Study: AXA1125 as a Treatment for Nonalcoholic Fatty Liver Disease

Axcella, a biotechnology company based out of Cambridge, Massachusetts, has released the results of AXA1125-003. According to Biotech 365, this study showed positive results for those with non-alcoholic fatty liver disease (NAFLD).

About Non-Alcoholic Fatty Liver Disease

NAFLD is an umbrella term for a number of liver conditions that affect those who consume little to no alcohol. It is characterized by an excess of fat in the liver. Those affected usually do not experience symptoms, but when they do occur they include fatigue and pain in the upper right section of the abdomen. While people may be asymptomatic, it is important to seek treatment as cirrhosis of the liver may occur. The exact reason for this condition is unknown, but medical professionals have linked it to obesity, high levels of fat in the blood, insulin resistance, and high blood sugar.

Doctors recommend eating a healthy diet, exercising, and maintaining a healthy weight to lower one’s risk of NAFLD. In fact, treatment for this condition consists of losing weight and maintaining a healthy lifestyle. If cirrhosis does occur, a liver transplant may be necessary.


This study was randomized, placebo-controlled, and multi-arm and meant to evaluate safety, tolerability, and efficacy of AXA1125 and AXA1957, both of which are oral drugs composed of amino acids and derivatives meant to support liver health. In order to assess these things, researchers used imaging tests and biomarkers. 102 participants were enrolled and given either AXA1125, AXA1957, or a placebo twice every day for 16 weeks.

Results showed that both of these medications were well-tolerated and favorable in efficacy over the placebo. Both saw sustained reductions of key biomarkers. Between the two drugs, AXA1125 produced better results.

Researchers are very pleased with these results, as they point to AXA1125 being the first therapy that directly treats NAFLD without changing the patient’s body weight or serum lipids. Hopefully this therapy will continue in its development and better the lives of those with NAFLD.

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