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 neurodegenerative diseases.

Harnessing the Gut-Brain Axis to Treat Neurological Disorders

Axial is taking a revolutionary approach to treating neurological diseases.  Based on the groundbreaking work from Dr. Sarkis Mazmanian’s lab at Caltech that demonstrated a biological link between Autism Spectrum Disorder (ASD) and Parkinson’s Disease (PD) via the gut-brain axis.  The idea of a gut-brain axis stems from (i) previous evidence that patients with neurological disorders experience gastrointestinal problems earlier in their disease and/or at the time of diagnosis and (ii) 70% of neurons in the peripheral nervous system, meaning those not in the brain or spinal cord, reside in the intestines and connect to the central nervous system via the vagus nerve.

Traditionally, any drug developed for neurological diseases must reach the brain through passage of the blood–brain barrier, a challenging goal to achieve.  Axial’s approach is to develop novel drugs that provide a localized, gut-restricted treatment, therefore obviating the need for systemic delivery of high doses to reach the brain.

In April, we announced positive topline data from our Phase 1b/2a clinical study of AB-2004 in ASD.  There are more than 1.2 million children in the US with ASD and no approved therapies for core symptoms. The only approved drugs for the irritability associated with ASD carry a black box warning. AB-2004 is given orally, stays in the GI tract, and removes certain metabolites implicated in ASD. It does not need to penetrate the brain to be effective, offering a creative and safer approach with potential for a much better safety and tolerability profile which could vastly improve patient outcomes. Further, Axial’s pipeline includes a Parkinson’s Disease (PD) program, a rare CNS program and a small molecule program in Oncology.

The company established operations in Boston in 2016 and is now located in Waltham, MA in a 25,000 sq. ft facility.  Axial was named one of Nature’s top academic spinouts in 2016, one of Fierce Biotech’s Fierce15 in 2017 and has received funding from blue chip investors including Longwood, Seventure, Domain and Taiho Ventures.