- Clinically relevant reductions in liver fat content, insulin
resistance and fibroinflammation markers observed with AXA1125
along with favorable tolerability, supporting its potential to be a
first-line NASH therapy
- Greater activity in key markers seen among subjects with type 2
diabetes receiving AXA1125
- Company plans to engage with FDA regarding IND submission for
AXA1125, proposed Phase 2b clinical trial in adult NASH and
pediatric development program
- Conference call to be held today at 8:30 a.m. ET
Axcella (Nasdaq: AXLA), a clinical-stage biotechnology company
focused on leveraging endogenous metabolic modulators (EMMs) to
pioneer a new approach for treating complex diseases and improving
health, today announced positive top-line data from
AXA1125-003.
AXA1125-003 is a placebo-controlled, randomized, multi-arm
clinical study assessing the impact of AXA1125 and AXA1957 on
safety, tolerability and effects on structures and functions of the
liver, as measured by a comprehensive panel of imaging and soluble
biomarkers related to metabolism, inflammation and fibrosis. Both
of these distinct product candidates are proprietary compositions
of amino acids and derivatives that have been designed to support
liver health. In this non-IND study, 102 adult non-alcoholic fatty
liver disease (NAFLD) subjects with presumed nonalcoholic
steatohepatitis (NASH), based on inclusion criteria, were enrolled
and dosed in a 2:2:2:1 ratio to receive AXA1125, one of two AXA1957
doses, or placebo administered twice daily for 16 weeks. Study
subjects were stratified based on the presence or absence of type 2
diabetes.
Results from the study showed that AXA1125 and AXA1957 were
generally well-tolerated, with sustained reductions noted for both
product candidates versus placebo in key biomarkers of metabolism,
inflammation and fibrosis over 16 weeks. Overall, as compared to
placebo, AXA1125 demonstrated larger and more consistent reductions
in clinically relevant biomarkers than AXA1957. Among subjects
receiving AXA1125, 39% achieved a ≥30% relative reduction in liver
fat content (MRI-PDFF), 39% achieved a ≥17 U/L reduction in alanine
aminotransaminase (ALT), and 35% achieved a ≥80 mSec reduction in
corrected T1 (cT1). Among the 11 subjects with type 2 diabetes
receiving AXA1125, a greater proportion achieved each of these
thresholds. Emerging evidence suggests that these thresholds of
activity increase the likelihood of histopathological improvement
in NASH subjects. Notably, the above results were seen without
impacting mean body weight or serum lipids.
Manu Chakravarthy, M.D., Ph.D., Chief Medical Officer of
Axcella, said, “In AXA1125-003, we were seeking to evaluate safety
and tolerability while also determining what differential responses
may be seen from AXA1125 and AXA1957 across markers of metabolism,
inflammation and fibrosis. It is gratifying that multifactorial
activity and a favorable tolerability profile were noted for
AXA1125 in this multi-arm, placebo-controlled randomized study,
which replicates findings from our previous clinical study in
subjects with NAFLD and type 2 diabetes. We believe that these
data, coupled with AXA1125’s oral route of administration and
favorable tolerability profile to date, reinforce its potential to
meaningfully improve the lives of patients with NASH.”
“These data indicate that AXA1125 holds the potential to be a
first-line therapy in NASH, with impressive, concordant metabolism,
inflammation and fibrosis activity as well as a favorable
tolerability profile with no meaningful changes to serum lipids and
body weight,” 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 AXA1125-003. “Additionally, in the majority of
subjects with type 2 diabetes receiving AXA1125, clinically
relevant thresholds of activity were observed in non-invasive tests
that suggest a higher probability of positive outcomes
histopathologically. This is a potential differentiator and is
particularly encouraging given that nearly 40% of the NASH
population is diabetic and the disease is known to be more severe
in these patients.”
Select Measures of Relevance at 16 Weeks
Measure
Placebo
AXA1125
AXA1957 Low
AXA1957 High
Subjects dosed
15
29
26
32
Mean relative reduction in liver fat
content (MRI-PDFF)
-6%
-23%
-20%
-8%
Subjects with ≥30% relative reduction in
liver fat content
8%
39%
23%
19%
Mean relative reduction in ALT
-7%
-22%
-19%
-21%
Subjects with ≥17 U/L reduction in ALT
25%
39%
32%
37%
Subjects with ≥80 mSec reduction in
cT1
17%
35%
23%
23%
Mean absolute change in proC3 (ng/mL)
-0.7
-3.4
-3.1
-4.1
* Mean values and percentages above only include subjects for
whom data was available at the week 16 timepoint.
AXA1125 and AXA1957 were both generally well tolerated in the
study. The adverse events (AEs) experienced in ≥10% of subjects
were gastrointestinal (diarrhea, nausea, reduced appetite) and
upper respiratory infection. Gastrointestinal AEs were generally
mild and transient, self-resolving in two to three weeks on
average. Two serious adverse events were reported, both of which
were determined to be unrelated to study product
administration.
“The findings from this clinical study further validate the
strength of our EMM platform and its ability to identify product
candidates with the potential to address complex diseases in a
multi-targeted manner,” said Bill Hinshaw, President and Chief
Executive Officer of Axcella. “Given the strength and consistency
of data on AXA1125, we have selected it as our product candidate
for NASH and have decided that we will not reinitiate our
AXA1957-002 pediatric study, which had recently been suspended due
to COVID-19. In the months ahead, we plan to engage with the U.S.
Food and Drug Administration (FDA) to discuss our investigational
new drug (IND) application for AXA1125, proposed Phase 2b clinical
trial in adults and pediatric development program. We give our
thanks to the many subjects and investigators who participated in
AXA1125-003 and will continue working diligently toward our goal of
providing them with an effective and safe treatment option.”
Conference Call Information
Axcella will host a conference call today at 8:30 a.m. ET to
discuss the top-line data from AXA1125-003. The conference call
webcast and accompanying slides will be made available shortly
before the start of the call on the company’s website at
www.axcellahealth.com in the Investors & News. To access the
call via telephone, please dial (866) 652-5200 (U.S. toll free) or
(412) 317-6060 (international) five minutes prior to the start
time. For those unable to listen in live, a webcast archive will be
available on the company’s website for 30 days following the
call.
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, including
AXA1125-003, 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 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 such portions
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 focused on
leveraging endogenous metabolic modulators (EMMs) to pioneer a new
approach for treating complex diseases and improving health. 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. Additional muscle- and
blood-related programs are in earlier-stage development. 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 the company’s EMM product candidates, including
AXA1125, the design, status and timing of the company’s ongoing
clinical studies and planned IND-enabled clinical trials, including
with respect to the company’s planned adult and pediatric clinical
trials for AXA1125, the subject and timing of the company’s
interactions with the FDA, including with respect to an IND
application, Phase 2b clinical trial in adults and pediatric
development plans for AXA1125, and the potential of the company’s
product candidates to impact health and/or disease, including
AXA1125’s potential in NASH. 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 planned 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 we are able to collect,
other potential impacts of COVID-19 on our business and financial
results, including with respect to our ability to raise additional
capital and operational disruptions or delays, , changes in law,
regulations, or interpretations and enforcement of regulatory
guidance, whether data readouts and/or FDA feedback support our
planned timing for an IND filing, clinical trial design and target
indication for AXA1125, the clinical development and safety profile
of the company’s product candidates and their health or therapeutic
potential, whether and when, if at all, the company’s product
candidates will receive approval from the FDA or other comparable
regulatory authorities, and for which, if any, indications,
competition from other biotechnology companies, past results from
clinical studies not being representative of future results, 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/20200506005139/en/
Company/Investor Contact Jason Fredette
jfredette@axcellahealth.com (857) 320-2236
Media Contact Azeem Zeekrya HDMZ azeem.zeekrya@hdmz.com
(312) 506-5244
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