Axial Therapeutics

Axial Therapeutics, a clinical-stage biopharmaceutical company, was established in 2016 to concentrate on the connection between the human gut microbiome and neurological diseases. The company's scientific foundation is built upon the research of its co-founder, Dr. Sarkis Mazmanian, a professor at the California Institute of Technology (Caltech) whose work has explored the gut-brain axis and its implications for central nervous system (CNS) disorders. Dr. Mazmanian's extensive background in microbiology and immunology, particularly his research into how gut bacteria can influence health and disease, provides the scientific underpinning for the company's therapeutic approach.

Axial Therapeutics is developing a pipeline of small molecule therapies designed to address the neurological symptoms of conditions like Parkinson's disease (PD) and Autism Spectrum Disorder (ASD). The company's strategy focuses on identifying and targeting specific bacterial metabolites originating in the gut that are believed to contribute to disease pathology. Rather than using live biotherapeutics, Axial's approach is to develop orally-administered small molecules that can remove these harmful metabolites from the gut before they enter the bloodstream and impact the brain. This business model is centered on the traditional biopharmaceutical pathway of drug discovery, clinical development, and eventual commercialization of approved therapies. Its primary clients are patients suffering from these neurological disorders, with the products intended to be prescribed by healthcare professionals.

The company's lead product candidate, AB-2004, is a gut-targeted, molecular therapy being investigated for its potential to reduce irritability and anxiety in children with Autism Spectrum Disorder. AB-2004 is designed to selectively absorb certain gut-derived metabolites that are thought to cross the blood-brain barrier and affect brain function. The key differentiator for this product is its focus on the gastrointestinal system to treat neurological symptoms, potentially offering a safer profile by minimizing systemic exposure. In 2022, Axial completed a Phase 2b clinical trial for AB-2004 in adolescents with ASD. Another significant milestone was the completion of a Series C financing round in 2021, which raised $37.25 million to advance its clinical programs for both ASD and Parkinson's disease.

Keywords: gut-brain axis, biopharmaceutical, neurological disorders, small molecule therapies, Parkinson's disease, Autism Spectrum Disorder, microbiome, clinical-stage, CNS disorders, gut-restricted therapeutics