- Multifactorial liver and systemic biological activity seen for
both AXA1125 and AXA1957 in adult subjects with NAFLD at 16 weeks
with onset as early as eight weeks
- Interim data support candidates’ potential to become
foundational NASH therapies
- Top-line data from full study expected in mid-2020
- Five clinical readouts expected in 2020 from Axcella’s liver
and hematology programs
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 provided an update from its ongoing clinical study in
adult subjects with non-alcoholic fatty liver disease (NAFLD) and
announced its anticipated milestones for 2020.
Interim Analysis of Ongoing Clinical
Study of AXA1125/AXA1957 (AXA1125-003)
Axcella has completed the enrollment of 102 adult subjects with
NAFLD in an ongoing randomized, dose-ranging study in which
subjects receive either AXA1125, one of two AXA1957 doses or
placebo for 16 weeks. This study, which is being conducted at 18
U.S. medical centers, is assessing the impact of AXA1125/AXA1957 on
safety, tolerability and physiology, as measured by a comprehensive
panel of imaging and soluble biomarkers related to metabolism,
inflammation and fibrosis.
An interim analysis has been conducted that includes data from
approximately half of the study population through the full 16
weeks of administration. The analysis shows that AXA1125 and both
doses of AXA1957 have been safe and well tolerated to date.
Additionally, both AXA1125 and AXA1957 demonstrated clinically
relevant responses on the three biological nodes fundamental to
liver health and disease: metabolism (MRI-PDFF and HOMA-IR),
inflammation (ALT, CK-18, cT1) and fibrogenesis (proC3). The onset
of response in some biomarkers was seen as early as the eight-week,
post-baseline assessment with continued improvement through 16
weeks.
“These interim non-invasive data indicate that AXA1125 and
AXA1957 are having a positive impact on multiple dysregulated
biological pathways related to health and disease that are common
in NAFLD/NASH patients,” said Stephen A. Harrison, M.D., the
principal investigator (PI) of the study, medical director of
Pinnacle Clinical Research in San Antonio, TX, and visiting
professor of Hepatology at the University of Oxford, UK. “These are
particularly encouraging early findings, providing hope that a
multifactorial effect can be generated from novel compositions of
endogenous metabolic modulators.”
These findings will be included in the company’s presentation at
the J.P. Morgan Healthcare Conference next week. Details regarding
this presentation will be provided in a separate press release.
“We are pleased with the swift pace of enrollment and the data
generated to date in our sizable clinical study of AXA1125 and
AXA1957. The interim analysis increases our confidence in the
potential for these candidates to become foundational therapeutics
for NASH patients,” said Bill Hinshaw, President and CEO of
Axcella. “Our excitement continues to build as we begin a major
year for the company, with five planned clinical readouts and the
initiation of our first planned Phase 2b/3 clinical trial in
2020.”
2020 Milestones
Liver Product Candidates
- AXA1125 and AXA1957 are being investigated in the
aforementioned ongoing clinical study in adult NAFLD subjects. The
company expects to report top-line data from this study in
mid-2020.
- AXA1957 is also being investigated in a placebo-controlled
ongoing clinical study enrolling approximately 30 adolescent
subjects with NAFLD (AXA1957-002). This study is assessing the
impact of AXA1957 on safety, tolerability and physiology. Top-line
data from this study are expected to be reported in the second half
of 2020.
- AXA1665 is being investigated in a 12-week, placebo-controlled
ongoing clinical study that is enrolling approximately 60 subjects
with mild and moderate hepatic insufficiency (AXA1665-002). This
study is assessing the impact of AXA1665 on safety, tolerability
and physiology. AXA1665 has been safe and well tolerated in
clinical studies conducted to date. The company expects to report
top-line data from the study in mid-2020. It also plans to submit
an IND and initiate a Ph2b/3 clinical trial in the second half of
2020 to study AXA1665’s ability to reduce the risk of recurrence of
overt hepatic encephalopathy (OHE).
Blood Product Candidate
- AXA4010, Axcella’s first hematology product candidate, is being
investigated in an ongoing clinical study to assess safety,
tolerability and impact on red blood cell physiology in
approximately 24 subjects with sickle cell disease ages 12 and
older in a staged, sequential design of three separate cohorts of
eight subjects, each for up to 12 weeks (AXA4010-001). Axcella
expects to report top-line data from this study in the second half
of 2020.
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 Ongoing Clinical Studies
Each of the company’s ongoing clinical studies are being
conducted as non-investigational new drug (IND) application
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 AXA1125/1957, subsequent 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 that are
engineered in distinct combinations and ratios to simultaneously
impact multiple biological pathways. Axcella’s pipeline includes
lead therapeutic candidates for overt hepatic encephalopathy (OHE)
and non-alcoholic steatohepatitis (NASH). 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 development potential of the company’s product
candidates, including AXA1665, AXA1125 and AXA1957, potential
expansion into new therapeutic fields, the ability of endogenous
metabolic modulators to impact dysregulated metabolism and health
and the timing of the company’s clinical studies and trials and the
timing of receipt of data from the same. 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
breadth and potential uses of the company’s pipeline of product
candidates, the potential of AXA1125 and AXA1957 to become
foundational therapies, interim and topline data readouts and
timing of the same, the planned timing of an IND filing for
AXA1665, the strength of the AXA Development Platform, the
efficiency of the company’s discovery and development approach, 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 U.S. Food and Drug Administration, or other
comparable regulatory authorities, and for which, if any,
indications, competition from other biotechnology companies, the
company’s liquidity, its ability to successfully develop product
candidates through current and future milestones on the anticipated
timeline, if at all, past results from non-IND clinical studies not
being representative of future results, and other risks identified
in the company’s SEC filings, including Axcella’s 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/20200108005145/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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