Anavex Announces U.S. FDA Orphan Drug Designation to ANAVEX®2-73 (blarcamesine) for the Treatment of Fragile X Syndrome
November 07 2022 - 7:00AM
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 reports the U.S. Food and Drug
Administration’s (FDA) has granted Orphan Drug Designation (ODD) to
ANAVEX®2-73 (blarcamesine) for the treatment of 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 Orphan Drug Designation highlights the
potential to expand the therapeutic profile of ANAVEX®2-73 into the
largest portion of autism spectrum disorder, Fragile X syndrome,”
said Christopher U Missling, PhD, President and Chief Executive
Officer of Anavex. “We look forward to working with the Fragile X
syndrome community to rapidly advance ANAVEX®2-73 as a potential
treatment for Fragile X syndrome while we continue to expand
late-stage clinical investigation of ANAVEX®2-73 as part of its
precision medicine platform technology for both neurodevelopmental
and neurodegenerative indications.”
The FDA's Orphan Drug Designation program
provides orphan status to drugs defined as those intended for the
treatment, diagnosis or prevention of rare diseases that affect
fewer than 200,000 people in the United States. Orphan drug
designation qualifies the sponsor of the drug for certain
development incentives, including tax credits for qualified
clinical testing, prescription drug user fee exemptions and
seven-year marketing exclusivity upon FDA approval.
Anavex Life Sciences’ product portfolio platform
includes drug lead candidate ANAVEX®2-73 for the treatment of
Alzheimer’s disease, Parkinson’s disease dementia, Parkinson’s
disease and Rett syndrome and ANAVEX®3-71 for schizophrenia,
frontotemporal dementia, and Alzheimer’s disease.
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 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 M1 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 RelationsTel: 516-662-9461Email:
andrew@barwicki.com
1 https://fragilex.org/understanding-fragile-x/fragile-x-101/prevalence/
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