Our Pipeline

Therapeutic Candidates

We have developed a wholly owned therapeutic pipeline that today consists of product candidates for two liver diseases, nonalcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. We have completed initial Clinical Studies of AXA1665 and AXA1125, and expect to initial Clinical Trials under IND with both of these product candidates in the first half of 2021.

Product Candidate Area of Focus Potential Indication Initial Clinical Studies1 Therapeutic Development2
AXA1125 Carat Liver NASH
N=32, 1 arm, 1 dose
N=102, 4 arms, 1 AXA1125 dose
Adult Trial: Ph2b
Initiation: 1H 2021

Pediatric Trial: TBD
Initiation: TBD

AXA1125 was designed to target multiple metabolic pathways supporting liver health. Initial clinical data has demonstrated AXA1125’s potential to influence three key nodes:

  • Metabolism
  • Inflammation
  • Fibrosis
Learn more about AXA1125
AXA1665 Carat Liver Overt Hepatic
N=16, 2 periods, 2 doses
N=60, 3 arms, 2 doses
Next Trial: Ph2
Initiation: 1H 2021

AXA1665 was designed to target multiple metabolic pathways supporting liver health. Initial clinical data has demonstrated AXA1665’s potential to influence three key nodes:

  • Ammonia handling
  • Amino acid balance
  • Muscle wasting
Learn more about AXA1665



  1. Initial Clinical Studies refers to Non-IND Clinical Studies initiated prior to a development path decision.
  2. Planned Clinical Trials, contingent upon allowance by the FDA. Timing based on current expectations and subject to risks, including those associated with the COVID-19 pandemic.
  3. Indication expected to be reduction in risk of overt hepatic encephalopathy recurrence.

Pipeline image as of August 5, 2020.

Early-stage pipeline
Group 35

Explore AXA1665

AXA1665 is our product candidate for the reduction in the risk of OHE recurrence.

Learn more

Explore AXA1125

AXA1125 is our product candidates for NASH.

Learn more

Areas of Application



The liver plays a key role in eliminating waste and metabolizing carbohydrates, lipids, and proteins. Therefore, decreased liver function can result in or contribute to loss of health and complex systemic diseases.

Our lead product candidates are for NASH and reduction in the risk of OHE recurrence. OHE is a serious complication of liver cirrhosis caused by an imbalance of amino acids and the body’s inability to process ammonia, which prompts muscle wasting and cognitive issues (encephalopathy). NASH is a complex systemic disease with three central nodes: inflammation, liver fibrosis, and dysregulated metabolism.



The circulatory system provides transportation of oxygen, cellular waste products, nutrients, and hormones. The function of blood cells and pathways of inflammation are critically interconnected with multiple metabolic processes.

We are in the early stages of development for a hematology product candidate, AXA4010. We believe that EMMs have the potential to support the normal function of red blood cells by enhancing the synthesis of hemoglobin, reducing inflammation, and improving vascular function.



The structure and function of skeletal muscle is not only essential for mobility, it also contributes to energy metabolism. Additionally, a decline in muscle mass has been associated with morbidity and mortality in chronic liver, kidney, and heart diseases.

We are in the early stages of development with our muscle product candidate, AXA2678, designed to support metabolic pathways relevant to muscle health. We believe EMMs have the potential to support protein synthesis in muscle, reduce inflammation, and support the development of muscle fibers.

Check out our latest publications, presentations and posters

We are publishing innovative research and presenting at leading conferences.

View publications

Our Clinical Approach

EMMs have a fundamental role in biology and function. Using the Axcella Knowledge Base, Axcella designs and develops novel EMM compositions to engage identified biologies and pathways. Axcella then selects whether to evaluate the candidate in a non-investigational new drug application (non-IND) Clinical Study under U.S. Food and Drug Administration regulations and guidance supporting research with food, or under an IND Clinical Trial. Axcella’s non-IND Clinical Studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. The company’s non-IND Clinical Studies include a substantial number of biomarkers that may inform biologies relevant to health but are not designed or intended to evaluate a product candidate’s ability to diagnose, cure, mitigate, treat or prevent a disease or other health condition. They are conducted at high-quality medical centers following Good Clinical Practices (GCPs), including Institutional Review Board (IRB) approval, and utilize well-recognized investigators. Using a combination of data from these studies and/or other relevant information, the company decides whether to advance a candidate’s development as a therapeutic or supplement (independently or in partnership), or terminate its development.

To date, Axcella has evaluated its current product candidates as investigational food products in non-IND Clinical Studies. Axcella has determined its lead compounds – AXA1665 and AXA1125 – to be therapeutic product candidates, meaning that pending and subject to final data readouts from ongoing non-IND Clinical Studies, any future development of these products will be under IND Clinical Trials. These IND Clinical Trials would therefore be designed to evaluate each candidate’s ability to diagnose, cure, mitigate, treat or prevent targeted diseases. The company’s current plans are to target OHE with AXA1665 and NASH with AXA1125 in IND Clinical Trials.

In the future, the company may choose to advance new product candidates with therapeutic potential directly into a Clinical Trial under IND.

This website refers to Axcella’s non-IND Clinical Studies as “Clinical Studies” and its planned IND-enabled Clinical Trials as “Clinical Trials.”

Our Platform