Anavex Life Sciences Announces First Patient Dosed in Phase 2 Clinical Trial of ANAVEX®2-73 for the Treatment of Rett Syndro...
March 18 2019 - 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 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 U.S. 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.
“We are very pleased to report the first patient
dosing of the ANAVEX®2-73 Rett syndrome Phase 2 study in the U.S.,”
said Christopher U Missling, PhD, President and Chief Executive
Officer of Anavex. “This is an important step toward achieving
clinical data for the third indication for ANAVEX®2-73 also
incorporating genomic precision medicine biomarkers.”
The Phase 2 study is a randomized double-blind,
placebo-controlled safety, tolerability, pharmacokinetic and
efficacy study of oral liquid ANAVEX®2-73 formulation to treat Rett
syndrome. Pharmacokinetic and dose finding will be investigated in
a total of 15 patients over a 7-week treatment period including
ANAVEX®2-73-specific genomic precision medicine biomarkers. All
patients who participate in the study will be eligible to receive
ANAVEX®2-73 under a voluntary open label extension protocol.1 This
study is part of a planned Anavex Rett syndrome program including
placebo-controlled safety and efficacy evaluation of ANAVEX®2-73
over a 3 month treatment period.
In addition to Rett syndrome, Anavex has ongoing
clinical development programs for ANAVEX®2-73 for the treatment of
Alzheimer’s disease and Parkinson’s disease dementia.
About the ANAVEX®2-73 Rett Syndrome Clinical Phase 2
Study
For patients who wish to learn more
about this study, please visit the IRB-approved website,
www.rettsyndrometrial.com.
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 Rettsyndrome.org
Rettsyndrome.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 a major private
funder of Rett syndrome research, Rettsyndrome.org has funded over
$46M 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, 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:Scott GordonCore
IRscottg@coreir.com
1 ClinicalTrials.gov Identifier: NCT03758924
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