- Initial clinical sites activated and patient screening
underway
- Potential for AXA1125 to serve as a first-line therapy for
patients with nonalcoholic steatohepatitis (NASH)
Axcella (Nasdaq: AXLA), a clinical-stage biotechnology company
pioneering a new approach to treat complex diseases and improve
health using endogenous metabolic modulator (EMM) compositions,
today announced that it has activated initial clinical sites and
begun patient screening for its EMMPACT Phase 2b clinical trial of
AXA1125, the company’s multi-targeted oral product candidate for
the treatment of NASH.
“NASH is the quintessential complex disease, involving the
dysregulation of numerous biological pathways and impacting an
enormous, heterogenous global population,” said Stephen A.
Harrison, M.D., Medical Director of Pinnacle Clinical Research in
San Antonio, TX, visiting professor of Hepatology at the University
of Oxford, UK and the principal investigator of EMMPACT. “These
factors have confounded many physicians and drug developers in the
past. Given its multi-modal mechanism, the activity and
tolerability seen in past clinical studies and the presumed safety
of its underlying amino acids, AXA1125 holds the potential to serve
as an ideal first-line NASH agent. We are excited to have EMMPACT
underway and are eager to gauge AXA1125’s histological impact.”
Axcella has branded this global trial EMMPACT based on the
potential for AXA1125, an EMM composition, to deliver meaningful,
multifactorial clinical benefits to patients with NASH. This
randomized, double-blind, placebo-controlled, multi-center
investigation will evaluate the efficacy and safety of AXA1125 in
patients with biopsy-confirmed F2/F3 NASH. Approximately 270
patients will be enrolled and randomized 1:1:1 to receive either
45.2 or 67.8 grams per day of AXA1125 or a matched placebo in two
divided doses for 48 weeks, with a four-week safety follow-up
period. Patients will be stratified based on the presence or
absence of type 2 diabetes.
“The initiation of EMMPACT is the latest in a series of exciting
recent milestones for Axcella and comes just one month after
clearing our IND application for AXA1125, which is a testament to
our team’s strong preparation and execution as well as our
investigators’ interest and engagement,” said Bill Hinshaw,
President and Chief Executive Officer of Axcella. “I am confident
in our ability to extend our successful track record and rapidly
enroll this clinical trial.”
EMMPACT will be conducted globally across more than 70 clinical
sites with a primary endpoint assessing the proportion of patients
with a biopsy-confirmed ≥2 point improvement in NAFLD Activity
Score (NAS) after the 48-week treatment period. Secondary endpoints
will include the proportion of patients achieving biopsy-confirmed
resolution of NASH without worsening of fibrosis and the proportion
of patients achieving a ≥1 stage improvement in fibrosis without
worsening of NASH. A range of non-invasive biomarkers, including
MRI-PDFF and Fibroscan, will be utilized for additional endpoints
and an interim analysis in the trial.
“In two consecutive clinical studies of AXA1125 in subjects with
presumed NASH, meaningful improvements were seen in non-invasive
measures of hepatic fat, insulin resistance, inflammation and
fibrosis,” said Alison Schecter, M.D., President of R&D at
Axcella. “We expect EMMPACT to provide robust insights on AXA1125’s
dose response, longer-term effects, histological impact and
potential differentiation in type 2 diabetics that can be leveraged
to expand our development efforts.”
Additional trial information can be found on
https://clinicaltrials.gov/ via the identifier NCT04880187.
About AXA1125 and Nonalcoholic
Steatohepatitis (NASH)
NASH is the most severe form of fatty liver disease and is
driven by multifactorial systemic dysregulation of pathways
associated with metabolism, inflammation and fibrosis. If left
untreated, this disease may ultimately lead to life-threatening
conditions such as cirrhosis or liver cancer, requiring liver
transplant. According to the Global Liver Institute’s U.S. NASH
Action Plan published in December 2020, up to 40 million people in
the U.S. alone are living with NASH and approximately 10% of U.S.
children are afflicted with this disease. Incidence is expected to
continue increasing in parallel with the obesity and type 2
diabetes epidemics. Currently, there are no approved drug therapies
for NASH in the United States.
AXA1125, Axcella’s product candidate for treatment of NASH, is a
composition of six amino acids and derivatives that is designed to
target multiple metabolic pathways known to affect the pathogenesis
of fatty liver disease. In prior clinical studies, this oral
product candidate has been safe, well tolerated and has
demonstrated the potential to reduce liver fat, inflammation and
fibrosis with a safe and well tolerated profile while avoiding an
impact on lipids and weight. AXA1125 is now in Phase 2b
development.
About Endogenous Metabolic Modulators
(EMMs)
EMMs are a broad family of naturally occurring molecules,
including amino acids, that regulate human metabolism. Axcella is
developing a range of novel product candidates that are comprised
of multiple EMMs engineered in distinct combinations and ratios to
simultaneously impact multiple metabolic pathways to modify the
underlying causes of various complex diseases and improve
health.
About Axcella’s Clinical
Studies
Each of the clinical investigations that the company has
completed to date have been conducted as non-investigational new
drug application (IND) clinical studies under U.S. Food and Drug
Administration regulations and guidance supporting research with
food. These studies evaluate product candidates for safety,
tolerability and effects on the normal structures and functions in
humans, including in individuals with disease. They are not
designed or intended to evaluate a product candidate’s ability to
diagnose, cure, mitigate, treat or prevent a disease. If Axcella
decides to further develop a product candidate as a potential
therapeutic, as is the case with AXA1665 and AXA1125, any
subsequent clinical studies will be conducted under an IND.
Internet Posting of
Information
Axcella uses its website, www.axcellahealth.com, as a means of
disclosing material nonpublic information and for complying with
its disclosure obligations under Regulation FD. Such disclosures
will be included on the company’s website in the “Investors and
News” section. Accordingly, investors should monitor this portion
of the company’s website, in addition to following its press
releases, SEC filings and public conference calls and webcasts.
About Axcella
Axcella is a clinical-stage biotechnology company pioneering a
new approach to treat complex diseases and improve health using
endogenous metabolic modulator (EMM) compositions. The company’s
product candidates are comprised of EMMs and their derivatives that
are engineered in distinct combinations and ratios to
simultaneously impact multiple biological pathways. Axcella’s
pipeline includes lead therapeutic candidates for non-alcoholic
steatohepatitis (NASH) and the reduction in risk of overt hepatic
encephalopathy (OHE) recurrence. For more information, please visit
www.axcellahealth.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 characteristics, competitive position, and
development potential of AXA1125, the potential for AXA1125 to
serve as a first-line NASH agent, the company’s ability to rapidly
enroll its clinical trials, and the company’s ability to address
other complex diseases utilizing EMM compositions. 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
IND-enabled 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
planned clinical trial of AXA1125, other potential impacts of
COVID-19 on the company’s business and financial results, including
with respect to the company’s 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 initiation 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.
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Jason Fredette jfredette@axcellahealth.com (857) 320-2236
Axcella Health (NASDAQ:AXLA)
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