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
diseases including Alzheimer’s disease, other central nervous
system (CNS) diseases, pain, and various types of cancer, today
announced new preclinical data for ANAVEX 2-73 in the
neurodevelopmental disorders Angelman syndrome, Fragile X syndrome
and Rett syndrome. The data was presented at the Antiepileptic Drug
Trials XIV 2017 Conference in Aventura, Florida.
Characterized as an autism spectrum disorder, Angelman syndrome
is a rare neuro-genetic disorder that occurs in one in 15,000 live
births. Individuals with Angelman syndrome exhibit severe
cognitive and physical impairments, including ataxia, intellectual
disability, speech impairment, sleep disorders, and seizures (the
latter being present in over 80 percent of affected
individuals).
ANAVEX 2-73 (10 mg/kg IP dosed daily for 14 days) was assessed
in the Ubiquitin-protein ligase E3A (Ube3a) mouse model of Angelman
syndrome for the development of audiogenic seizures in
3-4-month-old animals. Sponsored by FAST (Foundation for
Angelman Syndrome Therapeutics) and conducted in the laboratory of
Associate Professor Anne Anderson, MD, at Baylor College of
Medicine in Houston, Texas. The results indicated that ANAVEX 2-73
administration significantly reduced audiogenic-induced seizures
(p<0.01).
“We are impressed with the positive anti-seizure signal of
ANAVEX 2-73,” said Paula M. Evans, Chairperson of FAST. “Most
people with Angelman syndrome have recurrent seizures throughout
their lives, which is a serious challenge for the individual and
caretakers.”
ANAVEX 2-73 has previously been shown to normalize an array of
behavioral impairments in a mouse model of Fragile X (see
announcement from June 6, 2016). In a recent study sponsored by
Fraxa Research Foundation (FRAXA), the effects of ANAVEX 2-73 (1
mg/kg IP dosed twice daily for 14 days) on potential biomarkers
were evaluated in Fragile X mental retardation 1 knockout (Fmr1 KO)
mice. Vehicle-treated Fmr1 KO mice demonstrate significantly lower
brain-derived neurotrophic factor (BDNF) expression in the
hippocampus compared to wild-type mice. The experiments
demonstrated that ANAVEX 2-73 restored hippocampal BDNF expression
to normal levels (p<0.05). BDNF under-expression has been
observed in many neurodevelopmental and neurodegenerative
pathologies. BDNF signaling promotes maturation of both excitatory
and inhibitory synapses. ANAVEX 2-73 normalization of BDNF
expression could be a contributing factor for the positive data
observed in both neurodevelopmental and neurodegenerative
disorders.
In addition, ANAVEX 2-73 has been further evaluated in the MECP2
Rett syndrome disease mouse model. In an experiment sponsored by
Rettsyndrome.org, ANAXEX 2-73 was evaluated in automatic visual
response and respiration tests in 7-month old mice, an age at which
advanced pathology is evident. Vehicle-treated methyl-CpG binding
protein 2 (MECP2) mice demonstrated fewer automatic visual
responses than wild-type mice. Treatment with ANAVEX 2-73 (30
mg/kg/day PO) for four weeks significantly increased the automatic
visual response in the MECP2 Rett syndrome disease mouse
(p<0.05).
In Rett syndrome there are disturbances in respiration
characterized by an irregular breathing pattern and frequent apnea
that are common and debilitating. A trend was observed in MECP2
mice treated with ANAVEX 2-73 – that is, a reduction in apnea
counts to levels comparable to those observed in wild-type
animals.
“We are encouraged by these converging positive preclinical
findings, confirming the potential therapeutic effect of ANAVEX
2-73 in neurodevelopmental diseases,” said Christopher U. Missling,
PhD, President and Chief Executive Officer of Anavex. “We plan to
continue exploring the links between preclinical findings and
clinical manifestations in our ongoing translational research
efforts.”
The presentation is available on the Anavex website.
About Angelman SyndromeAngelman syndrome (AS)
is a rare neuro-genetic disorder that affects approximately one in
15,000 people – about 500,000 individuals worldwide. Children and
adults with Angelman syndrome typically have balance issues, motor
impairment and debilitating seizures. Some individuals never walk.
Most do not speak. Individuals with Angelman syndrome require
continuous care and are unable to live independently. They have a
normal life expectancy. Typical characteristics of Angelman
syndrome are not usually evident at birth. People with the disorder
have feeding difficulties as infants and noticeable delayed
development around 6-12 months of age. They need intensive
therapies to help develop functional skills. In most cases,
Angelman syndrome isn't genetically inherited. Angelman syndrome
affects every race and both genders. It is often misdiagnosed as
autism or cerebral palsy. For more information about Angelman
syndrome, please visit www.CureAngelman.org.
About FAST (Foundation for Angelman Syndrome
Therapeutics)FAST is a Section 501(c)(3) nonprofit
research organization singularly focused on funding research that
holds the greatest promise of treating Angelman syndrome. FAST is
the largest, non-governmental funder of Angelman-specific research.
Paula Evans, the mother of a young girl with Angelman syndrome,
founded FAST in 2008. The foundation is based in Downers Grove,
Ill.
About Fragile X Syndrome and Autism Spectrum
DisordersFragile X syndrome is the most common form of
inherited intellectual disability and the most frequent single gene
cause of autism, affecting approximately one in 4,000 males and one
in 6,000 females. The disorder is caused by the unstable expansion
of a CGG repeat in the FMR1 gene and abnormal methylation, which
results in suppression of FMR1 transcription and decreased protein
levels in the brain. The average age for diagnosis of Fragile X
syndrome in boys and girls is 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 Fragile X syndrome-autism spectrum disorders link
over the past decade. Since many children with Fragile X syndrome
are interested in social interactions, they may not meet the
diagnostic criteria for autism spectrum disorders, even though they
exhibit some features of autism spectrum disorders such as poor eye
contact, shyness, social anxiety, hand-flapping and sensory issues.
Autism is much more common in boys with Fragile X syndrome than in
girls with Fragile X syndrome. According to the Centers for Disease
Control and Prevention (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 disorders.
About FRAXAFRAXA’s mission is to find effective
treatments and a cure for children and adults with Fragile X and
related disorders such as autism. FRAXA has funded over $25,000,000
in biomedical research, yielding discoveries which can change the
lives of all families struggling with Fragile X. FRAXA Research
Foundation is a non-profit, 501(c)(3) organization which is
dedicated to funding biomedical research for improved treatment and
a cure for Fragile X, the leading inherited cause of intellectual
disability and autism.
About Rett SyndromeRett syndrome is a rare,
non-inherited genetic postnatal progressive neurodevelopmental
disorder that occurs almost exclusively in girls and leads to
severe impairments, affecting nearly every aspect of the child’s
life: their ability to speak, walk, eat and even breathe easily.
The hallmark of Rett syndrome is near constant repetitive hand
movements while awake. It is characterized by normal early growth
and development (6 to 18 months) followed by a slowing of
development, loss of purposeful use of the hands, distinctive hand
movements, slowed brain and head growth, problems with walking,
seizures and intellectual disability. There is currently no cure
for Rett syndrome and treatment of the disorder is symptomatic.
Management of symptoms is done through a multidisciplinary approach
utilizing medication for motor difficulties, breathing
irregularities and control of seizures through anticonvulsant
drugs. Rett syndrome is caused by mutations in the MECP2 gene and
strikes all racial and ethnic groups and occurs worldwide in
approximately one in every 10,000 to 15,000 live female births.
About Rettsyndrome.orgRettsyndrome.org is the
most comprehensive nonprofit organization dedicated to accelerating
research of treatments and a cure for Rett syndrome and related
disorders while providing information and family empowerment. As
the world’s leading private funder of Rett syndrome research,
Rettsyndrome.org has funded over $40M in high-quality,
peer-reviewed research grants and programs to date. The
organization hosts the largest global gathering of Rett researchers
and clinicians to establish research direction for the future.
Rettsyndrome.org, a 501(c)(3) organization, has earned Charity
Navigator’s most prestigious 4 star rating year after year. To
learn more about our work and Rett syndrome, visit
www.rettsyndrome.org or call (800) 818-7388 (RETT).
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 diseases including Alzheimer’s disease,
other central nervous system (CNS) diseases, pain and various types
of cancer. Anavex’s lead drug candidate, ANAVEX 2-73, recently
completed successfully a Phase 2a clinical trial for Alzheimer’s
disease. 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. It has also
exhibited anticonvulsant, anti-amnesic, neuroprotective and
anti-depressant properties in animal models, indicating its
potential to treat additional CNS disorders, including epilepsy and
others. The Michael J. Fox Foundation for Parkinson’s Research has
awarded Anavex a research grant to develop ANAVEX 2-73 for the
treatment of Parkinson’s disease to fully fund a preclinical study,
which could justify moving ANAVEX 2-73 into a Parkinson’s disease
clinical trial. ANAVEX 3-71, also targeting sigma-1 and M1
muscarinic receptors, is a promising preclinical drug candidate
demonstrating disease modifications against the major Alzheimer’s
hallmarks in transgenic (3xTg-AD) mice, including cognitive
deficits, amyloid and tau pathologies, and also with beneficial
effects on neuroinflammation and mitochondrial dysfunctions.
Further information is available at www.anavex.com. You can
also connect with the company
on Twitter, Facebook and LinkedIn.
Forward-Looking StatementsStatements 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 Development
Toll-free: 1-844-689-3939
Email: info@anavex.com
Investors:
Matthew Haines
River East Investor Relations, LLC
917-733-9297
mhaines@rivereastir.com
Media:
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Dobson Media Group
203-258-0159
dennisdobsonjr@dobsonmediagroup.com
Social Media:
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Gotham Communications
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