Activity demonstrated across targeted
biologies, including amino acid balance, ammonia handling, muscle
function and neurocognition
Presentation includes new subject-specific
neurocognition and muscle function data
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 positive data from the company’s AXA1665-002
clinical study were presented last night at the Digestive Disease
Week (DDW) 2021 Annual Meeting by Dr. Arun Sanyal, Professor in the
Virginia Commonwealth University (VCU) Department of Internal
Medicine and Education Core Director in the VCU Center for Clinical
and Translational Research.
AXA1665-002 was a placebo-controlled, randomized clinical study
that investigated the safety, tolerability and physiological impact
of AXA1665 in 60 subjects with mild (Child Pugh A) and moderate
(Child Pugh B) hepatic insufficiency. Subjects in the study were
randomized in a 2:2:1 ratio to receive either 29.4 g or 53.8 g of
AXA1665 or a matched placebo in three divided doses per day for 12
weeks with a four-week follow up.
Both doses of AXA1665 were shown to be safe and well tolerated
in the study. Additionally, activity was demonstrated across
multiple targeted biologies, including amino acid balance, ammonia
handling and muscle structure and function. Most importantly, dose
dependent, directionally consistent changes were noted across all
three psychometric tests that were utilized in AXA1665-002. These
included a statistically significant change in the psychometric
hepatic encephalopathy score (PHES) for patients receiving the 53.8
g dose of AXA1665 versus placebo. PHES is a highly specific
assessment to diagnose hepatic encephalopathy.
The DDW oral presentation included new subject-level data in
certain measures of neurocognition and muscle function.
Specifically, a positive change in PHES was seen from baseline to
week 12 in a dose-proportional manner in subjects receiving AXA1665
compared with placebo, with a majority of subjects in the high dose
arm achieving a clinically relevant ≥2 point improvement in PHES.
Additionally, while this study primarily enrolled a non-sarcopenic
population, a higher proportion of AXA1665 treated subjects
(26-40%) versus placebo (14%) achieved a ≥0.3 unit decrease in the
liver frailty index. Previous studies suggest that a ≥0.3 reduction
in the LFI score may correlate with an improved ability to conduct
activities of daily living in subjects with end-stage liver
disease.
“Given the clinical evidence supporting the role of muscle
dysfunction and sarcopenia in neurocognitive impairment and
morbidity in cirrhosis, there is a need for treatments that can
provide OHE patients with benefits beyond those provided by
conventional approaches,” said Dr. Sanyal. “Based on the finding
from this most recent clinical study, I believe AXA1665 provides a
novel approach to the treatment of OHE and holds the potential to
address important unmet needs.”
A video recording of Dr. Sanyal’s presentation at DDW 2021 is
now available in the Publications section of Axcella’s website at
https://axcellahealth.com/publications/.
Axcella is now initiating a 24-week Phase 2 clinical trial that
will compare the 53.8 g/day dose of AXA1665 versus placebo in
approximately 150 patients with more advanced liver disease who
have experienced at least one prior OHE event and have
neurocognitive dysfunction at screening. Additional trial
information can be found on https://clinicaltrials.gov/ via the
identifier NCT04816916.
About AXA1665 and Overt Hepatic
Encephalopathy (OHE)
Hepatic encephalopathy (HE), one of the most common
complications of cirrhosis, is a condition involving amino acid
imbalance, ammonia toxicity and muscle wasting, all of which
contribute to diminished brain function. OHE refers to the presence
of neurological abnormalities that are clinically apparent and do
not require specialized psychometric testing. OHE is well
established as a significant cause of morbidity and mortality in
the cirrhotic population and is an area that continues to have
unmet medical needs.
AXA1665, Axcella’s product candidate for reduction in risk of
recurrent OHE, is a composition of eight amino acids and
derivatives that is designed to target multiple metabolic pathways
intersecting key organ systems, including the liver, muscle and
gut. In prior clinical studies, this oral product candidate has
been safe, well tolerated and has demonstrated the potential to
improve ammonia handling, physical function, amino acid balance and
neurocognition with a safe and well tolerated profile. AXA1665 is
now in Phase 2 development.
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 AXA1665, the potential for AXA1665 to
improve upon the standard of care for OHE patients and address
unmet patient needs, 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 AXA1665, 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 AXA1665, the clinical development and safety
profile of AXA1665 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/20210524005208/en/
Company Contact Jason Fredette
jfredette@axcellahealth.com (857) 320-2236
Axcella Health (NASDAQ:AXLA)
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