Industry Spotlight: Axial Therapeutics

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.

As the scientific leader in the biological role of the microbiome gut-brain axis and its impact on neurological conditions, Axial Therapeutics is uniquely positioned to advance a fundamentally different therapeutic approach for the treatment of neurological diseases and disorders.

Their scientific focus on the microbiome gut-brain axis represents a transformative therapeutic approach that has potential to treat neurological diseases and disorders in ways never before possible.

Novel Medical Solutions for Patients with Autism and Parkinson’s Disease

Axial’s world-class expertise in microbiome gut-brain biology combines with the robust capabilities of their discovery platform to provide the foundation for their current and future pipeline of small molecule drugs for neurodegenerative diseases and neurodevelopmental disorders. Their lead programs are focused on the treatment of irritability in autism, and the slowing of disease progression in Parkinson’s disease.

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. AB-2004 has a unique mechanism of action of selectively absorbing 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 because, unlike most drugs, AB-2004 is designed to act in the gut and avoid being absorbed into the bloodstream. In addition, 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.

Based on this strong body of evidence, Axial has started a global Phase 2b study to further evaluate AB-2004 as a potential treatment for irritability associated with autism.

More information about the study as well as the location of clinics currently enrolling participants can be found here:

Company website

Axial Therapeutics at BRAIN Synchrony Symposia