Axcella Announces FDA IND Clearance Supporting Regulatory Path to Registration of AXA1125 for Long COVID Fatigue

Axcella Therapeutics, a clinical-stage biotechnology company pioneering novel approaches to treating complex diseases using multi-targeted endogenous metabolic modulator (EMM) compositions, has announced its Investigational New Drug (IND) application to initiate a phase 2b/3 trial in the U.S. for AXA1125 in the treatment of Long COVID Fatigue has been cleared by the U.S. Food and Drug Administration (FDA).

The company reported that it had received regulatory guidance from the FDA, supporting a trial that is designed to serve as the registration trial for patients with Long COVID Fatigue. The study design now has acceptance from both the U.S. and U.K. regulatory authorities. Last month, the company announced a regulatory path to registration of AXA1125 in the treatment of Long COVID Fatigue, and 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.

The guidance from the FDA and the MHRA follows the company’s 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. The study found that 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. Consistent with the Ph2a trial, the Ph2b/3 trial will enroll participants who have had fatigue for at least 12 weeks after COVID-19 infection. The primary endpoint will utilize the same patient reported outcome tool, the Chalder Fatigue Questionnaire (CFQ-11), to measure improvements in fatigue. Additional endpoints will evaluate improvements in physical function, quality of life, and ability to return to work. Participants will receive either placebo or AXA1125 for three months.

“We’re excited to now have the regulatory clearance from both the U.S. and U.K. authorities to advance our Long COVID program into a trial that can lead to registration,” said Bill Hinshaw, CEO of Axcella. “The guidance from the FDA demonstrates continued progress, following the MHRA milestone a few weeks ago, providing clear next steps in this important, ongoing, and as-yet untreated disease. There are millions of people around the world suffering from Long COVID Fatigue who are without options. Axcella has the leading program in the field, and this regulatory feedback provides the next step in developing a potential Long COVID Fatigue treatment.”

“We are pleased to obtain FDA clearance for this phase 2b/3 study, following our positive feedback from the MHRA,” said Margaret Koziel, M.D., Chief Medical Officer of Axcella. “Both on an individual and a societal level, the impact of Long COVID on the ability to have a full and productive life is significant, and we hope to offer a solution to these patients.”