Axcella Achieves Initial Milestones with its Hematology Product Candidate AXA4010

Axcella Health Inc. (Nasdaq: AXLA), a biotechnology company pioneering the research and development of novel multifactorial interventions to address dysregulated metabolism and support health, today announced key milestones in the development of AXA4010, the company’s first investigational hematology product candidate. Mechanistic data on AXA4010 will be presented at the American Society of Hematology’s (ASH) 2019 Annual Meeting. Additionally, the company has initiated enrollment of subjects with sickle cell disease (SCD) in a non-IND clinical study.

AXA4010 is composed of a novel combination of endogenous metabolic modulators (EMMs) designed to affect multiple pathways related to red cell membrane biology, hemolysis, endothelial cell and vascular function, and inflammation. Subjects with SCD have multiple metabolic derangements, making this an appropriate population to evaluate the potential impact of AXA4010 on blood structure and function.

"AXA4010 represents Axcella’s expansion beyond liver and muscle programs and demonstrates the strength and breadth of our development platform,” said Bill Hinshaw, president and chief executive officer of Axcella. "We are pleased that our data have been accepted for a presentation at ASH 2019, the world’s premier hematology congress. With a first clinical study now underway, we look forward to better understanding the safety and tolerability profile of AXA4010, investigating how it might influence blood physiology and, consequently, informing our overall development plans.”

ASH 2019 Presentation

Axcella will present mechanistic data related to AXA4010 at the ASH 2019 Annual Meeting being held December 7-10, 2019 at the Orange County Convention Center in Orlando, FL. The poster presentation will discuss metabolic profiling of SCD plasma samples and cellular data regarding the impact of AXA4010 on preclinical models of vascular adhesion, inflammation and red blood cell function.

Abstract Number: #124224
Title: A Novel Composition of Endogenous Metabolic Modulators, AXA4010, Impacts Adhesion, Inflammation and RBC Membrane Deformability in Preclinical Models of Sickle Cell Disease
Presentation Type: Poster
Session Date/Time: December 7 from 5:30-7:30 p.m. ET
Location: Orange County Convention Center, Hall B

Initiation of Non-IND Clinical Study

Axcella has enrolled its first subject with SCD in AXA4010-001 (NCT04134299), which is being conducted at multiple U.S. sites. This study is expected to enroll approximately 24 subjects ages 12 and older in a staged sequential design of three separate cohorts for up to 12 weeks. In addition to safety and tolerability, the study will assess the effects of AXA4010 on blood structure and function, including hemolysis, inflammation and vascular physiology. Axcella currently anticipates a data readout from this study in the second half of 2020.

About Endogenous Metabolic Modulators

Endogenous metabolic modulators (EMMs) are a broad family of molecules, including amino acids, which fundamentally impact and regulate human metabolism. Axcella’s AXA Candidates are comprised of EMMs that individually have a history of safe use as food. The company believes 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 in the "Investors and News” section of the company’s website. Accordingly, investors should monitor this portion of the website, in addition to following Axcella’s 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 AXA4010, 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 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 company’s liquidity, 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.

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