IND for phase 2b/3 trial submitted to the FDA

Axcella Therapeutics (Nasdaq: AXLA), a clinical-stage biotechnology company pioneering novel approaches to treating complex diseases using multi-targeted endogenous metabolic modulator (EMM) compositions, announced a regulatory path to registration of AXA1125 in the treatment of Long COVID Fatigue. The company reported that it had received regulatory guidance from The Medicines and Healthcare products Regulatory Agency (MHRA), the U.K.’s regulatory agency, supporting a single trial that could serve as the registration trial for patients with Long COVID fatigue, and aligning on key measurements, including primary endpoint and trial design. Axcella will be meeting with the MHRA in the near term to discuss the Innovative Licensing and Access Pathway (ILAP) application. The company further reported submission of an Investigational New Drug (IND) Application to the U.S. Food and Drug Administration (FDA) for a Phase 2b/3 trial.

“I urge the US government to use all the available mechanisms, including the RECOVER study infrastructure, to advance a clinical study of Axcella’s investigational drug in the U.S. as rapidly as possible. Patients are waiting.”

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These efforts follow submission of materials to both regulatory agencies including results from the Phase 2a randomized, double-blind, placebo-controlled investigation to evaluate the efficacy and safety of AXA1125 in patients with fatigue related to Long COVID. Notably, in this study subjects who received AXA1125 experienced clinically and statistically significant improvement in mental (p=0.0097) and physical (p=0.0097) fatigue scores compared to placebo subjects.

Long COVID is a persistent and growing long-term part of the pandemic, affecting an estimated one hundred million patients worldwide, with fatigue as the most commonly reported symptom. Recent estimates indicate that 15-20% of Americans with COVID have persisting health issues,ⁱ up to four million Americans are out of work due to Long COVID symptoms, and that Long COVID has contributed to approximately $1 trillion in lost earnings and $544 billion in increased medical spending.ⁱⁱ

“At Axcella, we are gratified and validated that there is a clear path to advance the Long COVID program into a potential registration trial with a leading regulator,” said Bill Hinshaw, CEO of Axcella. “This treatment could help millions of people around the world and there are no other agents that have demonstrated impact on the level of fatigue in a controlled trial. Many stakeholders have been eagerly anticipating a regulatory path in this new, important, and widespread disease and it is exciting to have this milestone achieved. With additional resources or collaboration, this program has the potential to advance and quickly reach patients in need.”

“We were pleased to have such constructive engagement with the MHRA, who have consistently recognized the urgent needs of patients and the healthcare system in the U.K. and have taken a rigorous and engaged, forward-looking approach to addressing Long COVID,” said Margaret Koziel, M.D., Chief Medical Officer of Axcella. “The tenor of the MHRA response and the results of our trial informed our recently completed FDA IND submission for our phase 2b/3 Long COVID trial. AXA1125 is the most advanced clinical-stage program for this devastating disease and we look forward to the opportunity to conduct a global trial that has the potential to rapidly enroll and submit for approval as the leading program in the field.”

“The statistically significant improvement in reported mental and physical fatigue among study participants receiving AXA1125 is a very encouraging finding for Long COVID patients, who often experience extreme and constant fatigue throughout their days,” added Betty Raman, M.D., Associate Professor of Cardiovascular Medicine at the Radcliffe Department of Medicine, University of Oxford, who led the phase 2a study.

“Given the devastating health and economic impact that Long COVID is having on millions of patients and their families, there is an urgent need for new treatments developed specifically for this population,” said Oved Amitay, Chief Executive Officer of Solve M.E., a non-profit organization that serves as a catalyst for critical research into diagnostics, treatments, and cures for myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), Long COVID and other post-infection diseases. “We are encouraged by data from Axcella’s phase 2 study in Long COVID chronic fatigue patients and the hope that AXA1125 provides to these patients, and by the opportunity to advance the program to a pivotal clinical trial.” Amitay, who also is the co-founder of the Long COVID Alliance, a network of patient-advocates, scientists, disease experts, and drug developers focused on educating policy makers and accelerating research into post-viral illnesses, added, “I urge the US government to use all the available mechanisms, including the RECOVER study infrastructure, to advance a clinical study of Axcella’s investigational drug in the U.S. as rapidly as possible. Patients are waiting.”