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Our Pipeline


According to the Centers for Disease Control and Prevention (CDC), approximately 1 in 44 children has been identified with Autism Spectrum Disorder (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. Although 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 disorders.


Medicines for treating core symptoms and co-occurring conditions associated with ASD such as irritability have long represented a significant area of unmet medical need. The prevalence and severity of challenges faced by people with autism and the lack of safe and effective treatment options make ASD an important focus of novel drug discovery to bring forward innovative medical interventions.


Axial is developing AB-2004, a therapeutic candidate for the reduction of irritability in children with autism.
Harnessing the Connection Between the Gut and the Brain

AB-2004 is Axial Therapeutic’s lead investigational therapy that targets the microbiome gut-brain axis and its role in co-occurring conditions associated with Autism Spectrum Disorder (ASD). AB-2004 is a gut-targeted, molecular therapeutic, which means it is designed to work in the gut only and does not enter other bodily tissues. We believe this mechanism may result in minimal side effects due to lack of exposure across other parts of the body, although this hypothesis needs to be verified in controlled clinical trials.



Novel Medical Solution for Irritability in Autism

AB-2004 is being studied as a potential new treatment for managing irritability associated with ASD in children. Irritability is a common co-occurring condition in children with autism for that limited treatment options currently exist and those that are approved can have significant side effects. 


Scientific evidence has shown there may be a link between bacteria commonly found in the digestive tract, and the brain which could contribute to certain characteristics, such as irritability, in children with ASD. AB-2004 is designed to adsorb certain substances produced by gut bacteria to reduce their ability to enter the bloodstream and reach the brain.   


Innovative Product to Improve Lives

The active ingredient in AB-2004 is a highly engineered form of spherical carbon designed with human safety and biological selectivity in mind, making it very different from activated charcoal. Each sphere of AB-2004 consists of a network of pores that allows it to selectively adsorb metabolites that may contribute to characteristics associated with ASD like irritability and anxiety.

AB-2004 is a tasteless and odorless powder formulation that can be mixed with soft food which makes taking the medication very easy. Finally, AB-2004 will not discolor your hands or your food.

AB-2004 has several critical attributes that make it potentially ideal for chronic clinical use and differentiate it from activated charcoal available over the counter.




activated charcoal

Commitment to Patient Safety

AB-2004 is produced using a manufacturing process that meets the highest global standards for safety and quality. This highly controlled manufacturing process, done under strict regulatory control, guarantees the highest level of product quality.

Body of Medical Evidence

AB-2004 is being studied for irritability associated with ASD based a growing body of scientific evidence showing bacterial substances produced by bacteria commonly found in the gut can contribute to characteristics like irritability and anxiety (insert link to nature medicine press release). In a previous clinical study involving autistic adolescents, AB-2004 was shown to be safe and well tolerated. 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.

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