- AXA1665 Phase 2 trial initiation planned for Q2
- AXA1125 Phase 2b trial initiation planned for Q2
- Company decides not to expand enrollment in AXA4010-001
following cohort 1 readout
- Pipeline expansion opportunities to be announced in 2021
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 clearance of the company’s first investigational
new drug (IND) filing and provided an update on its product
candidates, research and development activities and expected
milestones for 2021.
“The year 2020 was a time of tremendous accomplishment for
Axcella as we reported positive data for our lead candidates,
AXA1665 and AXA1125, and prepared for their later stage clinical
trials,” said Bill Hinshaw, President and Chief Executive Officer
of Axcella. “We are excited to begin 2021 by announcing the
clearance of our first investigational new drug (IND) filing, which
enables us to advance AXA1665 into Phase 2 with a wealth of
clinical data just four years after this candidate was designed.
This validates our accelerated, highly informed development
approach, which enables us to de-risk candidates and maximize our
resources. As we enter what we expect to be a momentous 2021, our
attention is firmly focused on clinical trial execution for our
lead candidates and the expansion of our EMM composition
pipeline.”
AXA1665 Program Update
Axcella announced today that its IND application has been
cleared by the U.S. Food and Drug Administration (FDA) for AXA1665,
the company’s oral product candidate for the reduction in risk of
recurrent overt hepatic encephalopathy (OHE). In the second quarter
of 2021, Axcella plans to initiate a Phase 2 trial of AXA1665.
The Phase 2 trial will be a randomized, double-blind,
placebo-controlled, multi-center study evaluating the efficacy and
safety of AXA1665 in patients who have experienced at least one
prior OHE event and have neurocognitive dysfunction at screening.
Approximately 150 patients on either lactulose ± rifaximin
(stratified by rifaximin use) will be enrolled and randomized 1:1
to receive either 53.8 grams per day of AXA1665 or a
calorie-matched placebo in three divided doses for 24 weeks, with a
two-week safety follow-up period.
The trial will be conducted globally with the primary endpoint
assessing change in the psychometric hepatic encephalopathy score
(PHES) after 24 weeks of treatment. Secondary endpoints will
include the proportion of patients experiencing an OHE breakthrough
event; time to first OHE breakthrough event, including time to
hospitalization; changes in physical function; and patient-reported
outcomes. Other endpoints include measures of circulating ammonia,
amino acids, and inflammation-related markers.
“Over the course of the past few years, we have advanced AXA1665
from initial concept and design through two clinical studies
enrolling more than 70 subjects with liver disease, demonstrating
its multifactorial therapeutic potential,” said Manu Chakravarthy,
M.D., Ph.D., Chief Medical Officer of Axcella. “We are excited to
now initiate a global Phase 2 clinical trial assessing AXA1665’s
efficacy in patients with OHE. Ultimately, our goal is to bring a
new multi-targeted oral treatment option that improves upon today’s
standard of care for OHE patients and more comprehensively
addresses their unmet needs.”
In 2020, Axcella reported positive top-line data from
AXA1665-002. In this clinical study, AXA1665 was observed to be
well tolerated with a strong safety profile. Dose-dependent changes
were noted across measures of amino acid metabolism and
neurocognition over 12 weeks in subjects with mild and moderate
hepatic insufficiency. These included statistically-significant (p
<0.05) improvements in the Fischer Ratio and the psychometric
hepatic encephalopathy score (PHES) in the AXA1665 high dose arm
vs. placebo. Additionally, clinically-relevant trends were seen in
certain measures of nitrogen/ammonia handling and physical function
in the AXA1665 arms versus placebo.
AXA1125 Program Update
Axcella completed a successful Type B pre-IND meeting with the
FDA in late 2020 and is now working actively on its IND application
for AXA1125, the company’s oral product candidate for nonalcoholic
steatohepatitis (NASH). Subject to FDA clearance, Axcella plans to
proceed directly into a 48-week placebo-controlled Phase 2b paired
biopsy clinical trial enrolling adult patients with biopsy-proven
NASH, with the primary endpoint based on liver histology. This
trial is expected to be initiated in the second quarter of
2021.
In 2020, Axcella reported positive top-line data from
AXA1125-003. In this clinical study, AXA1125 was observed to be
well tolerated with a strong safety profile in subjects with
presumed NASH. Additionally, sustained reductions versus placebo
over 16 weeks were noted in virtually all key biomarkers of
metabolism, inflammation and fibrosis, including MRI-PDFF, HOMA-IR,
ALT and ProC3. Among subjects with type 2 diabetes enrolled in the
study, reductions in most biomarkers were even greater versus
placebo. Based on AXA1125’s multi-targeted design and these data,
Axcella believes this candidate holds the potential to serve as a
first-line NASH monotherapy for both adult and pediatric patients
and may be used in combination with other agents if required.
AXA4010 Program Update
AXA4010 has been under investigation in an open label pilot
clinical study, AXA4010-001. An initial cohort of nine subjects
with sickle cell disease (SCD) was enrolled and received AXA4010 in
two 26-gram doses per day for up to 12 weeks. This first cohort was
intended to allow the company to a) efficiently assess this
candidate’s safety, tolerability and effects on blood structure and
function, including red blood cell metabolism/hemolysis,
inflammation and vascular function/adhesion, and b) decide whether
to expand enrollment to additional cohorts.
Clinical data from this first cohort were recently received.
Most of the reported adverse events, including all serious adverse
events, were considered to be associated with underlying disease.
Five subjects reported mild to moderate gastrointestinal events
(diarrhea and nausea) that were considered to be possibly related
to AXA4010. Biomarker data from this cohort suggest activity in
targeted biologies, including generally positive trends in markers
related to inflammation. Other markers related to hemolysis and
vascular adhesion demonstrated greater variability. Based on the
totality of the findings to date, Axcella has decided not to expand
enrollment in the AXA4010-001 study.
R&D Update
Axcella sees the potential to develop a range of EMM
compositions for a variety of diseases and conditions, including
those related to metabolism, muscle atrophy, mitochondrial biology,
neuroprotection, inflammation and immunology. The company has
characterized approximately 75 potential therapeutic applications
for EMMs and is now applying platform advances and program
learnings to assess pipeline expansion opportunities.
Axcella plans to provide an update on its research and
development activities and share details about opportunities it has
identified for pipeline expansion later in 2021. As demonstrated
with AXA1125 and AXA1665, the company sees the opportunity for
rapid, efficient and highly informed clinical development for its
future product candidates.
About Endogenous Metabolic Modulators
(EMMs)
EMMs are a broad family of 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 root 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 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 trials 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 AXA1665, AXA1125 and potential future EMM
compositions, the potential for AXA1665 to reduce OHE events and
improve the quality of life for cirrhotic patients, the potential
for AXA1125 to serve as a first-line NASH monotherapy for both
adult and pediatric patients and be used in combination with other
agents if required, the design, status and timing of the company’s
planned Phase 2 clinical trial of AXA1665 and planned Phased 2b
clinical trial of AXA1125, the timing and outcome of IND
application submissions, the intended results of the company’s
strategy and approach and the company’s ability to address other
complex diseases and conditions 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 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 trials of AXA1665 and 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 initiation plans and timing, clinical trial design
and target indications for AXA1665 and AXA1125, the clinical
development and safety profile of AXA1665 and AXA1125 and their
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/20210111005285/en/
Company Contact Jason Fredette
jfredette@axcellahealth.com (857) 320-2236
Axcella Health (NASDAQ:AXLA)
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