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Results from Axial Therapeutics Phase1b/2a Study of AB-2004 in Adolescents with Autism Spectrum Disorder Met Primary Endpoint and Reduced Irritability and Other Symptoms

Results to be Presented at The BRAIN Foundation Synchrony 2020 Conference

Axial R&D Leader to Chair Day-long Session on Clinical Trials for ASD and New Therapeutics

WALTHAM, Mass., Dec. 3, 2020 – Axial Therapeutics Inc., a clinical-stage biotechnology company dedicated to building a unique class of gut-restricted therapeutics for central nervous system (CNS) disorders and conditions, today announced that A. Stewart Campbell, Ph.D., Axial’s Senior Vice President of Research and Development, will present new data from Axial’s Phase 1b/2a study of AB-2004 in male adolescents with autism at The BRAIN Foundation Synchrony 2020 conference Dec. 6.

The findings from the open-label clinical study demonstrated that AB-2004, Axial’s first-in-class, orally administered therapeutic, met the primary endpoint demonstrating safety, tolerability and adherence with no drug-related adverse events. In addition, significant improvements in subjects with high irritability or high anxiety were observed, as well as improvements in social withdrawal and gastrointestinal symptoms (GI). Specifically, the data showed that AB-2004 reduced several key GI neuroactive microbial metabolites (NMMs™) measured in plasma and urine.

The Phase 1b/2a study evaluated the safety and tolerability of AB-2004 in males aged 12 to 17 with autism spectrum disorder (ASD) across three sites in Australia and New Zealand. Each subject had a confirmed diagnosis of autism via the Autism Diagnostic Observation Schedule (ADOS) and suffered from GI symptoms. Each subject was dosed for 8 weeks with a low dose administered for 2 weeks, a mid-dose for 2 weeks and a high dose for 4 weeks, followed by a 4-week washout and recovery. All behavioral analyses and sample collection were conducted at the clinical sites.

“We are excited about our positive efficacy and safety data from our AB-2004 Phase 1b/2a study. These results support our gut-restricted approach and novel hypothesis of reducing NMMs™ to have a significant positive impact on ASD-related behaviors without drug-related side effects,” said David H. Donabedian, Ph.D., Co-founder and Chief Executive Officer of Axial Therapeutics.

“We intend to file our Investigational New Drug application shortly and initiate our Phase 2, placebo-controlled study of AB-2004 soon thereafter,” added Donabedian.

About ASD

According to the Centers for Disease Control and Prevention (CDC), about 1 in 59 children has been identified with ASD. Core symptoms of ASD include impairments in social interaction, communication and the presence of stereotyped repetitive behaviors. Comorbidities are extensive and diverse, and include GI dysfunction, irritability, anxiety, ADHD, metabolic abnormalities, allergies, autoimmune disorder, neuroinflammation and epilepsy. GI dysfunction is estimated to occur in 40 to 70% of individuals with ASD.

The only products currently approved to treat the irritability associated with ASD are antipsychotics. Due to the safety and side effect issues associated with these drugs, their labels carry black-box warnings.
The severity of core and non-core ASD symptoms and the lack of safe and effective treatments illustrate the need for new therapeutics to address this significant unmet medical need.

About the Presentation at Synchrony Symposium

Dr. Campbell’s presentation entitled “Safety, tolerability and preliminary efficacy of AB-2004, a gut-restricted molecule targeting microbial metabolites, in an adolescent population with autism spectrum disorder (ASD)” will occur at 2:30 p.m. PST Dec. 6 and available via streaming for all registered Synchrony Symposium participants. That same date Dr. Campbell will also be leading the day’s discussion focused on ASD clinical trials and potential new treatments with representatives from UCSF’s Center for ASD and NDDs, Weill Cornell Medicine Center for Autism and the Developing Brain, Boston’s Lurie Center for Autism, and Yale School of Medicine Child Study Center and others.

About Axial Therapeutics

Axial Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery and development of gut-restricted, small molecule therapeutics for central nervous system (CNS) disorders. The company is leveraging its expertise in the gut-brain axis and its unique drug development platform to advance novel therapies that have the potential to transform the treatment paradigm in neurological diseases. Axial is advancing a pipeline of small molecule product candidates with lead clinical programs that address underlying pathology and resulting symptoms in Autism Spectrum Disorder (ASD) and Parkinson’s Disease (PD), and additional preclinical initiatives in small molecules in other areas of autism and PD, as well as oncology and rare CNS diseases.

Contacts for Axial Therapeutics


Mike Beyer
Sam Brown, Inc.


Jeffrey Young, CFO

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