Axcella Health (AXLA) Announces Launch of Clinical Program to Develop Treatment for Long COVID

October 26, 2021 6:58 AM EDT

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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, today announced a new clinical program to investigate AXA1125 as a potential treatment for patients with Long COVID, a complex condition also known as Post COVID-19 Condition and Post-Acute Sequelae of COVID-19 (PASC). The United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) accepted a Phase 2a clinical trial authorization (CTA) submission from Axcella on October 22, 2021.

Led by researchers at the Radcliffe Department of Medicine at the University of Oxford (U.K.), the clinical trial is planned to begin by the end of this year at the Oxford Centre for Clinical Magnetic Resonance Research, with top-line data expected by mid-2022. This new program is among the topics to be discussed at Axcella’s R&D Day, which is taking place today beginning at 10 a.m. EDT.

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. Betty Raman, British Heart Foundation Oxford Centre of Research Excellence Clinical Transition Intermediate Fellow from Oxford University’s Radcliffe Department of Medicine. “With no approved Long COVID therapies, the need for continued innovation is urgent. I am pleased to be leading an investigation of AXA1125 to understand its potential to restore cellular energetics and address patients’ needs.”

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. Alison Schecter, President of R&D at Axcella. “In two prior successful clinical studies and in preclinical models, AXA1125 has demonstrated an ability to restore mitochondrial function and improve energetic efficiency via increased fatty acid oxidation, restored cellular homeostasis, and reduced inflammation. This provides us with confidence about its potential to help the growing number of patients who are suffering from COVID’s debilitating effects long after contracting the virus.”

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 (NASH) in the EMMPACT Phase 2b clinical trial (NCT04880187). This global, placebo-controlled trial was initiated in the second quarter of 2021 and is enrolling approximately 270 subjects with biopsy-proven NASH for a 48-week treatment period. A second product candidate, AXA1665, is under investigation in patients with overt hepatic encephalopathy (OHE) in the EMMPOWER Phase 2 clinical trial (NCT04816916). This global, placebo-controlled trial, also initiated in the second quarter of 2021, is enrolling approximately 150 subjects with a history of OHE for a 24-week treatment period.

Bill Hinshaw, Axcella President and CEO, noted, “Today’s announcement further demonstrates the power of our model and is the result of a confluence of events, including the ongoing global pandemic, new findings about Long COVID’s manifestation and drivers, and Axcella’s recent scientific, clinical, and regulatory successes with AXA1125. With the impending initiation of this Phase 2a trial and with EMMPOWER and EMMPACT already underway, we are positioned for multiple potentially transformational data readouts ahead.”

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