Axcella Announces Issuance of Broad Patent Covering EMM Compositions

• Covers NASH candidates AXA1125 and AXA1957 as well as other Endogenous Metabolic Modulator (EMM) compositions
• Expands Axcella’s existing composition and method of use portfolio
• Reinforces the strength of Axcella’s strategy to protect its first-mover advantage

CAMBRIDGE, Mass.--(BUSINESS WIRE) November 20, 2019 --Axcella Health (Nasdaq: AXLA), a biotechnology company pioneering the research and development of novel multifactorial interventions to address dysregulated metabolism and support health, today announced the issuance of U.S. Patent 10,471,034. This is the third EMM-related patent that Axcella has received to date, and it covers an array of EMM compositions, including AXA1125 and AXA1957, the company’s product candidates for non-alcoholic steatohepatitis (NASH). Issued earlier this month, Patent 10,471,034 broadens the coverage for composition of matter claims, independent of use.

“This issuance follows our two prior patents related to specific EMM compositions and methods of treating liver dysfunction and liver disease,” said Paul F. Fehlner, J.D., Ph.D., Senior Vice President and Chief Intellectual Property Officer of Axcella. “We believe our growing patent portfolio reinforces Axcella’s leadership position in utilizing EMMs to address metabolic dysregulation while also underscoring the pioneering nature of the company’s AXA Design Platform.”

Axcella develops novel compositions of EMMs that are designed to regulate multiple metabolic pathways simultaneously to address complex diseases and support overall health. AXA1125 and AXA1957 were formulated to impact metabolic, inflammatory and fibrotic pathways implicated in liver health and the development of NASH and non-alcoholic fatty liver disease (NAFLD). Axcella is currently conducting a non-IND clinical study to assess the impact of AXA1125 and AXA1957 on safety, tolerability and physiology in more than 100 adult subjects with NAFLD. The company also has announced its intent to file an IND for AXA1125 and/or AXA1957 for NASH.

“Our unique design platform and human-focused development model provides a powerful lens for understanding the benefits that EMM compositions can deliver in a wide range of areas,” said Bill Hinshaw, President and Chief Executive Officer of Axcella. “Over the course of the past three years, Axcella has grown its intellectual property estate in a rapid and methodical manner. With more than 130 patents pending and several issuances now complete, we believe we are well positioned to extend our first-mover advantage in harnessing the power of EMMs.”

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 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.

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 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.

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 AXA Candidates, including AXA1957 and AXA1125, potential expansion into new therapeutic fields, the ability of endogenous metabolic modulators to impact dysregulated metabolism and health and the timing of the company’s 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 the company’s pipeline of product candidates, the strength of the AXA Development Platform, the efficiency of the company’s discovery and development approach, the regulatory pathway, clinical development and safety profile of AXA Candidates and their health or therapeutic potential, whether and when, if at all, AXA Candidates will receive approval from the U.S. Food and Drug Administration and for which, if any, indications, competition from other biotechnology companies, the ability of its patent portfolio to protect its AXA Candidates, its ability to successfully develop 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 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.