Robust Technology to Target Disease
The technical foundation of Axial’s unique gut-targeted therapeutic approach is a robust platform for the identification of new disease targets and the design and delivery of small molecule drugs with an initial focus in neurodegenerative diseases and neurodevelopmental disorders. Our scientific framework provides the platform and data for establishing the mechanistic role of the microbiome in disease pathology and its influence on disease symptoms.
Leveraging our Microbial Inspired Therapeutics® Discovery Platform, Axial Establishes the Role of the Gut Microbiome in Disease Through a Rigorous Scientific Process:
Establish causation: show the microbiome actively contributes to a disease/condition (human samples, germ free mouse models, humanized mouse models)
Identify targets: identify specific products of the microbiome that are elevated or deleted in people with a given disease or condition (data mining using metagenomics, metabolomics, clinical data)
Validate targets: demonstrate that manipulation of targets (turning on/off or dialing up or down) leads to changes in disease severity, disease course, or onset (engineered mouse models)
Determine target “druggability”: demonstrate that gut-selective small molecules modulate a target and lead to a predicted pharmacological effect (medicinal chemistry and engineered mouse models)
Optimize leads: optimize small molecules for clinical evaluation with a focus on chemistry, ADME/pharmacokinetics and pharmacology
Identify biomarkers: establish traditionally relevant disease biomarkers that can be assessed in clinical trials
Axial’s goal is to identify and validate targets in the microbiome that contribute to disease and then develop gut-selective small molecule drugs against those targets. Our multi-disciplinary team of microbiologists, biochemists and medicinal chemists, pharmacologists and clinical experts have allowed us to create a novel and groundbreaking approach to drug development. We believe our approach to developing drugs that target the microbiome to treat a broad array of diseases will deliver first-in-class medicines for the many patients who need them.
