Our Pipeline

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.

INDICATION
ASSET
PRECLINICAL
PHASE 1
PHASE 2

Autism- Associated Irritability

Parkinson's

Disease

Oncology

AB-2004

AB-2004 PTR*

AB-5006

AB-6000 series (Program 1)

AB-6000 series (Program 2)

AB-2004: Redefining the treatment of irritability in autism

 

Axial’s lead product candidate, AB-2004, is a gut-restricted molecular therapy being developed for the treatment of irritability associated with autism (NCT04895215). AB-2004 has a unique mechanism of action of selectively sequestering certain bacterially-derived metabolites in the gut before they enter the bloodstream and reach the brain. Axial’s gut-targeted approach minimizes potential for side effects due to lack of systemic exposure of the drug. In a Phase 1b/2a clinical trial, AB-2004 was safe and well-tolerated, exhibiting no drug-related serious adverse events, consistent with a large body of existing human safety data (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.

 

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.