Axcella Announces Positive Interim Data from Phase 2b EMMPACT Study of AXA1125 in Nonalcoholic Steatohepatitis (NASH)

Subjects enrolled with biopsy confirmed NASH experienced clinically and statistically significant improvements in liver stiffness as measured by FibroScan, a non-invasive measure of liver fibrosis

Subjects with NASH experienced clinically and statistically significant improvements in alanine aminotransferase (ALT), a measure of liver cell inflammation, at both dose levels of AXA1125

Findings demonstrate improvement in hepatic fat as measured by MRI-PDFF

AXA1125 continues to demonstrate a safe and well tolerated profile

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Excerpt from the Press Release:

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Axcella Therapeutics (Nasdaq: AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using multi-targeted endogenous metabolic modulator (EMM) compositions, today reported interim results from their ongoing global Phase 2b randomized, double-blind, placebo-controlled, dose ranging EMMPACT study to evaluate the safety, tolerability, and efficacy of AXA1125 for the treatment of NASH. These interim results report findings regarding the effects of AXA1125 administration on selected outcome measures after 12- and 24-weeks of treatment.

This interim analysis was preplanned to be conducted when enrollment reached 30% of the target of 270 subjects with biopsy confirmed stage 2 or 3 NASH across all trial arms. Data from this ongoing blinded study included 82 subjects at week 12 and 58 subjects at week 24; approximately half of the subjects have type 2 diabetes mellitus (T2DM). In addition to effects on hepatic fat and ALT, previously reported in 2 other studies, this study also included vibration controlled transient elastography (FibroScan), a widely accepted and accessible non-invasive test (NIT) that assesses both liver fat and stiffness. Specifically, the study examines liver stiffness, changes of which have been correlated with improvements in liver fibrosis and outcomes in clinical studies. Study participants were randomized 1:1:1 to receive either a placebo or 22.6g or 33.9g of AXA1125 twice daily.

At 24-weeks there were statistically significant improvements in the liver stiffness measurement (LSM) compared to placebo in the high dose arm for all subjects. Absolute changes in LSM were 0.13, -2.01, and -4.07 kilopascals (kPa) in the placebo, low dose and high dose arms, respectively (p= 0.0992 and 0.0096 for the low and high dose, respectively, compared to placebo).

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