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Our Pipeline

Our Programs


Axial is translating its innovative Microbial-Inspired Therapeutics® discovery platform into a pipeline of gut-targeted, small molecule drugs with defined mechanisms of action that mitigate the impact of disease-linked bacteria in the gut with an initial focus in neurodegenerative diseases and neurodevelopmental disorders. Axial’s lead programs are in Autism Spectrum Disorder and Parkinson’s disease. We also have demonstrated preclinical proof-of-concept in our liver disease and oncology programs, and are en route to identifying drug candidates.

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AB-2004: Redefining the treatment of irritability in autism


Axial’s lead product candidate, AB-2004, is a gut-restricted molecular therapy being developed as a treatment for irritability associated with autism. Our scientific founders have shown that bacterial metabolites produced in the gut can enter the bloodstream and reach the brain where they impact development of certain brain cells in regions of the brain associated with emotional control and behavior. AB-2004 has the unique mechanism of action of selectively adsorbing these metabolites in the gut before they enter the bloodstream and reach the brain. In a Phase 1b/2a clinical trial in adolescents with autism, AB-2004 was safe and well-tolerated and exhibited no drug-related serious adverse events (ACTRN12618001956291). AB-2004 was also shown to reduce several microbial metabolites implicated in autism and showed evidence of improving key behaviors, including irritability and anxiety. Axial’s gut-targeted approach minimizes the potential for side effects because, unlike most drugs, AB-2004 is designed to act in the gut and avoid being absorbed into the bloodstream.

Based on this strong body of evidence, Axial undertook and recently completed enrollment in a global Phase 2b randomized placebo-controlled clinical study to further evaluate AB-2004 as a potential treatment for irritability associated with autism. More information about the study can be found at


AX-5006: Novel gut-selective small molecule candidate for Parkinson’s disease

Axial's pipeline also includes AX-5006, a gut-selective, small molecule therapy aimed at slowing disease progression and improving the quality of life of people with Parkinson's disease (PD). In preclinical research, Axial has established that certain gut bacteria, specifically through the production of bacterial amyloids, contribute to gastrointestinal symptoms, motor symptoms, and brain pathology in PD. AX-5006 is designed to reduce bacterial amyloid aggregation in the gut, thereby alleviating symptoms and attenuating disease progression. Being gut-targeted, it is anticipated to have a favorable safety profile.

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