Benzinga | Sep 9, 2021 09:49AM EDT

Axcella's First-of-Its-Kind NASH Treatment Shows Promising Early Results in Human Trials

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The American Journal of Gastroenterology recently published findings from a clinical study of AXA1125, a groundbreaking potential treatment for nonalcoholic steatohepatitis (NASH) developed by Axcella Health Inc. (NASDAQ:AXLA). The pioneering clinical-stage biotech company has shown the potential to reset multiple targeted biologies that are dysregulated in patients with complex diseases using proprietary combinations of amino acids and metabolites. AXA1125 combines six amino acids to treat the drivers of NASH -- a harmful and progressive liver disease that currently impacts up to 40 million Americans and still is without an FDA-approved treatment.

The Need for an Effective Treatment for NASH

NASH is an advanced type of liver disease that can develop when nonalcoholic fatty liver disease (NAFLD) goes untreated -- something that can happen often since NAFLD typically has few or no symptoms beyond a mild sense of fatigue or discomfort that patients might not consider serious enough to seek a diagnosis for.

NAFLD is now the most common chronic liver condition in the United States, affecting an estimated 80 to 100 million Americans. According to a recent study from the Global Liver Institute, up to 40 million of these patients, including up to 10% of our children, have progressed to the advanced disease NASH, and this population is expected to continue growing.

With no FDA-approved treatment yet available, patients with NASH are limited to lifestyle changes that are aimed at driving significant weight loss, which many patients find difficult to achieve and maintain. As the disease progresses, NASH can lead to cirrhosis, the need for liver replacement, liver cancer and death.

The prevalence and severity of NASH has sparked a race to develop effective therapies for the disease. Globally, this unmet need already represents a more than $1.1 billion market, even without an FDA-approved drug, and is expected to balloon to $21.4 billion by 2025 as more and more manufacturers join the race to find an effective treatment.

AXA1125 Moves Closer to Becoming the Multitargeted Treatment Patients Need

Axcella is a top contender in that race. With AXA1125 now being investigated in a Phase 2b clinical trial, the clinical-stage company has already seen promising results. The drug candidate is a combination of six amino acids that work by resetting disease pathways related to liver metabolism, inflammation and fibrosis (liver scarring).

NAFLD and NASH are conditions in which the liver can't properly metabolize fatty acids or carbohydrates, leading to a toxic build up of lipids and increased insulin resistance. AXA1125 has shown the potential to jumpstart that metabolism while also impacting downstream drivers of liver scarring.

The multitargeted drug candidate has been investigated in two prior clinical studies, including one that was completed in 2020. The study enrolled 102 subjects with presumed NASH and found that those who were given AXA1125 saw significant reductions in key measures of liver fat, inflammation and fibrosis compared with those given a placebo.

In fact, nearly 40% of the people who received AXA1125 saw their liver fat reduced by at least 30% in just 16 weeks. Additionally, AXA1125's effects were even more notable among those subjects who had presumed NASH and type 2 diabetes. AXA1125 was also safe and well tolerated in this study.

Bolstered by these strong findings, Axcella launched a Phase 2b clinical trial in April 2021. This study will enroll and monitor 270 subjects over the course of 48 weeks to assess the longer-term potential of AXA1125 as a treatment for NASH.

With early trials already showing significant positive results, AXA1125 could become an FDA-approved treatment for NASH, finally offering the millions of patients battling the disease a potential way to avoid its often deadly consequences.