17 July 2019 - - US-based biotechnology company Axial Biotherapeutics has dosed the first patient in its Phase 1b/2a clinical trial of AB-2004. In animal models, AB-2004 has demonstrated the ability to repair leaky gut and improve repetitive behaviour, anxiety and Autism Spectrum Disorder -related sensorimotor gating deficits by removing key microbial metabolites, the company said.
ASD is an indication that currently has no approved therapies for the treatment of the core symptoms.
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.
According to the Center for Disease Control, about 1 in 59 children has been identified with Autism Spectrum Disorder. 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 dysfunction, metabolic abnormalities, allergies, autoimmune disorders, neuroinflammation 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.
Axial Biotherapeutics is a clinical stage biopharmaceutical company developing 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.