Pioneering the development of Microbial Inspired Therapeutics®

Axial is a scientific leader focusing on the interface between the gut microbiome and the human body. Rooted in over a decade of ground-breaking research, Axial employs unique and proprietary preclinical models to identify and validate targets and generate drug candidates designed to address the microbiome-driven pathology of a particular disease.

Axial’s Microbial Inspired Therapeutics® discovery platform has the potential to treat any disease impacted by the microbiome. Axial is translating its platform into a clinical pipeline of small molecule therapeutics with an initial focus on autism (Phase 2) and Parkinson’s disease (Phase 1 in 2024), and with emerging programs in liver disease and oncology.

The Microbiome Gut Interface: An Untapped Source for New Drug Targets

Microbiome Gut Interface - infographic

Axial is identifying new drug targets in the microbiome enabling the discovery and development of gut-selective small molecule therapeutics that can be broadly applied to many diseases.  

The Intersection of the Gut Microbiome and Disease

The human gut is home to a variety of microbes, including over 10 trillion bacteria and 500 bacterial species. This collection of organisms and their components (genes) are called the microbiota and microbiome, respectively.

Most of the bacteria in the gut are necessary and beneficial to human health, contributing to critical daily functions including digestion, metabolic function, immune system balance, and mental health. While these bacteria most often exist together in balance, when this balance is disrupted, a condition called dysbiosis, it can contribute to or cause disease.

Many diseases and disorders are associated with dysbiotic gut microbiome, but it is not always clear whether the altered microbiome is a contributor to disease or a consequence of the disease, or both. Understanding if and how the gut microbiome contributes to disease is essential for determining whether a new therapeutic may be developed by targeting the gut microbiome.

Central to determining whether and how the gut microbiome contributes to disease requires that we understand the different avenues of bidirectional communication between the gut microbiota and the human body. Three of the most studied avenues include the autonomic nervous system, the circulatory system and the immune system.

  • The autonomic nervous system includes: 1) the vagus nerve, which represents the main component of the parasympathetic nervous system, oversees a vast array of crucial bodily functions, including control of mood, immune response, digestion, and heart rate; 2) the enteric nervous system, sometimes referred to as our ‘second brain,’ which consists of hundreds of millions of neurons that extend along the length of the digestive tract and communicates bidirectionally between the GI tract and the brain. Together with the gut microbiota, these are often referred to as the “gut-brain axis”.
  • The circulatory system: Bacteria in the gut produce certain substances called metabolites that can enter the bloodstream from the gut, circulate throughout all tissues in the body, including the brain. Some of these metabolites have been shown to influence brain function and structure and contribute to neurological diseases and conditions.
  • The immune system: Most of the body’s immune cells exist in the gut where microbes are constantly educating and challenging our immune system. In doing so, this enables our immune system to mature and be ready to respond to threats such as infectious disease and cancer. Many other diseases, including neurological diseases such as Parkinson’s, Alzheimer’s, and multiple sclerosis, are associated with inflammation in the brain and central nervous system, a condition called “neuroinflammation”. Gut microbes and their products have been shown to be significant contributors to neuroinflammation.

Blazing a New Trail in the Treatment of Neurological Diseases and Disorders

Many studies clearly demonstrate that changes in the gut microbiome correlate with disease. Our science and our technology platform allow us to move beyond correlation. Our focus on the microbiome gut-brain axis represents a transformative therapeutic approach with the potential to treat neurological diseases and disorders in ways never before thought possible.

Recent research has shown that alterations and irregularities in the gut microbiome influence functions controlled by the nervous system, including emotions, communication, anxiety, stress response and motor function. Our science is rooted in the cutting-edge research of our scientific co-founder Dr. Sarkis Mazmanian at Caltech. Dr. Mazmanian’s groundbreaking work established several key concepts in the biology of the gut-brain axis, its role in neurodegenerative diseases and neurodevelopmental disorders, and the basis of the influence of the gut microbiome in people with autism and Parkinson’s disease (PD). We are applying this research to the discovery and development of Microbial Inspired Therapeutics® across multiple indications for people who need them.