The Food and Drug Administration (FDA) has granted Fast Track designation to AXA1125 for the treatment of nonalcoholic steatohepatitis (NASH) with liver fibrosis.

AXA1125 is an orally active mixture of amino acids (endogenous metabolic modulator compositions) designed to target pathways related to metabolism, inflammation, and fibrosis in NASH. The Company is currently investigating AXA1125 in patients with noncirrhotic NASH with fibrosis in the phase 2b EMMPACT trial ( Identifier: NCT04880187).

“Despite the fact that NASH is an epidemic that is impacting tens of millions of people here in the US alone, there are no approved treatments today,” said Axcella President and CEO Bill Hinshaw. “We are pleased that, after reviewing the compelling data from our NASH program including those from 2 prior clinical studies, the FDA recognized AXA1125’s potential to address the needs of patients with this serious and chronic disease.”

The multi-targeted mechanism of action was described in a 16-week, randomized clinical study published in the American Journal of Gastroenterology. Additionally, AXA1125-associated metabolic profile changes were reported in a poster presentation at AASLD’s The Liver Meeting 2021. 

AXA1125 is also being evaluated as a potential treatment for patients with fatigue-predominant post-acute sequelae of SARS-CoV-2 in a phase 2a trial ( Identifier: NCT05152849).


Axcella Therapeutics announces FDA Fast Track designation for AXA1125 in NASH. News release. Axcella Therapeutics. Accessed February 14, 2022.