Axcella™ is developing a wholly-owned pipeline currently comprising five programs focused on the metabolic functions of the liver, muscle and blood. AXA1665 is our first drug product candidate for hepatic encephalopathy (HE). We have not made a development path decision for any of our other AXA Candidates. An overview of our AXA™ Candidates and their status are illustrated below.
Definitions: NAFLD = non-alcoholic fatty liver disease; NASH = nonalcoholic steatohepatitis; OHE = overt hepatic encephalopathy.
1. In the above pipeline chart, ‘‘Development Path Decision’’ reflects the point in a program at which we decide whether to develop an AXA Candidate as a drug product candidate under an IND, develop it as a non-drug product candidate, or terminate development. We have made a decision to develop AXA1665 as a drug product candidate and anticipate interacting with the FDA again prior to submitting an IND. We have not made a development path decision for any of our other AXA Candidates. “Non-IND, IRB-Approved Clinical Studies’’ to refer to Institutional Review Board-approved, or IRB-approved, clinical studies conducted in humans with our AXA Candidates under U.S. Food and Drug Administration, or FDA, regulations and guidance supporting research with food outside of an IND (prior to any decision to develop an AXA Candidate as a drug product under an IND or a non-drug product). In these food studies, based on our understanding of FDA regulations and guidance, we evaluate in humans, including individuals with disease, an AXA Candidate for safety, tolerability and effects on the normal structures and functions of the body. These studies are not designed or intended to evaluate an AXA Candidate’s ability to diagnose, cure, mitigate, treat or prevent a disease, as these would be evaluated in clinical trials under an IND if we decide to develop an AXA Candidate as a drug or therapeutic.
2. We believe that this Clinical Trial has the potential to serve as a registrational (pivotal) Clinical Trial, subject to continuing IND discussions and allowance by the FDA.