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New Autism Drug Designed to Improve Quality of Life

October 13, 2023

A new drug designed to improve the quality of life in people with autism spectrum disorder (ASD) is in the works.

The medication, known as AB-2004, is an oral absorbent which is meant to attach itself to certain gastrointestinal (GI) metabolites in order to decrease the amount of these metabolites in the gut, and ultimately reduce certain symptoms of autism. 

New Autism Drug Designed to Improve Quality of Life

What does the gut have to do with autism you ask? Well, some people with autism struggle with GI issues. It is also not uncommon for some autistic individuals to have certain alterations in their gut microbiome or GI tract. 

The suggestion that bacteria in the gut could be the cause of certain behaviors is controversial, and yet AB-2004 appears to be targeted to reduce behaviors that could be driven by gut microbial metabolites in the blood of people with autism.

How AB-2004 has been tested

A human pilot study taking place in New Zealand and Australia was designed to test the safety of AB-2004. The trial included 30 adolescents with autism and GI symptoms. After enrolment, the participants had to undergo a series of behavioral tests as well as assessments that measured gastrointestinal symptoms.

According to the study: “AB-2004 was shown to have good safety and tolerability across all dose levels, and no drug-related serious adverse events were identified.”

The participants were required to gradually increase the dose of the medication for eight weeks based on their body mass. They were instructed to take 2.25 grams daily at the beginning of the trial and approximately six grams daily at the end.

Four weeks after the end of the trial, participants were requested to undergo a behavioral evaluation, and samples of urine, blood, and stool were collected.

The study found “significant reductions in specific urinary and plasma levels of gut bacterial metabolites were observed between baseline and end of AB-2004 treatment, demonstrating likely target engagement”. Furthermore, researchers observed improvements in multiple exploratory behavioral endpoints, most significantly in post hoc analysis of anxiety and irritability, as well as GI health, after eight weeks of treatment.

In conclusion

The results of the study suggest that AB-2004 reduced levels of gut microbial metabolites in plasma and urine after eight weeks of treatment. The authors of the study reported they saw evidence of improvement in ASD-related symptoms, namely, anxiety and irritability. 

Even though AB-2004 has been tested, it is still in its trial phase, and there are still many hypotheticals. The study shows there could be a need for safe therapeutic treatment to reduce the role bacterial metabolites have on autism symptoms. Hopefully, in the future, AB-2004 can provide the treatment people are looking for.


Campbell A, S, Needham B, D, Meyer C, R, Tan J, Conrad M, Preston G, Bolognani F, Rao S, G, Heussler H, Griffith R, Guastella A, J, Janes A, C, Frederick B, Donabedian D, H & Mazmanian S, K.(2022, February 14) Safety and target engagement of an oral small-molecule sequestrant in adolescents with autism spectrum disorder: an open-label phase 1b/2a trial. Nature Medicine


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