Axial Therapeutics Announces Age Expansion in Global Phase 2b Clinical Trial for Irritability Associated with Autism Spectrum Disorder (ASD)

-- Children aged 5 to 17 with autism are now eligible to enroll in the clinical trial of AB-2004, a gut targeted, molecular therapeutic --

-- Novel small molecule microbiome approach focused on gut-brain axis connection offers potential alternative to anti-psychotic treatments --

WOBURN, Mass., April 04, 2023 (GLOBE NEWSWIRE) -- Axial Therapeutics, a clinical-stage biopharmaceutical company dedicated to the development of gut-targeted, small molecule therapeutics for neurological conditions and oncology, today announced it is expanding the age eligibility criterion to include children ages 5 to 17 years, previously restricted to ages 13 to 17 years. The age expansion is part of a protocol amendment in the company’s global Phase 2b clinical trial for lead candidate, AB-2004, a gut-targeted, molecular therapeutic that is being studied for alleviating the irritability associated with Autism Spectrum Disorder (ASD).

“We are pleased to announce that children ages 5 to 17 are now eligible to participate in our ongoing Phase 2b clinical trial of AB-2004 for the treatment of irritability associated with ASD, and we look forward to finalizing enrollment later this year,” said Stewart Campbell, Ph.D., chief executive officer, Axial Therapeutics. “This is significant because many children with autism struggle with irritability and anxiety. AB-2004 offers potential hope of a new therapeutic option for autism-related irritability that may improve the daily lives of children with autism while also avoiding the side effects and risks of the currently available medications, providing a potential alternative for these children.”

Approximately 1 in 44 children in the U.S. and 1 in 100 children worldwide have been identified with ASD according to the Centers for Disease Control and Prevention (CDC) and World Health Organization (WHO), respectively. Core traits of autism include altered social interaction, impaired verbal and non-verbal communication, and the presence of stereotyped repetitive behaviors. Co-occurring conditions that impact quality of life are extensive and diverse, and include irritability, anxiety, attention deficit hyperactivity disorder (ADHD), allergies, autoimmune disorder, neuroinflammation, and epilepsy. Research studies have reported that irritability impacts the majority of the autistic pediatric population. Irritability associated with autism presents with varying severity and manifests in a range of behaviors including aggressive behavior, self-harming, isolation, and emotional meltdowns.

About the AB-2004 Phase 2b Trial

The Phase 2b trial is a global, randomized, double-blind, placebo-controlled study evaluating the efficacy, safety, and tolerability of AB-2004 in children aged 5 to 17 who have been diagnosed with autism. In total, the study will enroll an estimated 140 patients who will receive either AB-2004 or placebo.

The primary efficacy endpoint of the study is the mean change in the Aberrant Behavior Checklist Irritability subscale (ABC-I) from baseline to week 8 for AB-2004. Key secondary outcome measures include the mean change in the Clinical Global Impression-Severity (CGI-S) from baseline to week 8; and the number of participants who report treatment emergent adverse events from baseline to week 8.

The AB-2004 Phase 2b study is being conducted at multiple clinical trial centers in the United States, Australia and New Zealand. The Phase 2b study enrollment is expected to be complete by year end 2023. More information about the U.S. trial is available at (NCT04895215). To learn more, visit the clinical trial’s website at

About AB-2004

AB-2004 is a first-in-class, molecular therapeutic that targets the microbiome gut-brain axis and its role in autism. AB-2004 has a unique mechanism of action that selectivity sequesters certain bacterially derived metabolites in the gut before they enter the bloodstream and reach the brain. Axial’s gut-targeted approach minimizes the potential for side effects due to a lack of systemic exposure to the drug.

Axial is evaluating AB-2004 in a Phase 2b clinical trial based upon a large body of existing human safety data. In a Phase 1b/2a clinical trial conducted in an adolescent ASD patient population, AB-2004 was safe and well-tolerated, exhibiting no drug-related serious adverse events. AB-2004 was also shown to reduce several key microbial metabolites implicated in autism and showed evidence of improving key co-occurring conditions including irritability and anxiety (Nature Medicine, 2022). Formulated as a powder that can be mixed with food, AB-2004 offers convenient oral dosing for the ASD community.

About Axial Therapeutics

Axial Therapeutics is a clinical-stage biopharmaceutical company dedicated to improving the lives of people with neurological conditions and oncology through the development of gut-targeted, small molecule therapeutics. The company’s primary focus is based on its scientific leadership in the biological role of the microbiome-gut-brain axis and its influence on the central nervous system. Harnessing its unique expertise in the microbiome, 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 neurological disorders and disease pathology, progression, and symptoms. The company is advancing a pipeline of “microbial-inspired therapeutics™” for conditions with significant unmet patient need, including autism and Parkinson’s disease, and is also pursuing pre-clinical discovery of gut-targeted therapies in oncology. Axial’s lead product candidate is AB-2004, a molecular therapeutic in Phase 2b clinical trials for the treatment of irritability in children with autism. For more information, visit

Contacts for Axial Therapeutics

Maura Gavaghan
Red House Communications

Jeffrey Young, CFO