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,
Parkinson’s disease, Rett syndrome and other central nervous system
(CNS) diseases, today announced that it has dosed the first patient
in its AVATAR Phase 2 double-blind, randomized, placebo-controlled,
safety and efficacy trial of ANAVEX®2-73 for the treatment of Rett
syndrome, a rare and catastrophic neurodevelopmental disease.
ANAVEX®2-73 has already received orphan drug
designation from the FDA for the treatment of Rett syndrome.
Terence J. O'Brien, MB, BS, MD, FRACP, FRCPE,
FAHMS, FAES, principal investigator of the AVATAR study and Van
Cleef Roet Professor of Medicine (Neurology) and Head, Department
of Neuroscience & Deputy Head, Central Clinical School, Monash
University as well as Program Director – Alfred Brain, Director of
Neurology & Deputy Director of Research, Alfred Health stated,
”Rett syndrome is a devastating disease for patients and families,
for which there are currently no treatments that improve the course
of the disease. There is an urgent need for high quality,
evidence-based treatments for Rett syndrome patients.”
“We are excited to announce the first patient
dosed in our AVATAR trial of ANAVEX®2-73 for Rett syndrome,” said
Christopher U Missling, PhD, President and Chief Executive Officer
of Anavex. “We have made great effort to develop a patient-centric
clinical trial, using our learnings from broad dialogue with the
Rett syndrome community.”
The Phase 2 study, AVATAR, is a randomized
double-blind, placebo-controlled study to evaluate the safety and
efficacy of oral liquid ANAVEX®2-73 formulation to treat Rett
syndrome. Eligible patients with will be randomized to receive once
daily oral liquid ANAVEX®2-73 (combined-active group, 18 patients)
or placebo (12 patients) for up to 7 weeks (the placebo-controlled
period of the trial) including ANAVEX®2-73-specific genomic
precision medicine biomarkers. The study will enroll approximately
30 patients aged 18 or higher, and is being run at the Clinical
Trials Units of The Alfred and the Royal Melbourne Hospitals in
Victoria, Australia. All patients who participate in the study will
be eligible to receive ANAVEX®2-73 under a voluntary open label
extension protocol. This study is part of an Anavex Rett syndrome
program including further studies of ANAVEX®2-73, among them an
ongoing Phase 2 study in the US1.
In addition to Rett syndrome, Anavex has ongoing
clinical development programs for ANAVEX®2-73 for the treatment of
Alzheimer’s disease2 and Parkinson’s disease dementia3.
More information about the AVATAR study can be
found at www.anavex.com, or www.clinicaltrials.gov, identifier:
NCT03941444.
About Rett Syndrome
Rett 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 ANAVEX®2-73
ANAVEX®2-73 activates the Sigma-1 receptor (S1R)
protein, which serves as a molecular chaperone and functional
modulator involved in restoring homeostasis. In a Phase 2a
Alzheimer’s disease (AD) study, ANAVEX®2-73 has shown dose
dependent improvement in exploratory endpoints of cognition (MMSE)
and activities of daily living (ADCS-ADL). Full genomic analysis of
ANAVEX®2-73 Phase 2a AD patients was performed. The ANAVEX®2-73
Phase 2 Rett syndrome study design includes genomic biomarkers
identified in the ANAVEX®2-73 Phase 2a AD study. Studies of
ANAVEX®2-73 in a mouse model with a MECP2-null mutation that causes
neurological symptoms that mimic Rett syndrome, 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 for four weeks significantly increased
the automatic visual response in the MECP2 Rett syndrome disease
mouse (p<0.05). Additionally, chronic oral dosing daily for 6.5
weeks of ANAVEX®2-73 starting at ~5.5 weeks of age was conducted in
the MECP2 HET Rett syndrome disease mouse model assessed the
different aspects of muscular coordination, balance, motor learning
and muscular strengths, some of the core deficits observed in Rett
syndrome. Administration of ANAVEX®2-73 resulted in both
significant and dose related improvements in an array of these
behavioral paradigms in the MECP2 HET Rett syndrome disease model.
These experiments were sponsored by Rettsyndrome.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 diseases 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, recently
completed a successful 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. 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 preclinical 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
neuroinflammation and mitochondrial dysfunction. Further
information is available at www.anavex.com. You can also connect
with the company
on Twitter, Facebook 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 & Media:Email:
ir@anavex.com
1 ClinicalTrials.gov Identifier: NCT03758924
2 ClinicalTrials.gov Identifier: NCT03790709
3 ClinicalTrials.gov Identifier: NCT03774459
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