Axial Biotherapeutics Announces Completion of Enrollment in Phase 1b/2a Clinical Trial Evaluating AB-2004 in Patients with Autism Spectrum Disorder

Waltham, Mass., November 18, 2019 — Axial Biotherapeutics, a biotechnology company dedicated to building a unique class of gut-targeted therapeutic programs for neurodegenerative diseases and neurodevelopmental disorders, today announced the completion of enrollment in its Phase 1b/2a clinical trial evaluating AB-2004. AB-2004 is a first-in-class therapeutic that removes certain metabolites from the gastrointestinal (GI) tract, in patients with Autism Spectrum Disorder (ASD).


“The completion of enrollment in our AB-2004 Phase 1b/2a clinical trial marks an important milestone for Axial as we now have all subjects enrolled in this first clinical trial within our target population of ASD,” said David Donabedian, Ph.D., Co-founder and Chief Executive Officer of Axial Biotherapeutics. “We look forward to a further advancing this program to help a significantly underserved ASD patient population. We plan to report topline data from this trial next year.”


The Phase 1b/2a clinical trial is designed to examine the safety and tolerability of AB-2004 in up to 25 male adolescent subjects with ASD. Exploratory endpoints include changes in bacterial metabolite levels, GI function, gut microbiome and behavior.


About AB-2004


AB-2004 is a first-in-class, orally administered therapeutic, targeting Autism Spectrum Disorder (ASD). In a preclinical mouse model of ASD, AB-2004 reduced urinary levels of 4-EPS and other key metabolites, repaired leaky gut and improved repetitive behavior, anxiety and ASD-related sensorimotor gating deficits. The company anticipates topline data from the Phase 1b/2a study in 2020.


About Autism Spectrum Disorder


According to the Center for Disease Control (CDC), about 1 in 59 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. Comorbidities are extensive and diverse, and include gastrointestinal (GI) dysfunction, metabolic abnormalities, allergies, autoimmune disorders, neuroinflammation, anxiety and epilepsy. GI dysfunction is estimated to occur in 40-70% of individuals with ASD. The medical severity of core and non-core ASD symptoms and the lack of safe and effective long-term treatments argue strongly for pursuing fundamentally new measures to address this significant unmet medical need.


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 in neurological diseases. Axial is advancing a pipeline of small molecule product candidates with lead clinical programs that address underlying pathology and resulting symptoms in Autism Spectrum Disorder (ASD) and Parkinson’s Disease (PD), and additional preclinical initiatives in small molecules in other areas of autism and PD, as well as oncology and rare CNS diseases.


Contacts for Axial Therapeutics


Media:

Mike Beyer
Sam Brown, Inc.
312-961-2502
mikebeyer@sambrown.com


Investors:

Jeffrey Young, CFO
781-701-8467
jeffrey@axialtx.com