- Initial clinical sites activated and patient screening
underway
- Potential to improve the standard of care for cirrhotic
patients by addressing multiple key drivers of overt hepatic
encephalopathy (OHE)
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
patient screening for its global Phase 2 clinical trial of AXA1665,
the company’s multi-targeted oral product candidate for the
reduction in risk of recurrent overt hepatic encephalopathy
(OHE).
“OHE is a condition that has devastating consequences for
patients and families and places a substantial economic burden on
the overall healthcare system,” said Andres Duarte-Rojo, M.D.,
M.S., Ph.D., Department of Medicine, Division of Gastroenterology,
Hepatology and Nutrition at the University of Pittsburgh and the
trial’s principal investigator. “Given the limitations of today’s
standards of care and the lack of clinical development activity
beyond ammonia-focused agents, it also is an area in dire need of
medical innovation. In prior clinical studies, AXA1665 has
demonstrated the potential to have a multifactorial benefit to
patients inclusive of improvements in amino acid balancing, ammonia
metabolism, muscle function and neurocognition. I look forward to
investigating AXA1665’s ability to replicate these findings and
reduce OHE events in EMMPOWER.”
Axcella has branded the Phase 2 trial EMMPOWER based on the
potential for AXA1665, an EMM composition, to help patients,
physicians and other caregivers overcome significant challenges
associated with cirrhosis and OHE. This randomized, double-blind,
placebo-controlled, multi-center investigation will evaluate 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 lactulose ± rifaximin
(stratified by rifaximin use) will be 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 four-week safety
follow-up period.
“The initiation of EMMPOWER is an important milestone, ushering
in an exciting new era of clinical development for Axcella as we
seek to tackle a variety of complex diseases utilizing proprietary,
multi-targeted EMM compositions,” said Bill Hinshaw, President and
Chief Executive Officer of Axcella.
The EMMPOWER trial will be conducted globally at more than 70
clinical sites with a primary endpoint assessing the proportion of
patients with a ≥2 point increase in the psychometric hepatic
encephalopathy score (PHES) after the 24-week treatment period. The
trial’s key secondary endpoints will focus on the proportion of
patients experiencing an OHE breakthrough event and time to first
OHE breakthrough event, including time to hospitalization. Other
secondary endpoints include changes in physical function, including
the liver frailty index, and patient-reported outcomes. Measures of
circulating ammonia, amino acid profile, and inflammation-related
markers will also be included as endpoints.
“By taking on the core drivers of OHE more comprehensively, we
not only see the potential for AXA1665 to improve today’s standard
of care, but also to eventually become a first-line treatment
option,” said Alison Schecter, M.D., President of R&D at
Axcella. “On behalf of the Axcella organization, we would like to
express our gratitude to the many patients and dozens of clinical
global sites that will be involved in this trial as we seek to
transform the OHE treatment paradigm.”
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 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 AXA1665, the potential for AXA1665 to
improve upon the standard of care for OHE patients and reduce OHE
events, 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210630005091/en/
Company Jason Fredette jfredette@axcellahealth.com (857)
320-2236
Axcella Health (NASDAQ:AXLA)
Historical Stock Chart
From Aug 2024 to Sep 2024
Axcella Health (NASDAQ:AXLA)
Historical Stock Chart
From Sep 2023 to Sep 2024