Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq:
AVXL), a clinical-stage biopharmaceutical company developing
differentiated therapeutics for the treatment of neurodegenerative
and neurodevelopmental disorders including Alzheimer’s disease,
Parkinson’s disease, Rett syndrome and other central nervous system
(CNS) disorders, today announced the peer-reviewed publication in
American Journal of Medical Genetics identifying a blood biomarker
for assessing treatment effect of ANAVEX®2-73 in Fragile X
Syndrome, which will be also included in the planned clinical trial
of ANAVEX®2-73 in Fragile X Syndrome.
Fragile X Syndrome (FXS) is the most common form
of inherited intellectual disability and the most frequent single
gene cause of autism spectrum disorder with an estimated population
of approximately 62,500 in the US and 1,088,500 worldwide.1 At
present, there is no approved treatment for Fragile X Syndrome.
The findings demonstrate that treatment effect
of ANAVEX®2-73 resulting in reversal of hyperactivity, restoration
of associative learning and reduction of anxiety in a mouse model
of Fragile X Syndrome are also associated with improvements in key
blood signaling biomarkers, which are measurable in patients with
Fragile X Syndrome as well.
Previously reported improvements in cognitive
and behavioral paradigms in a mouse model of Fragile X Syndrome,
after ANAVEX®2-73 administration, are associated with parallel
improvements in peripheral lymphocyte signaling abnormalities
(i.e., decrease in activated/phosphorylated Akt and ERK).2 Fragile
X Syndrome is characterized by multiple cell signaling
abnormalities, including increased activation of the PI3K/Akt/mTOR
and MAPK/ERK pathways. These have been demonstrated in brain
samples from mouse models as well as in lymphocytes from
patients.
The present publication, “Brain cell signaling
abnormalities are detected in blood in a murine model of Fragile X
syndrome and corrected by Sigma-1 receptor agonist Blarcamesine,”
is one of the first studies showing that key signaling
abnormalities can also be detected and corrected in mouse
lymphocytes, providing a solid foundation for the use of lymphocyte
signaling biomarkers in clinical trials involving ANAVEX®2-73. The
study was supported by the FRAXA Research Foundation.
“Evaluations of lymphocyte cell signaling in
mouse models of Fragile X Syndrome are feasible and support
corresponding assessments in affected individuals,” said Walter E.
Kaufmann, M.D., Chief Scientific Officer of Anavex and
corresponding author of the publication. “These analyses have the
potential for monitoring response to treatment, particularly for
drugs correcting multiple pathway abnormalities such as sigma-1
receptor agonist ANAVEX®2-73. Implications of this work extend
beyond FXS to most neurologic disorders associated with abnormal
cell signaling.”
Data suggests that activation of the sigma-1
receptor is pivotal to restoring neural cell homeostasis and
promoting neuroplasticity.3
“These additional biomarker findings in Fragile
X Syndrome provide further evidence of potential to expand the
therapeutic profile of ANAVEX®2-73 into the largest portion of
addressable market of autism spectrum disorder, Fragile X
Syndrome,” said Christopher U Missling, PhD, President and Chief
Executive Officer of Anavex. “We look forward to initiating a
double-blind, placebo-controlled Phase 2/3 ANAVEX®2-73 study in
Fragile X Syndrome. This is further evidence of the potential of
ANAVEX®2-73 as a platform technology of precision medicine."
The paper can be accessed online at:
https://onlinelibrary.wiley.com/doi/10.1002/ajmg.a.62853
Anavex Life Sciences’ product portfolio platform
includes orally available small molecule drug lead candidate
ANAVEX®2-73 for the treatment of Alzheimer’s disease, Parkinson’s
disease and Rett syndrome and ANAVEX®3-71 for frontotemporal
dementia.
About Fragile X Syndrome and Autism
Spectrum Disorder
Fragile X Syndrome is the most common form of
inherited intellectual disability and the most frequent single gene
cause of autism, affecting approximately 1 in 4,000 males and 1 in
6,000 females. The disorder is caused by the unstable expansion of
a CGG repeat in the FMR1 gene that leads to abnormal methylation
and suppression of FMR1 transcription with the resulting decrease
in protein levels in the brain and other tissues. The average age
of Fragile X Syndrome diagnosis for boys and girls are 35 to 37
months and 42 months, respectively. Behavioral abnormalities,
including autism spectrum disorder, are common.
Autism spectrum disorder is a behavioral
diagnosis while Fragile X Syndrome is a medical/genetic diagnosis.
Many studies have evaluated the link between Fragile X Syndrome and
autism spectrum disorder over the last few decades. Since many
children with Fragile X Syndrome are interested in social
interactions, they may not meet the diagnostic criteria for autism
spectrum disorder, even though they exhibit some features such as
poor eye contact, shyness, social anxiety, hand-flapping and
sensory issues. Autism is much more common in boys than in girls
with Fragile X Syndrome. According to the CDC, a national parent
survey found that 46% of males and 16% of females with Fragile X
Syndrome have been diagnosed or treated for autism spectrum
disorder.
About FRAXA Research Foundation
FRAXA’s mission is to find effective treatments
and ultimately a cure for Fragile X Syndrome. FRAXA directly funds
research grants and fellowships at top universities around the
world. FRAXA partners with biomedical and pharmaceutical companies,
large and small, to bridge the gap between research discoveries and
actual treatments. Treatments for Fragile X Syndrome are likely to
help people affected by autism, Alzheimer’s, and other brain
disorders. More information is available at www.fraxa.org.
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a
publicly traded biopharmaceutical company dedicated to the
development of novel therapeutics for the treatment of
neurodegenerative and neurodevelopmental disorders including
Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other
central nervous system (CNS) diseases, pain, and various types of
cancer. Anavex’s lead drug candidate, ANAVEX®2-73 (blarcamesine),
has successfully completed a Phase 2a clinical trial for
Alzheimer’s disease, a Phase 2 proof-of-concept study in
Parkinson’s disease dementia and both a Phase 2 and a Phase 3 study
in adult patients with Rett syndrome. ANAVEX®2-73 is an orally
available drug candidate that restores cellular homeostasis by
targeting sigma-1 and muscarinic receptors. Preclinical studies
demonstrated its potential to halt and/or reverse the course of
Alzheimer’s disease. ANAVEX®2-73 also exhibited anticonvulsant,
anti-amnesic, neuroprotective, and anti-depressant properties in
animal models, indicating its potential to treat additional CNS
disorders, including epilepsy. The Michael J. Fox Foundation for
Parkinson’s Research previously awarded Anavex a research grant,
which fully funded a preclinical study to develop ANAVEX®2-73 for
the treatment of Parkinson’s disease. ANAVEX®3-71, which targets
sigma-1 and muscarinic M1 receptors, is a promising clinical stage
drug candidate demonstrating disease-modifying activity against the
major hallmarks of Alzheimer’s disease in transgenic (3xTg-AD)
mice, including cognitive deficits, amyloid, and tau pathologies.
In preclinical trials, ANAVEX®3-71 has shown beneficial effects on
mitochondrial dysfunction and neuroinflammation. Further
information is available at www.anavex.com. You can also connect
with the company on Twitter, Facebook, Instagram and LinkedIn.
Forward-Looking Statements
Statements in this press release that are not
strictly historical in nature are forward-looking statements. These
statements are only predictions based on current information and
expectations and involve a number of risks and uncertainties.
Actual events or results may differ materially from those projected
in any of such statements due to various factors, including the
risks set forth in the Company’s most recent Annual Report on Form
10-K filed with the SEC. Readers are cautioned not to place undue
reliance on these forward-looking statements, which speak only as
of the date hereof. All forward-looking statements are qualified in
their entirety by this cautionary statement and Anavex Life
Sciences Corp. undertakes no obligation to revise or update this
press release to reflect events or circumstances after the date
hereof.
For Further Information:
Anavex Life Sciences Corp.Research &
Business DevelopmentToll-free: 1-844-689-3939Email:
info@anavex.com
Investors:Andrew J.
BarwickiInvestor RelationsTel: 516-662-9461Email:
andrew@barwicki.com
1
https://fragilex.org/understanding-fragile-x/fragile-x-101/prevalence/2
https://www.nature.com/articles/s41598-021-94079-73 Advances in
Experimental Medicine and Biology Volume 964 (2017) Sigma
Receptors: Their Role in Disease and as Therapeutic Targets.
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