7 May 2019 - - US-based biotechnology company Axial Biotherapeutics has presented new preclinical data on AB-2004, its orally administered, drug candidate that has demonstrated the ability to repair leaky gut and improve repetitive behaviour, anxiety, and ASD-related sensorimotor gating deficits by removing key microbial metabolites in animal models with Autism Spectrum Disorder, the company said.

The company presented the data in a poster presentation at the International Society for Autism Research (INSAR) 2019 annual meeting in Montreal, Canada.

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.

According to the Center for Disease Control 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 disfunction, metabolic abnormalities, allergies, autoimmune disorders, neuroinflammation and epilepsy. GI disfunction is estimated to occur in 40-70 percent 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.

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.