1. Initial Clinical Studies refers to Non-IND Clinical Studies initiated prior to a development path decision.
2. Planned Clinical Trial, contingent upon final data readouts from ongoing Clinical Studies and allowance by the FDA.
3. Indication expected to be reduction in risk of overt hepatic encephalopathy recurrence.
4. We believe that this has the potential to serve as a registrational Clinical Trial, contingent upon final data readout from ongoing Clinical Study and allowance by the FDA.
About Axcella’s Development Model and 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 a product 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 product 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, AXA1125 and AXA1957 – to be therapeutic product candidates, meaning that pending and subject to final data readouts from ongoing non-IND clinical studies and FDA feedback, any future development of these product candidates will be under IND clinical trials. These IND clinical trials would therefore be designed to evaluate each product 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/AXA1957 in IND clinical trials.
In the future, if Axcella decides to exclusively pursue therapeutic development for a new product candidate, it will initiate clinical development under an IND-enabled trial.
This page of our website refers to Axcella’s non-IND clinical studies as “Clinical Studies” and its planned IND-enabled clinical trials as “Clinical Trials.”
Note: Pipeline status depicted above is as of March 2020.