Therapy Areas: AIDS & HIV
Axial Therapeutics Receives FDA Clearance of IND Application and Initiates Phase 2b Trial for AB-2004, Lead Gut-Targeted, Molecular Therapeutic for the Treatment of Irritability in Autism Spectrum Disorder
27 October 2021 - - US-based biopharmaceutical company Axial Therapeutics has received clearance from the US Food and Drug Administration of its investigational new drug application for lead candidate, AB-2004, a gut-targeted, molecular therapeutic for the treatment of irritability associated with ASD, the company said.

In addition to the FDA clearance, clinical trial authorizations have been received for AB-2004 in Australia and New Zealand.

By advancing AB-2004 further into the clinic, Axial is aiming to develop new therapeutic options to help manage irritability associated with ASD, and potentially other co-occurring conditions that can prevent children with autism from fully participating in daily life.

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, 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. Formulated as a powder that can be mixed with food, AB-2004 offers convenient oral dosing for the ASD community.

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 13 to 17 who have been diagnosed with ASD.

The study will enroll an estimated 195 patients who will receive low or high doses of AB-2004 or placebo.

The primary efficacy endpoint of the study is the mean change in irritability using the Aberrant Behavior Checklist from baseline to week 8 for AB-2004 high dose.

Secondary outcome measures include the mean change in the ABC-I score (Irritability) from baseline to week 8 for AB-2004 low dose; the mean change in the Clinical Global Impression-Severity (CGI-S) from baseline to week 8 for AB-2004 high and low dose; and the number of participants who reported treatment emergent adverse events from baseline to week 8.

The AB-2004 Phase 2b study will be conducted at approximately 20 clinical trial centers in the United States and approximately seven sites in Australia and New Zealand and is expected to generate topline data in 2023.

According to the Centers for Disease Control and Prevention, approximately 1 in 54 children has been identified with ASD.

Core symptoms of ASD include impairments in social interaction, communication, and the presence of stereotyped repetitive behaviors.

Co-occurring conditions that impact quality of life are extensive and diverse, and include irritability, anxiety, ADHD, allergies, autoimmune disorder, neuroinflammation, and epilepsy. Physicians have reported irritability impacts a majority of pediatric ASD patients.

The presentation of ASD-associated irritability can vary with severity and age and can be caused by a broad array of different factors, including lack of sleep, the inability to communicate pain, and mental health conditions.

Axial Therapeutics is a clinical-stage biopharmaceutical company focused on neurological disorders and diseases.
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