A GROWING PIPELINE OF GUT-TARGETED, SMALL MOLECULE DRUGS
Axial is translating its innovative drug discovery platform into a pipeline of gut-targeted, small molecule therapeutics for neurodegenerative diseases and neurodevelopmental disorders with lead indications in Autism Spectrum Disorder and Parkinson’s disease. We are also pursuing preclinical discovery of gut-targeted therapies in oncology.
AB-2004: Redefining the treatment of irritability in autism
Axial’s lead product candidate, AB-2004, is a gut-restricted molecular therapy being developed as a treatment for irritability associated with autism. AB-2004 has a unique mechanism of action of selectively absorbing certain bacterially-derived metabolites in the gut before they enter the bloodstream and reach the brain. Axial’s gut-targeted approach minimizes the potential for side effects because, unlike most drugs, AB-2004 is designed to act in the gut and avoid being absorbed into the bloodstream. In addition, in a Phase 1b/2a clinical trial in adolescents with autism, AB-2004 was safe and well-tolerated and exhibited no drug-related serious adverse events (ACTRN12618001956291). AB-2004 was also shown to reduce several microbial metabolites implicated in autism and showed evidence of improving key behaviors, including irritability and anxiety.
Based on this strong body of evidence, Axial has started a global Phase 2b study to further evaluate AB-2004 as a potential treatment for irritability associated with autism. More information about the study as well as the location of clinics currently enrolling participants can be found here: www.theautismstudy.com.
AB-5006: Novel gut-restricted candidate for Parkinson’s disease
Axial's pipeline also includes AB-5006, a gut-restricted, small molecule therapy aimed at slowing disease progression and improving the quality of life of people with Parkinson's disease (PD). AB-5006 acts by inhibiting aggregation of bacterial amyloid proteins in the gut, which Axial has shown actively contributes to alpha-synuclein pathology and motor dysfunction in animal models of PD.