Axial Biotherapeutics Announces Publication of Preclinical Data Highlighting the Link Between Human Gut Microbiota and Behavioral Symptoms of Autism Spectrum Disorder in Mouse Models

WALTHAM, Mass.–(BUSINESS WIRE)–Axial Biotherapeutics, a biotechnology company dedicated to building a
unique class of gut-targeted programs for neurodegenerative diseases and
neurodevelopmental disorders, today announced the publication of new
findings in Cell by Sarkis K. Mazmanian, Ph.D., the Company’s
scientific co-founder, and California Institute of Technology (Caltech)
researchers, demonstrating that the colonization of germ-free mice with
gut microbiota from human donors with Autism Spectrum Disorder (ASD)
induces hallmark behaviors of ASD as compared to control,
typically-developing (TD) gut microbiota.

The publication, entitled “Human Gut Microbiota from Autism Spectrum
Disorder Promote Behavioral Symptoms in Mice,” is the first to suggest a
direct link between gut microbiota and the pathophysiology and
behavioral outcomes of ASD. This link further supports Axial
Biotherapeutics’ foundational scientific rationale, suggesting that the
gut microbiome regulates behavior in mice via the production of
neuroactive metabolites.

“We are pleased to see these new preclinical insights from Sarkis and
his team, which further enhance our understanding of the link between
the human gut microbiome and the brain as it relates to ASD and other
CNS diseases. It is exciting to build upon our hypothesis rooted in this
innovative research as evidence continues to show that non-genetic, yet
heritable gut microbiota may contribute to human behaviors,” said David
Donabedian, Ph.D., and Co-founder and Chief Executive Officer of Axial
Biotherapeutics. “As we continue to advance our clinical-stage
candidate, AB-2004, we look forward to drawing on these foundational
insights to better understand the complexities of altered ASD human
behaviors.”

“ASD manifests as changes in complex human behaviors, and is likely the
outcomes of genetic and environmental factors. Through our research, we
have been able to see a clear induction of hallmark ASD behaviors in
germ-free mice that harbor human ASD gut microbiota, which supports
Axial’s approach to ASD-symptom treatment,” said Dr. Mazmanian, the Luis
& Nelly Soux Professor of Microbiology in the Division of Biology and
Biological Engineering at Caltech and scientific co-founder of Axial
Biotherapeutics. “Our findings represent additional evidence for a new
scientific paradigm in which specific bacterial metabolites produced by
gut bacteria modulate behaviors in experimental animals, and potentially
in people. While more work is needed, this study and several other
recent publications suggest that gut-brain connections contribute to the
pathophysiology of some forms of ASD.”

In November 2016, Axial Biotherapeutics entered into a license agreement
with Caltech for worldwide exclusivity to related intellectual property
in applications for neurological diseases and disorders. Patient
screening for the Phase 1b/2a clinical trial of AB-2004 was initiated in
the first quarter of 2019.

About Autism Spectrum Disorder

According to the Center for Disease Control (CDC) about 1 in 59 children
has been identified with ASD. Core symptoms of ASD include impairments
in social interaction, communication and the presence stereotyped
repetitive behaviors. Comorbidities are extensive and diverse, and
include gastrointestinal (GI) disfunction, metabolic abnormalities,
allergies, autoimmune disorders, neuroinflammation and epilepsy. GI
disfunction 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 Biotherapeutics

Axial Biotherapeutics is a clinical stage biopharmaceutical company
pioneering novel science focused on the interaction between the brain
and the gut to mitigate the causes and symptoms of CNS and other
gut-derived diseases. The Company has built a pipeline of novel
small-molecules and live biotherapeutics with lead programs to address
the significant unmet patient needs associated with Parkinson’s Disease
and Autism Spectrum Disorder.

Contacts

Julie Seidel
Stern Investor Relations, Inc.
212-362-1200
[email protected]