Will Data From Liver Disease, Sickle Cell Disease Trials Give Axcella A Boost?

Shares of Axcella Health Inc. (AXLA) have been dismally performing since their listing on the NASDAQ on May 9, 2019. The stock is down more than 70 percent over the last 1 year.

Axcella is a clinical-stage biotechnology company focused on developing novel multi-targeted EMM compositions that have potential applications in complex diseases.

What are EMMs?

EMMs or endogenous metabolic modulators are a set of molecular families, including amino acids, bile acids, other intermediary substrates, and hormones. They serve as master regulators and signaling agents for metabolic pathways, providing the potential for a multifactorial approach to treat complex diseases, according to the Company.

Pipeline:

The Company has three investigational drugs – AXA1665 for the reduction in risk of overt hepatic encephalopathy (OHE) recurrence, AXA1125 for nonalcoholic steatohepatitis (NASH) and AXA4010 for sickle cell disease.

Overt hepatic encephalopathy is one of the most common complications of cirrhosis, a late-stage liver disease. It is characterized by a decline in brain function due to a buildup of toxins – primarily ammonia – in the blood.

AXA1665, an EMM composition of 8 amino acids and derivatives, works by targeting ammonia toxicity, amino acid imbalance, and muscle wasting.

A 12-week, placebo-controlled, randomized study investigating the safety, tolerability, and physiological impact of two doses of AXA1665 in 60 subjects with mild and moderate hepatic insufficiency is underway.

Nonalcoholic steatohepatitis (NASH), a condition caused by a buildup of fat in the liver, is characterized by liver inflammation and damage.

A randomized, single blind study that evaluated whether AXA1125 or AXA1957 were safe and well-tolerated in subjects with non-alcoholic fatty liver disease (NAFLD) was successfully completed earlier this month. The trial showed clinically relevant reductions in liver fat content, insulin resistance, and fiber inflammation in subjects with NAFLD receiving AXA1125.

An open-label study evaluating the safety and tolerability of AXA4010 in adult and adolescent subjects with sickle cell disease is ongoing.

Near-term Catalysts:

— The top-line data from the study of AXA1665 in subjects with mild and moderate hepatic insufficiency is expected in the third quarter of 2020.

— The Company is planning to seek FDA clearance to initiate a potential phase IIb/III registrational clinical trial of AXA1665 for the reduction in risk of OHE recurrence in the fourth quarter of this year.

— Discussion with the FDA regarding a proposed phase IIb clinical trial of AXA1125 in adults and pediatric NASH patients is scheduled for the second half of the year.

— The top-line data from Cohort 1 of the AXA4010 trial in sickle cell disease is expected in the fourth quarter.

Cash position:

The Company, which ended March 31, 2020, with cash and cash equivalents of $75.5 million, raised gross proceeds of about $60.1 million in a public offering this month.

AXLA has traded in a range of $2.25 to $12.25 in the last 1 year. The stock closed Wednesday’s trading at $4.20, down 0.47%.

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