Axcella Provides Progress Updates for Liver Program Candidates AXA1125 and AXA1957

• Completed enrollment in AXA1125 and AXA1957 study in adults with NAFLD
• Initiated enrollment of AXA1957 study in adolescents with NAFLD
• Developing AXA1125 and AXA1957 as therapeutic agents for NASH

CAMBRIDGE, Mass.--(BUSINESS WIRE)--October 7, 2019-- Axcella Health Inc., (Nasdaq: AXLA) (“Axcella or the Company”) a biotechnology company pioneering the research and development of novel multifactorial interventions to address dysregulated metabolism and support health, today announced liver program progress updates regarding the development of AXA1125 and AXA1957.

AXA1125 and AXA1957 are novel compositions of endogenous metabolic modulators (EMMs) designed to simultaneously support metabolic, inflammatory and fibrotic pathways associated with liver health. Axcella has completed enrollment in a 16-week, randomized, placebo-controlled, Non-IND, IRB-Approved Clinical Study to assess safety, tolerability and physiological impact of AXA1125 and AXA1957 on structures and functions in approximately 105 adult subjects with Non-Alcoholic Fatty Liver Disease (NAFLD). The Company currently anticipates the data readout from this study in the second half of 2020.

“NAFLD is one of, if not the most common chronic liver diseases we face today. The more aggressive component of NAFLD, non-alcoholic steatohepatitis (NASH), is found in up to 25% of these patients and can progress to cirrhosis, liver decompensation and hepatocellular carcinoma,” said Stephen Harrison, M.D., Professor of Hepatology at the Radcliffe Department of Medicine, Oxford University, UK and Principal Investigator on this study. “Due to this growing epidemic and lack of currently available therapies, we are keen to find effective interventions, and I am very pleased with the progress of the ongoing Axcella study.”

Additionally, in the third quarter of 2019 Axcella initiated a placebo-controlled, randomized, Non-IND, IRB Approved Clinical Study to assess safety, tolerability and physiological impact of AXA1957 on structures and functions in adolescent subjects with NAFLD. The Company anticipates the data readout from this study in the second half of 2020.

“Fatty liver in obese children is becoming a huge public health burden given its strong association with type 2 diabetes, future cardiovascular risk and many other complications,” said Professor Saul Faust M.D., Ph.D., Director of the Southampton National Institute for Health Research Clinical Research Facility and Principal Investigator on this study. “To date, we have been encouraged by the level of interest of clinical investigators, their adolescent subjects and parents in connection with participating in the ongoing Axcella study.”

Given the significant unmet need, the data generated to date and the potential of multifactorial interventions such as EMMs in liver disease, Axcella will be developing AXA1125 and AXA1957 as therapeutic candidates in NASH under an IND.

“I am delighted that we have achieved these important milestones including completing enrollment in our adult study and initiating our pediatric program,” commented Bill Hinshaw, President and Chief Executive Officer of Axcella. “Our distinct development platform has rapidly produced three therapeutic investigational liver assets, including our lead candidate, AXA1665 for the treatment of hepatic encephalopathy, or HE. We look forward to advancing these candidates as therapeutics in collaboration with the medical community, patients and their families.”

About Endogenous Metabolic Modulators

Endogenous metabolic modulators (EMMs) are a broad family of molecules, including amino acids, which fundamentally impact and regulate human metabolism. Our AXA Candidates are comprised of EMMs that individually 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 modulate multiple metabolic pathways implicated both in complex diseases and overall health.

About Non-IND, IRB-Approved Clinical Studies

Axcella conducts Institutional Review Board (IRB)-approved, non-investigational new drug application (Non-IND) clinical studies in humans with its AXA candidates under U.S. Food and Drug Administration regulations and guidance supporting research with food. 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 endogenous metabolic modulators, or EMMs, engineered in distinct ratios, designed to target and maximize the fundamental role that EMMs play in regulating multiple metabolic functions. Axcella’s AXA Candidates are generated from its proprietary, human-focused AXA Development Platform. Axcella believes its expertise and capabilities in EMMs position it to become a preeminent biotechnology company reprogramming metabolism to address a diverse set of complex diseases and support health. Axcella’s AXA Development Platform has already produced a pipeline of product candidates in programs targeting liver, muscle and blood. Axcella was founded by Flagship Pioneering. For more information, visit www.axcellahealth.com.

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Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the development potential of our AXA Candidates, including AXA1125, AXA1957 and AXA1665, potential expansion into new therapeutic fields, the ability of endogenous metabolic modulators to impact dysregulated metabolism and health and the timing of our clinical studies and the timing of receipt of data from the same. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the breadth of our pipeline of product candidates, the strength of our AXA Development Platform, the efficiency of our discovery and development approach, the clinical development and safety profile of our AXA Candidates and their health or therapeutic potential, whether and when, if at all, our AXA Candidates will receive approval from the U.S. Food and Drug Administration and for which, if any, indications, competition from other biotechnology companies, our liquidity, our ability to successfully develop our AXA Candidates through current and future milestones on the anticipated timeline, if at all, past results from Non-IND, IRB-Approved Clinical Studies not being representative of future results, and other risks identified in our SEC filings, including our Quarterly Report on Form 10-Q and subsequent filings with the SEC. We caution you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. We disclaim any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent our views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. We explicitly disclaim any obligation to update any forward-looking statements.