Axcella Completes Enrollment of Subjects in AXA1665-002 Study

Axcella (Nasdaq: AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced that it has completed subject enrollment in its ongoing AXA1665-002 clinical study. This 12-week, placebo-controlled, randomized study is investigating the safety, tolerability and physiological impact of two doses of AXA1665 in 60 subjects with mild and moderate hepatic insufficiency. Ongoing safety monitoring in this study supports that both doses of AXA1665 have been well tolerated to date. Additionally, a comprehensive panel of biomarkers are being measured related to amino acid metabolism, ammonia, muscle structure and function including body composition, and neurocognition.

"We continue to be pleased by the speed at which we have been able to enroll subjects in our clinical studies, including AXA1665-002. We believe this is due in large part to the motivation of investigators to work with us and their intrigue about the potential impact of our EMM compositions,” said Bill Hinshaw, President and CEO of Axcella. "We look forward to completing this ongoing clinical study and sharing top-line data in mid-2020. Assuming supportive data and FDA feedback, we plan to initiate an IND-enabled potential Phase 2b/3 registrational clinical trial in the second half of 2020. We believe EMMs may benefit many people who are afflicted by complex diseases, including overt hepatic encephalopathy (OHE), which is a condition for which patients still have limited treatment options.”

Axcella plans to investigate AXA1665 as an oral product candidate for overt hepatic encephalopathy, a disease that is associated with amino acid imbalance, dysregulated ammonia metabolism, and muscle wasting.

About Endogenous Metabolic Modulators (EMMs)

EMMs are a broad family of 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 root causes of various complex diseases and improve health.

About Axcella’s Ongoing Clinical Studies

Each of the company’s ongoing clinical studies are being conducted as non-investigational new drug (IND) application clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate’s ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125/1957, subsequent studies will be conducted under an IND.

Internet Posting of Information

Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company’s website in the "Investors and News” section. Accordingly, investors should monitor such portions of the company’s website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

About Axcella

Axcella is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company’s product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella’s pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the prevention of recurrent overt hepatic encephalopathy (OHE). Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

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 the company’s EMM product candidates, including AXA1665, to impact dysregulated metabolism and benefit individuals with complex diseases, the potential of AXA1665 to benefit patients with OHE, the timing of the company’s ongoing clinical studies and planned IND-enabled clinical trials and the timing of receipt and disclosure 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 and potential uses of the company’s pipeline of product candidates, including the potential for AXA1665 to benefit OHE patients, whether planned data readouts and disclosures are positive and support our believes regarding EMMs and AXA1665’s potential ability to benefit not just healthy but also diseased patients, including patients with OHE, and the planned timing of our disclosures regarding data readouts, whether data readouts and/or FDA feedback support our planned timing for an IND filing, clinical trial design and target indication for AXA1665, the strength of the AXA Development Platform, the efficiency of the company’s discovery and development approach, the clinical development and safety profile of the company’s product candidates and their health or therapeutic potential, whether and when, if at all, the company’s product candidates will receive approval from the U.S. Food and Drug Administration, or other comparable regulatory authorities, and for which, if any, indications, competition from other biotechnology companies, the company’s liquidity, its ability to successfully develop product candidates through current and future milestones on the anticipated timeline, if at all, past results from non-IND clinical studies not being representative of future results, and other risks identified in the company’s SEC filings, including Axcella’s Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims 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 the company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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