MHRA guidance aligns on key measurements for a
registration trial, including primary endpoint and trial design
elements
IND for phase 2b/3 trial submitted to the
FDA
Axcella to host a conference call Tuesday,
January 24 at 8:00 a.m. ET; To register, click here
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.
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.”
Investor Conference Call Information
Axcella will host a live conference call and webcast at 8:00
a.m. ET on Tuesday, January 24, 2023. To access the live conference
call, please dial 844-808-7139 (domestic) or 412-902-0127
(international) and refer to “Axcella Health.” A webcast of the
call will also be available under "Events and Presentations" in the
Investors section of the Axcella Health website at
https://ir.axcellatx.com/. The archived webcast will be available
on Axcella’s website approximately two hours after the conference
call and will be available for 90 days following the call.
About Axcella Therapeutics (Nasdaq: AXLA)
Axcella is a clinical-stage biotechnology company pioneering a
new approach to treat complex diseases using compositions of
endogenous metabolic modulators (EMMs). 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 for the treatment of Long COVID, NASH, and the reduction
in risk of OHE recurrence. The company’s unique model allows for
the evaluation of its EMM compositions through non-IND clinical
studies or IND clinical trials. For more information, please visit
www.axcellatx.com.
Forward-Looking Statements
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 potential utility of AXA1125 as a treatment of Long
COVID and the Company’s anticipated regulatory pathway for AXA1125
and the timing and potential success thereof. 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 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 AXA1125, the clinical development and safety
profile of AXA1125 and its 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 SEC
filings, including Axcella’s Annual Report on Form 10-K, Quarterly
Report on Form 10-Q and subsequent filings with the SEC. The
company cautions you not to place undue reliance on any
forward-looking statements, which speak only as of the date they
are made. Axcella disclaims any obligation to publicly update or
revise any such statements to reflect any change in expectations or
in events, conditions or circumstances on which any such statements
may be based, or that may affect the likelihood that actual results
will differ from those set forth in the forward-looking statements.
Any forward-looking statements contained in this press release
represent the company’s views only as of the date hereof and should
not be relied upon as representing its views as of any subsequent
date. The company explicitly disclaims any obligation to update any
forward-looking statements.
i Bull-Otterson L, Baca S, Saydah S, et al. Post–COVID
Conditions Among Adult COVID-19 Survivors Aged 18–64 and ≥65 Years
— United States, March 2020–November 2021. MMWR Morb Mortal Wkly
Rep 2022;71:713–717. DOI:
http://dx.doi.org/10.15585/mmwr.mm7121e1external icon ii Bach K.
New data shows long Covid is keeping as many as 4 million people
out of work. Brookings Institute. August 24, 2022.
https://www.brookings.edu/research/new-data-shows-long-covid-is-keeping-as-many-as-4-million-people-out-of-work/#footnote-3
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version on businesswire.com: https://www.businesswire.com/news/home/20230123005745/en/
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