Axcella Therapeutics Launches Clinical Program to Develop Treatment for Patients with Long COVID
- Potential to play leading role in addressing Long COVID fatigue and muscle weakness via AXA1125’s mitochondrial and bioenergetic impact
- Phase 2a clinical trial to be initiated by researchers at
Oxford Universityby year-end; top-line data readout anticipated by mid-2022
- Program to be discussed at Axcella’s R&D Day today at
10 a.m. EDT
Led by researchers at the
More than 240 million cases of COVID-19 have been reported worldwide to date1, and it is estimated that nearly a quarter of these people suffer from the virus’s long-term effects2. These patients continue to experience a wide range of symptoms months after their initial diagnosis. Similar to many other conditions and diseases, mitochondrial dysfunction is increasingly being implicated as a key driver of Long COVID-induced fatigue, which is the most common symptom associated with the condition3,4.
“Long COVID is having a truly devastating impact on countless people around the world, leaving many with a sense of hopelessness. It is widely recognized that mitochondrial dysfunction may contribute to the profound fatigue associated with this condition,” said lead researcher Dr.
The Phase 2a trial will be a randomized, double-blind, placebo-controlled investigation to evaluate the efficacy and safety of AXA1125 in patients with exertional fatigue related to Long COVID. Approximately 40 patients 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.
“While Long COVID’s enormous patient and socioeconomic burden has become readily apparent, its underlying pathophysiology is now emerging,” said Dr.
The trial’s primary endpoint will assess the improvement of mitochondrial function within the skeletal muscle from baseline to Day 28 as measured by changes in phosphocreatine (PCr) recovery time via 31-phosphorus magnetic resonance spectroscopy (MRS). PCr recovery time is a well-established and highly sensitive measure that has been strongly correlated with a registrational endpoint (i.e., 6-minute walk test) in a number of other diseases in which fatigue and muscle weakness play a central role, including amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy, and chronic kidney disease. Key secondary endpoints include lactate levels, a 6-minute walk test, fatigue scores, and safety and tolerability.
AXA1125 is also currently being investigated in patients with non-alcoholic steatohepatitis (
Axcella R&D Day Today
Axcella’s platform and each of its clinical programs will be discussed at the company’s R&D Day, which takes place today,
Betty Raman, Radcliffe Department of Medicine, University of Oxford, United Kingdom(Topic: Long COVID)
Stephen Harrison, Medical Director of Pinnacle Clinical Research, San Antonio, TXand Visiting Professor of Hepatology, University of Oxford, United Kingdom(Topic: NASH)
Eric Lawitz, VP, Scientific and Research Development, The Texas Liver Instituteand Clinical Professor of Medicine, University of Texas Health San Antonio(Topic: OHE)
Elliot Tapper, Director, Cirrhosis Program and Associate Professor of Medicine, University of Michigan(Topic: OHE)
A webcast of this event can be accessed by visiting the “Investors & News” section of the company’s website, www.axcellatx.com. A webcast replay will be available for 90 days following the presentation.
Internet Posting of Information
Axcella uses its website, www.axcellatx.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 this portion of the company’s website, in addition to following its press releases,
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 reset multiple biological pathways, improve cellular energetics, and restore homeostasis. 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 (
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 and AXA1665, the potential for these product candidates to address patients’ unmet needs, and the timing of the company’s clinical trial initiations and 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 of COVID-19 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
1 New York Times. https://www.nytimes.com/interactive/2021/world/covid-cases.html
2 A Detailed Study of Patients with Long-Haul COVID, A FAIR Health White Paper,
3 Carfì A, et al. Persistent Symptoms in Patients After Acute COVID-19. JAMA. 2020
4 Bindu D, et al. Redox imbalance links COVID-19 and myalgic encephalomyelitis/chronic fatigue syndrome. PNAS.
Source: Axcella Therapeutics