Axcella Presents Late-Breaker Data Demonstrating Lead AXA™ Candidate AXA1665 Impacted Markers Related to Liver Health

“Cirrhosis complications are impacted by multiple drivers including the dysregulation of amino acid and urea cycle metabolism, muscle wasting, and gut health. There is a clear unmet need for approaches that address these multiple drivers,” said Dr. Sanyal.

AXA1665, Axcella’s lead candidate, was designed to target multiple metabolic pathways intersecting key organ systems, including liver, muscle and gut to maintain liver health. Primary assessments from the study in subjects with mild and moderate hepatic insufficiency showed that AXA1665 was well tolerated in the study, and secondary assessments demonstrated that AXA1665:

• Increased the basal (fasted) Fischer’s ratio by 40%;
• Decreased plasma ammonia area under the curve; and
• Tended to maintain a leaner phenotype and muscle function.

“I am very pleased to see additional positive results from our liver pipeline. These data suggest that AXA1665 impacts amino acid and ammonia homeostasis and tends to maintain a leaner phenotype with clinically relevant functional improvements,” said Manu Chakravarthy, M.D., Ph.D., Senior Vice President, Clinical Development and Chief Medical Officer of Axcella. “These findings support our belief that AXA1665 has the potential to directly address the multifactorial risk-factors of liver health and disease.”

“We are delighted to have these important data presented at EASL,” said Bill Hinshaw, President and Chief Executive Officer of Axcella. “AXA1665 progressed from biological hypothesis to readout of initial Non-IND, IRB-Approved Clinical Study data in less than 18 months. We are highly encouraged by these results and are actively advancing our AXA1665 clinical program. Data from our predictive, rapid and scalable human-focused AXA Development Platform continue to support EMMs as novel multifactorial assets with the potential to support health or become therapeutically relevant.”

About The International Liver Congress™

This annual congress is the biggest event in the EASL calendar, attracting scientific and medical experts from around the world to learn about the latest in liver research. Attending specialists present, share, debate and conclude on the latest science and research in hepatology, working to enhance the treatment and management of liver disease in clinical practice. This year, the congress is expected to attract approximately 10,000 delegates from all corners of the globe. The International Liver Congress™ 2019 will take place from 10–14 April 2019 at the Reed Messe Wien Congress and Exhibition Center, Vienna, Austria.

About The European Association for the Study of the Liver (EASL)

Since its foundation in 1966, this not-for-profit organization has grown to over 4,000 members from all over the world, including many of the leading hepatologists in Europe and beyond. EASL is the leading liver association in Europe, having evolved into a major European association with international influence, and with an impressive track record in promoting research in liver disease, supporting wider education and promoting changes in European liver policy.

About Endogenous Metabolic Modulators

Endogenous metabolic modulators, or EMMs, are a broad family of molecules, including amino acids, which fundamentally impact and regulate human metabolism. Our AXA Candidates are anchored by EMMs that have a history of safe use as food. We believe that, unlike conventional targeted interventions currently used to address dysregulated metabolism, EMM compositions have the potential to directly and simultaneously support and modulate multiple metabolic pathways implicated both in complex diseases and overall health.

About Non-IND, IRB-Approved Clinical Studies

Axcella conducts non-investigational new drug application (Non-IND), Institutional Review Board (IRB)-approved clinical studies in humans with its AXA Candidates under U.S. Food and Drug Administration regulations and guidance supporting research with food outside of an IND. In these studies, Axcella evaluates in humans, including in individuals with disease, AXA Candidates for safety, tolerability and effects on the normal structures and functions of the body. Non-IND, IRB-Approved Clinical Studies are not designed or intended to evaluate an AXA Candidate’s ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop an AXA Candidate as a potential therapeutic, subsequent studies will be conducted under an IND.

About Axcella Health

Axcella is designing and developing AXA candidates, compositions of EMMs engineered in distinct ratios, designed to target and maximize the fundamental role that EMMs play in regulating multiple metabolic functions. Our AXA Candidates are generated from our proprietary, human-focused AXA Development Platform. We believe our expertise and capabilities in EMMs position us to become a preeminent biotechnology company reprogramming metabolism to address a diverse set of complex diseases and support health. Our AXA Development Platform has already produced a pipeline of product candidates in programs targeting liver, muscle and central nervous system. We were founded by Flagship Pioneering.

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