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) diseases, today announced that preclinical data of
ANAVEX®2-73 (blarcamesine) in Fragile X Syndrome were published in
the peer-reviewed journal, Scientific Reports.
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 study evaluated doses of ANAVEX®2-73 in Fmr1
knockout (KO) mice, a validated animal model for the disease, which
resulted in the reversal of hyperactivity and restoration of
associative learning as well as reduction in anxiety-like and
perseverative behaviors. Positron emission tomography (PET) and ex
vivo autoradiographic studies, using the highly selective SIGMAR1
PET ligand [18F]FTC-146, demonstrated ANAVEX®2-73’s dose dependent
receptor occupancy. Additionally, ANAVEX®2-73 also restored pAkt
and BDNF levels in the hippocampus, which are signaling pathways
particularly affected in Fragile X Syndrome. ANAVEX®2-73 also
showed a good safety profile.
"Testing novel drugs that can safely improve the
symptoms of Fragile X Syndrome is a high priority,” said Walter E.
Kaufmann, M.D., Chief Medical Officer of Anavex and corresponding
author of the paper. “The present findings support the viability of
SIGMAR1 as a therapeutic target in Fragile X Syndrome, and the
clinical potential of ANAVEX®2-73 (blarcamesine) in Fragile X
Syndrome and other neurodevelopmental disorders.”
The study, “Effects of the Sigma-1 Receptor
Agonist Blarcamesine in a Murine Model of Fragile X Syndrome:
Neurobehavioral Phenotypes and Receptor Occupancy,” is the basis
for a Phase 2/3 ANAVEX®2-73 study in Fragile X Syndrome. The fact
that the investigation involved chronic administration, as opposed
to acute dosing, provides additional evidence in favor of the
clinical use of ANAVEX®2-73. Since the behavioral paradigms reflect
the involvement of multiple cortical and subcortical regions, their
marked improvement by ANAVEX®2-73 suggest widespread activation of
SIGMAR1 by the drug and modulation of multiple neural pathways.
Indeed, the observation of normalization of pAkt and BDNF levels
after ANAVEX®2-73 administration, in a brain region critical for
cognition and behavior, is also a finding with important
implications for Fragile X Syndrome and other synaptic
disorders.
Altogether, these neurobehavioral, biochemical,
and imaging data demonstrate that corresponding doses of
ANAVEX®2-73 that yield measurable receptor occupancy are effective
for substantially correcting key synaptic and behavioral phenotypes
in Fmr1 KO mice. The data also suggest that these positive effects
are mediated by SIGMAR1 activation in multiple brain regions, where
ANAVEX®2-73 binds to the receptor in a dose-dependent and
genotype-independent manner. The study was supported by the FRAXA
Research Foundation.
“We look forward to initiating a double-blind,
placebo-controlled Phase 2/3 ANAVEX®2-73 study in Fragile X
Syndrome,” said Christopher U Missling, PhD, President and Chief
Executive Officer of Anavex. “We are intrigued about the clear
preclinical data providing potential to expand the therapeutic
profile of ANAVEX®2-73 into the largest portion of addressable
market of autism spectrum disorder, Fragile X Syndrome. This is
further evidence of the potential of ANAVEX®2-73 as a platform
technology of precision medicine."
The full paper can be accessed online at:
www.nature.com/articles/s41598-021-94079-7.
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 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) diseases, pain, and various types of
cancer. Anavex’s lead drug candidate, ANAVEX®2-73 (blarcamesine),
successfully completed a Phase 2a clinical trial for Alzheimer’s
disease and recently a Phase 2 proof-of-concept study in
Parkinson’s disease dementia and a Phase 2 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 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 Relations
Tel: 516-662-9461Email: andrew@barwicki.com
1 https://fragilex.org/understanding-fragile-x/fragile-x-101/prevalence/
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