can also face depression, anxiety, and irritability. Thus, affecting their overall quality of life. Although the exact mechanism behind the disorder remains unclear, previous studies have suggested a role between the gut microbiome and ASD. For example, the metabolite 4-ethylphenyl sulfate (4EPS) is often dysregulated in the fecal and blood samples of people with ASD. Moreover, injection of the metabolite into rodents causes behavioural symptoms. Therefore, researchers at Axial Therapeutics have developed a gut-targeted potential autism drug. The team recently released published results from the Phase 1 clinical trial of its new drug.

The drug, AB-2004, binds and removes certain gut bacteria metabolites. Thus, preventing these metabolites from reaching the brain. Moreover, since the drug itself does not get absorbed, it prevents the development of any systemic effects.

The Phase 1 trial comprised 30 adolescents with both ASD and gastrointestinal complications in Australia and New Zealand. Researchers aimed to assess the drug’s safety and tolerability, and its effect on specific gut-derived metabolites. According to the results, the drug did not cause any serious adverse events among the participants. Moreover, anxiety and irritability scores reduced significantly for all participants over the course of the trial. The study further highlighted the persistence of behavioural improvements even after weeks of the treatment ending.

The data highlighted in Nature Medicine further support our novel therapeutic strategy focused on specific targets in the gut microbiome. We strongly believe that AB-2004 can improve the quality of life of many children with autism.

Dr. A. Stewart Campbell, lead author

Drug Improves Quality of Life

Although there is no cure for autism, several behavioural and communication therapies help children develop new social skills. Moreover, certain children may also require antipsychotics and antidepressants for treating severe behavioural problems such as anxiety and aggressiveness. However, these drugs often cause severe side effects in individuals. AB-2004, therefore, provides a safer and more effective alternative for treating these symptoms.

The lack of safe and effective treatment options for co-occurring conditions associated with ASD, such as irritability and anxiety, exacerbate the daily challenges faced by children and their families.

Robert L. Hendren, principal investigator of Phase 2 trial

Based on the successful results, the team has now commenced Phase 2 trials for the drug. Researchers aim to recruit 195 children aged 13 to 17 years with ASD and gastrointestinal symptoms. The trial will assess the safety, efficacy, and tolerability of AB-2004 among the participants.


Stewart Campbell, A., Needham, B.D., Meyer, C.R. et al. Safety and target engagement of an oral small-molecule sequestrant in adolescents with autism spectrum disorder: an open-label phase 1b/2a trial. Nat Med (2022). https://doi.org/10.1038/s41591-022-01683-9


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