- Multi-targeted oral candidate with the potential to be a
first-line treatment option for adult and pediatric patients with
NASH
- Phase 2b clinical trial in adults expected to begin in Q2
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 achieved a key milestone with U.S. Food
and Drug Administration (FDA) clearance of an Investigational New
Drug (IND) application for AXA1125, enabling the company to proceed
directly into a Phase 2b clinical trial. AXA1125 is Axcella’s
multi-targeted oral product candidate for nonalcoholic
steatohepatitis (NASH), a chronic and progressive liver disease
impacting up to 40 million people in the U.S. alone.
“This IND follows close on the heels of AXA1665’s IND clearance
earlier this year, ushering in an exciting new era for Axcella as
we seek to tackle a variety of complex diseases and address
important unmet needs for patients utilizing multi-targeted EMM
compositions,” said Bill Hinshaw, President and Chief Executive
Officer of Axcella. “I am pleased by our team’s strong execution in
completing two robust FDA submissions and readying us for the start
of our highly efficient, later-stage clinical trials.”
Axcella expects to initiate its Phase 2b clinical trial in the
second quarter of 2021. This randomized, double-blind,
placebo-controlled, multi-center trial will evaluate the efficacy,
safety and tolerability of AXA1125 in adult 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 trial 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.
“AXA1125 leverages a mechanism that engages multiple
pathophysiologic pathways involved in the development of NASH using
a modality with well-precedented safety, which is particularly
important given the many comorbidities associated with this chronic
disease,” said Alison Schecter, M.D., President of R&D at
Axcella. “In past clinical studies, compelling activity has been
seen with AXA1125 across a range of non-invasive biomarkers, with
enhanced effects in type 2 diabetics. Our goal in the Phase 2b is
to affirm AXA1125’s impact via histology and further strengthen its
profile as a compelling candidate for first-line treatment in
NASH.”
The upcoming Phase 2b clinical trial follows two earlier non-IND
clinical studies in which reductions were seen with AXA1125 in key
measures of hepatic fat, insulin resistance, inflammation and
fibrosis with a safe and well tolerated profile. Presentations
containing those findings can be found by visiting
https://axcellahealth.com/publications/.
About AXA1125 and Nonalcoholic
Steatohepatitis (NASH)
NASH is the most severe form of non-alcoholic fatty liver
disease (NAFLD). This chronic, complex disease is associated with
significant morbidity and mortality globally, and it is estimated
to impact up to 40 million Americans, including up to 10% of
American children. Despite its severity and increasing prevalence,
there are currently no approved therapies for NASH in the United
States.
AXA1125, Axcella’s NASH product candidate, is a composition of
six amino acids and derivatives that is designed to target multiple
metabolic pathways involved in NASH’s progression from metabolism
to inflammation to fibrosis. In prior clinical studies, this oral
product candidate has been safe and well tolerated and has
demonstrated clinically meaningful reductions in a range of
well-accepted, non-invasive NASH biomarkers, with the most
pronounced activity noted in subjects with type 2 diabetes. AXA1125
is now entering Phase 2b development in adults with NASH. Axcella
also plans to investigate AXA1125 in pediatric NASH.
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 company’s clinical studies to date are or 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 were not designed or intended to
evaluate a product candidate’s ability to diagnose, cure, mitigate,
treat or prevent a disease. INDs were recently cleared for
Axcella’s lead product candidates, AXA1665 and AXA1125, allowing
for the investigation of these candidates’ efficacy, safety and
tolerability in subsequent clinical trials.
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 design, status and timing of
the company’s planned Phase 2b clinical trial of AXA1125, 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 our 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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version on businesswire.com: https://www.businesswire.com/news/home/20210413005207/en/
Jason Fredette jfredette@axcellahealth.com (857) 320-2236
Axcella Health (NASDAQ:AXLA)
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