Benzinga | Aug 16, 2021 07:58AM EDT

Axcella Highlights Publication In The American Journal Of Gastroenterology Of Results From AXA1125-003 In Subjects With Presumed NASH

AXA1125 demonstrates meaningful reductions in key measures of liver metabolism, inflammation, and fibrosis

Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced the publication of results from the company's AXA1125-003 clinical study in The American Journal of Gastroenterology entitled "Safety, Tolerability, and Biologic Activity of AXA1125 and AXA1957 in Subjects With Nonalcoholic Fatty Liver Disease." Based on the positive findings from this study, Axcella recently initiated its EMMPACT Phase 2b clinical trial of AXA1125 in patients with biopsy-confirmed nonalcoholic steatohepatitis (NASH).

"As our largest clinical investigation completed to date, AXA1125-003 served as an important confirmation of AXA1125's potential to generate meaningful benefits for patients with NASH utilizing a multi-targeted mechanism," says Dr. Alison Schecter, Axcella's President of Research and Development. "We are pleased to share data in this important publication to broaden awareness about this EMM composition's potential to serve as a compelling first-line treatment for NASH. On behalf of our team, I would like to thank the participants who took part in the study."

AXA1125-003 was a placebo-controlled, randomized, multi-arm clinical study that enrolled 102 subjects with presumed NASH and assessed the impact of AXA1125 and AXA1957 on safety, tolerability and effects on structures and functions of the liver, as measured by a comprehensive panel of imaging and soluble biomarkers related to metabolism, inflammation, and fibrosis. Study subjects were stratified based on the presence or absence of type 2 diabetes.

Results from the study showed that AXA1125 and AXA1957 were generally well-tolerated, with sustained reductions noted for both product candidates versus placebo in key biomarkers of metabolism, inflammation and fibrosis over 16 weeks. Overall, as compared to placebo, AXA1125 demonstrated larger and more consistent reductions in clinically relevant biomarkers than AXA1957. Among subjects receiving AXA1125, 39% achieved a ?30% relative reduction in liver fat content (MRI-PDFF), 39% achieved a ?17 U/L reduction in alanine aminotransaminase (ALT; a marker of inflammation), and 35% achieved a ?80 mSec reduction in corrected T1 (cT1; a marker of fibrosis). Among subjects with type 2 diabetes receiving AXA1125, a greater proportion achieved each of these thresholds. Emerging evidence suggests that these thresholds of activity increase the likelihood of histopathological improvement in NASH subjects. Notably, the above results were seen without impacting mean body weight or serum lipids.

Initiated in April 2021, EMMPACT is an ongoing randomized, double-blind, placebo-controlled, multi-center Phase 2b clinical trial that is evaluating the efficacy and safety of AXA1125 in patients with biopsy-confirmed F2/F3 NASH. Approximately 270 patients are being enrolled and randomized 1:1:1 to receive either 45.2 or 67.8 grams per day of AXA1125 or a matched placebo in two divided doses for 48 weeks, with a four-week safety follow-up period. Patients are stratified based on the presence or absence of type 2 diabetes. Additional information can be found on https://clinicaltrials.gov/ via the identifier NCT04880187.