Axcella Therapeutics Announces Initiation of AXA1125 Long COVID Phase 2a Clinical Trial

• Patient screening underway in the United Kingdom in trial to assess oral product candidate’s potential to address Long COVID fatigue and muscle weakness
• Top-line data readout anticipated by mid-2022

Axcella Therapeutics (Nasdaq: AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using multi-targeted endogenous metabolic modulator (EMM) compositions, and researchers at the Oxford University Radcliffe Department of Medicine today announced that patient screening is now underway in a Phase 2a trial that is investigating AXA1125, an oral product candidate, as a potential treatment for Long COVID, also known as Post COVID-19 Condition and post-acute sequelae of SARS-COV-2 infection (PASC).

“There already has been a tremendous amount of interest in participating in this trial among patients suffering from Long Covid fatigue,” said lead researcher Dr. Betty Raman, British Heart Foundation Oxford Centre of Research Excellence Clinical Transition Intermediate Fellow. “This is not surprising given the sheer number of patients impacted by this syndrome, the life-changing symptoms that they are experiencing and the complete absence of treatment options today. We are keen to evaluate AXA1125’s impact on the mitochondria and muscle function and its potential to improve functional outcomes for patients who are in desperate need.”

The Phase 2a is a randomized, double-blind, placebo-controlled trial that is evaluating the efficacy and safety of AXA1125 in patients with exertional fatigue related to Long COVID. Approximately 40 patients in the United Kingdom will be enrolled and randomized evenly to receive either 67.8 grams per day of AXA1125 or a matched placebo in two divided doses for 28 days, with a one-week safety follow-up period.

“There is mounting evidence suggesting that infection with the COVID-19 virus can impair the mitochondria’s ability to act as the ‘powerhouse of the cell,’ manifesting in Long COVID fatigue and muscle weakness,” said Dr. Alison Schecter, President of R&D at Axcella. “Our preclinical and clinical findings to date demonstrate that AXA1125 holds the potential to improve multiple aspects of mitochondrial biology, including increasing energetics and reducing inflammation. This provides us with hope that we can make a real difference for tens of millions of patients suffering from this debilitating condition.”

The primary endpoint in this Phase 2a trial is the change from baseline to Day 28 in phosphocreatine (PCr) recovery time as measured by 31-phosphorus magnetic resonance spectroscopy (pMRS). In past clinical trials, this highly sensitive and well-established measure of mitochondrial function has been correlated with the six-minute walk test, which has been a registrational endpoint in other conditions involving fatigue, such primary pulmonary hypertension and exertional angina. Key secondary endpoints in the Phase 2a trial include lactate levels (a serum marker of mitochondrial dysfunction), six-minute walk test, fatigue scores, and safety and tolerability.

Top-line data are expected to be reported from the Phase 2a trial in mid-2022.

More than 270 million cases of COVID-19 have been reported worldwide to date, and a recent study estimates that nearly a quarter of Americans who contract COVID-19 experience Long COVID symptoms. Research indicates that fatigue is the most common of these symptoms, impacting a majority of Long COVID patients.

About AXA1125

AXA1125 is a proprietary multi-targeted EMM composition consisting of five amino acids and an amino acid derivative that works via multiple biological pathways. Preclinical and clinical data have shown this oral product candidate’s potential to increase fatty acid oxidation, ATP production, ketogenesis and mitochondrial bioenergetics, contributing to meaningful reductions in key markets of liver fat, insulin resistance, inflammation, and fibrosis. AXA1125 is currently being investigated in the EMMPACT Phase 2b clinical trial in nonalcoholic steatohepatitis (NASH) (NCT04880187) and a Phase 2a clinical trial in Long COVID (NCT05152849).

About the University of Oxford and The Radcliffe Department of Medicine

Oxford University has been placed number 1 in the Times Higher Education (THE) World University Rankings for the sixth year running, and at the heart of this success is the University’s ground-breaking research and innovation. Oxford is world-famous for research excellence and home to some of the most talented people from across the globe. The University’s work helps the lives of millions, solving real-world problems through a huge network of partnerships and collaborations. The breadth and interdisciplinary nature of our research sparks imaginative and inventive insights and solutions. Through its research commercialization arm, Oxford University Innovation, Oxford is the highest university patent filer in the UK and is ranked first in the UK for university spinouts, having created more than 200 new companies since 1988. Over a third of these companies have been created in the past three years.

The Radcliffe Department of Medicine (RDM) is one of the two main departments of medicine at the University of Oxford and aims to tackle some of the world’s biggest health challenges by integrating innovative basic biology with cutting edge clinical research. The RDM has internationally renowned programs in a range of areas including cardiovascular sciences, diabetes and endocrinology, immunology, hematology and pathology. https://www.rdm.ox.ac.uk/.

About Axcella Therapeutics (Nasdaq: AXLA)

Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using endogenous metabolic modulator (EMM) compositions. The company’s product candidates are comprised of EMMs and derivatives that are engineered in distinct combinations and ratios to restore cellular homeostasis in multiple key biological pathways and improve cellular energetic efficiency. Axcella’s pipeline includes lead therapeutic candidates in Phase 2 development for the reduction in risk of overt hepatic encephalopathy (OHE) recurrence, the treatment of Long COVID, and the treatment of non-alcoholic steatohepatitis (NASH). The company’s unique model allows for the evaluation of its EMM compositions through non-IND clinical studies or IND clinical trials. For more information, please visit www.axcellatx.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 characteristics, competitive position and development potential of AXA1125, the potential for it to address patients’ unmet needs, and the timing of the company’s clinical trial readouts. 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 potential impact of COVID-19 on the company’s ability to conduct and complete its ongoing or planned clinical studies and clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its clinical trials of AXA1665 and AXA1125, other potential impacts on the company’s business and financial results, including with respect to its ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company’s clinical trial plans and timing, clinical trial design and target indications for AXA1665 and AXA1125, the clinical development and safety profile of AXA1665 and AXA1125 and their therapeutic potential, whether and when, if at all, the company’s product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company’s target indications, and other risks identified in the company’s SEC filings, including Axcella’s Annual Report on Form 10-K, 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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