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Axial Biotherapeutics Presents New Preclinical Data from AB-2004 Program Demonstrating Reduction of Microbiome-Derived Metabolites in Mouse Models with Autism Spectrum Disorder at the International Society for Autism Research (INSAR) 2019 Annual Meeting

May 2, 2019 Off By BusinessWire

– Preclinical results support development of AB-2004 as
potential treatment for gastrointestinal dysfunction and associated
behavioral symptoms of Autism Spectrum Disorder –

– Patient screening for Phase 1b/2a clinical trial of AB-2004
initiated in 1Q19 –

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, presented new preclinical data on AB-2004,
its orally administered, drug candidate that has demonstrated the
ability to repair leaky gut and improve repetitive behavior, anxiety,
and ASD-related sensorimotor gating deficits by removing key microbial
metabolites in animal models with Autism Spectrum Disorder (ASD). The
Company presented the data in a poster presentation at the International
Society for Autism Research (INSAR) 2019 Annual Meeting in Montreal,
Canada.

“We are pleased to present additional preclinical data which
substantiates the potential of our platform to remove microbial based
metabolites, by which we can restore gastrointestinal (GI) function and
improve behavior associated with CNS and gut-derived diseases,” said
David H. Donabedian, Ph.D., Co-founder and CEO of Axial Biotherapeutics.
“We believe that altered microbial metabolite profiles can drive ASD
symptoms, including behavior abnormalities, leaky gut and other GI
issues. We believe that AB-2004 has the potential to alter these
symptoms and we look forward to continuing patient screening for our
Phase 1b/2a program and progressing this product further into the
clinic.”

The main highlights from the poster presentation titled,
“Characterization of GI barrier integrity and gut microbiome-derived
metabolites in BTBR, Shank3 and Cntnap2 mouse models of ASD and
demonstration of AB-2004 as a potential mitigating therapeutic” include:

  • The Cntnap2-/- mouse model accurately recapitulated the leaky gut
    phenotype and elevated levels of the gut microbiome-derived metabolite
    4-EPS that have been reported in ASD patients
  • Treatment with AB-2004 effectively restored GI integrity and reduced
    elevated 4-EPS levels in Cntnap2-/- mice
  • The Cntnap2-/- model has been identified as a promising and
    translationally relevant animal model for the development of
    microbiome-inspired therapies for the effective treatment of GI and
    behavioral dysfunctions in ASD
  • These data support the development of AB-2004 as a treatment for GI
    dysfunction in ASD and potentially behavioral symptoms through
    reduction of pathologically active microbiome-derived metabolites

Axial is currently screening ASD adolescents for its Phase 1b/2a
clinical trial of AB-2004.

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 of 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. To learn more, visit https://www.axialbiotherapeutics.com/.

Contacts

Investor:
Julie Seidel
Stern Investor Relations
(212)
362-1200
[email protected]