Benzinga | Aug 27, 2020 08:09AM EDT

Axcella Highlights Late-Breaker Data From AXA1125-003 Clinical Study Presented At The EASL Digital International Liver Congress

Poster presentation contains data demonstrating AXA1125's multifactorial effects on markers of metabolism, inflammation and fibrosis in subjects with NAFLD

Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, announced that data are being presented today in a late-breaker poster regarding key findings from its AXA1125-003 clinical study by Stephen A. Harrison, M.D., Medical Director of Pinnacle Clinical Research in San Antonio, TX, visiting professor of Hepatology at the University of Oxford, UK. The poster presentation is entitled "Multifactorial Effects of AXA1125 and AXA1957 Observed on Markers of Metabolism, Inflammation and Fibrosis: A 16-Week Randomized Placebo-Controlled Study in Subjects With Nonalcoholic Fatty Liver Disease (NAFLD) With and Without Type 2 Diabetes (T2D)."

"Based on the multifactorial effects seen in initial clinical investigations and its well-tolerated profile to date, AXA1125 is among the most intriguing candidates in development for nonalcoholic steatohepatitis (NASH)," said Dr. Harrison. "Notable reductions in virtually all non-invasive biomarkers -- including liver fat content (MRI-PDFF), alanine aminotransferase (ALT) and corrected T1 (cT1) -- were seen in subjects receiving AXA1125 versus placebo in the 003 clinical study. Interestingly, mean reductions in these measures were even greater among subjects with T2D, providing the potential for a highly differentiated profile within the NASH field."

AXA1125-003 was a placebo-controlled, randomized, multi-arm clinical study assessing the impact of AXA1125 and AXA1957 on safety, tolerability and effects on structures and functions of the liver. A total of 102 adult subjects with presumed NASH were enrolled and dosed in a 2:2:2:1 ratio to receive AXA1125, one of two AXA1957 doses, or placebo administered twice daily for 16 weeks. Study subjects were stratified based on the presence or absence of T2D, with approximately 40 percent of the subjects having T2D.

Highlights from the presentation include:

* The AXA1125 study arm consistently achieved numerically greater reductions from baseline in biomarkers of liver fat and fibroinflammation versus placebo, with AXA1957 showing activity in a number of key biomarkers, but with less consistent directional change than AXA1125

* A reduction from baseline was seen in the AXA1125 arm compared with placebo at weeks 8 and 16 in: Liver fat biomarker MRI-PDFF HOMA-IR ALT CK-18 M65 ProC3 cT1

* In the AXA1125 arm, 39 percent of subjects achieved a ?30 percent relative reduction in MRI-PDFF, 39 percent achieved a ?17 U/L reduction in ALT from baseline, and 54 percent achieved a ?40 millisecond absolute reduction in cT1

* AXA1125 and AXA1957 were both generally well tolerated in the study, with low rates of discontinuations and with all product-emergent adverse events being mild or moderate

As announced earlier this year in conjunction with Axcella's top-line data announcement, the company plans to advance AXA1125 into a Phase 2b clinical trial in adult NASH in the first half of 2021.