Our Pipeline

Therapeutic Candidates

We have developed a wholly-owned therapeutic pipeline that today consists of product candidates for three indications: the reduction in risk of recurrent overt hepatic encephalopathy (OHE), the treatment of nonalcoholic steatohepatitis (NASH) and the treatment of Long COVID. We have completed initial Clinical Studies of AXA1665 and AXA1125, and now have later-stage Clinical Trials underway or planned for each of these indications.

Product Candidate Potential Indication Initial Clinical Studies1 Therapeutic Development2
AXA1665 Carat Overt Hepatic Encephalopathy
AXA1665-001 N=16, 2 arms, 2 doses
AXA1665-002 N=60, 3 arms, 2 doses
EMMPOWERSM Phase 2 Clinical Trial N=~150, 2 arms, 1 dose
AXA1125 Carat Adult NASH
AXA1125-002 N=32, 1 arm, 1 dose
AXA1125-003 N=102, 4 arms,
1 AXA1125 dose
EMMPACTSM Phase 2b Clinical Trial N=~270, 3 arms, 2 doses
AXA1125 Carat Pediatric NASH
Next Step: Regulatory Engagement
AXA1125 Carat Long COVID
Phase 2a Clinical Trial N=~40, 2 arms, 1 dose




  1. Initial Clinical Studies refers to Non-IND Clinical Studies initiated prior to a development path decision.

Pipeline image as of October, 2021.

Group 35

Explore AXA1665

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

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Explore AXA1125

AXA1125 is our product candidates for NASH.

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We are publishing innovative research and presenting at leading conferences.

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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 initially evaluated its 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 all future development of these products will be under IND Clinical Trials. These IND Clinical Trials are designed to evaluate the ability to diagnose, cure, mitigate, treat or prevent targeted diseases. The company is targeting 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 IND-enabled Clinical Trials as “Clinical Trials.”

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