Axcella Announces Publication of Data Describing Attenuation of Muscle Atrophy with AXA2678 in Short-Term Muscle Disuse Study

• Administration of AXA2678 was well-tolerated with no significant safety issues over 28 days
• AXA2678 attenuated muscle mass loss and reduced intramuscular fat accumulation in healthy young male study subjects
• Data supports the ability to use endogenous metabolic modulators to address dysregulated metabolism and support health

CAMBRIDGE, Mass.--July 31, 2019--Axcella Health, (Nasdaq: AXLA) (“Axcella” or the “Company”), a biotechnology company pioneering the research and development of novel multifactorial interventions using novel compositions of endogenous metabolic modulators to address dysregulated metabolism and support health, announced the publication of data describing the safety, tolerability and impact of investigational product AXA2678 on short-term muscle disuse atrophy in young healthy men.

“Muscle disuse leads to significant reduction in both mass and function, and often does not completely recover even upon return to normal activity. The observed positive effects with AXA2678 on muscle atrophy, fat accumulation and overall recovery are particularly impactful since atrophy was attenuated during immobilization, and muscle loss in this setting is hard to mitigate,” said Stuart M. Phillips, Ph.D., Principal Investigator of the study.

In this 28-day Non-IND, IRB-approved double-blind, randomized, placebo-controlled pilot clinical study, AXA2678 was assessed for safety, tolerability and its effects on muscle structure and function in healthy subjects with immobilization-induced acute muscle atrophy. Primary assessments from the study showed that AXA2678 was well tolerated with no significant safety issues observed during the study period. Secondary assessments demonstrated that AXA2678:

• Attenuated muscle disuse atrophy during immobilization;
• Mitigated intramuscular fat accumulation commonly seen during disuse atrophic conditions;
• Tended to improve both peak force and time to attain peak force in the immobilized limb compared to placebo.

Additional details from the study are available in the Frontiers publication titled, “A novel amino acid composition ameliorates short-term muscle disuse atrophy in healthy young men”.

“This is the first published data from our AXA muscle program, and I’m pleased that the results support the design hypothesis of EMMs to positively impact both muscle structure and function during acute immobilization,” said Manu Chakravarthy, M.D., Ph.D., Senior Vice President, Clinical Development and Chief Medical Officer of Axcella. “These data support the continued investigation of AXA2678 for disuse-related muscle atrophy and possibly other metabolic muscle diseases.”

“We are encouraged by these important results”, said Bill Hinshaw, President and Chief Executive Officer of Axcella. “Positive safety and tolerability data in this Non-IND, IRB-approved clinical study expands the body of data that demonstrate the positive impact that an AXA can have on important biologies and strengthens the support for EMMs as a novel approach with significant potential.”

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.

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 AXA2678, 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.