CAMBRIDGE, Mass.– 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.
