Axcella Health (AXLA) Announces AXA1125's IND Clearance for Treatment of NASH

April 13, 2021 7:28 AM EDT
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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 that it has achieved a key milestone with U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for AXA1125, enabling the company to proceed directly into a Phase 2b clinical trial. AXA1125 is Axcella’s multi-targeted oral product candidate for nonalcoholic steatohepatitis (NASH), a chronic and progressive liver disease impacting up to 40 million people in the U.S. alone.

“This IND follows close on the heels of AXA1665’s IND clearance earlier this year, ushering in an exciting new era for Axcella as we seek to tackle a variety of complex diseases and address important unmet needs for patients utilizing multi-targeted EMM compositions,” said Bill Hinshaw, President and Chief Executive Officer of Axcella. “I am pleased by our team’s strong execution in completing two robust FDA submissions and readying us for the start of our highly efficient, later-stage clinical trials.”

Axcella expects to initiate its Phase 2b clinical trial in the second quarter of 2021. This randomized, double-blind, placebo-controlled, multi-center trial will evaluate the efficacy, safety and tolerability of AXA1125 in adult patients with biopsy-confirmed F2/F3 NASH. Approximately 270 patients will be 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 will be stratified based on the presence or absence of type 2 diabetes.

The trial will be conducted globally across more than 70 clinical sites with a primary endpoint assessing the proportion of patients with a biopsy-confirmed ≥2 point improvement in NAFLD Activity Score (NAS) after the 48-week treatment period. Secondary endpoints will include the proportion of patients achieving biopsy-confirmed resolution of NASH without worsening of fibrosis and the proportion of patients achieving a ≥1 stage improvement in fibrosis without worsening of NASH. A range of non-invasive biomarkers, including MRI-PDFF and Fibroscan, will be utilized for additional endpoints and an interim analysis in the trial.

“AXA1125 leverages a mechanism that engages multiple pathophysiologic pathways involved in the development of NASH using a modality with well-precedented safety, which is particularly important given the many comorbidities associated with this chronic disease,” said Alison Schecter, M.D., President of R&D at Axcella. “In past clinical studies, compelling activity has been seen with AXA1125 across a range of non-invasive biomarkers, with enhanced effects in type 2 diabetics. Our goal in the Phase 2b is to affirm AXA1125’s impact via histology and further strengthen its profile as a compelling candidate for first-line treatment in NASH.”

The upcoming Phase 2b clinical trial follows two earlier non-IND clinical studies in which reductions were seen with AXA1125 in key measures of hepatic fat, insulin resistance, inflammation and fibrosis with a safe and well tolerated profile. Presentations containing those findings can be found by visiting

About AXA1125 and Nonalcoholic Steatohepatitis (NASH)

NASH is the most severe form of non-alcoholic fatty liver disease (NAFLD). This chronic, complex disease is associated with significant morbidity and mortality globally, and it is estimated to impact up to 40 million Americans, including up to 10% of American children. Despite its severity and increasing prevalence, there are currently no approved therapies for NASH in the United States.

AXA1125, Axcella’s NASH product candidate, is a composition of six amino acids and derivatives that is designed to target multiple metabolic pathways involved in NASH’s progression from metabolism to inflammation to fibrosis. In prior clinical studies, this oral product candidate has been safe and well tolerated and has demonstrated clinically meaningful reductions in a range of well-accepted, non-invasive NASH biomarkers, with the most pronounced activity noted in subjects with type 2 diabetes. AXA1125 is now entering Phase 2b development in adults with NASH. Axcella also plans to investigate AXA1125 in pediatric NASH.

About Endogenous Metabolic Modulators (EMMs)

EMMs are a broad family of naturally occurring molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the underlying causes of various complex diseases and improve health.

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