Anavex Life Sciences Announces Completion of ANAVEX(®)2-73 (blarcamesine) EXCELLENCE Phase 2/3 Rett Syndrome Clinical Trial
June 06 2023 - 6:30AM
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 announced the completion of dosing of all
participants of the placebo-controlled EXCELLENCE Phase 2/3 study
ANAVEX®2-73-RS-003 in pediatric patients with Rett syndrome. The
Company expects to announce topline results from this study in the
second half of this year.
ANAVEX®2-73 (blarcamesine) is an orally
available, small-molecule activator of the sigma-1 receptor
(SIGMAR1) which, data suggest, is pivotal to restoring cellular
homeostasis and promoting neuroplasticity.1
The completion of the randomized,
placebo-controlled EXCELLENCE Phase 2/3 study ANAVEX®2-73-RS-003
for the treatment of 92 pediatric patients with Rett syndrome ages
≥ 5 years to 17 (inclusive) was preceded by the successful
completion of both placebo-controlled Phase 2 U.S.
(ANAVEX®2-73-RS-001)2, and Phase 3 AVATAR (ANAVEX®2-73-RS-002)3
studies in adult patients with Rett syndrome.
The multi-center, double-blind clinical
EXCELLENCE study (ANAVEX®2-73-RS-003)4 in pediatric patients is
measuring safety, tolerability, and efficacy of daily oral
ANAVEX®2-73 (blarcamesine) doses or placebo. After completing the
double-blind study, eligible participants are able to join a
voluntary open-label extension study of ANAVEX®2-73
(blarcamesine).
In communication with the FDA, the Company
received the Agency’s input on the study endpoints, which were
utilized in this clinical study. The Rett Syndrome Behavior
Questionnaire (RBSQ) total score and Clinical Global Impression
Improvement Scale (CGI-I) score are co-primary endpoints in the
statistical analysis plan with specified linear mixed-effects
models for repeated measures (MMRM) as the primary analysis
methods.
ANAVEX®2-73 (blarcamesine) had previously
received Fast Track designation, Rare Pediatric Disease designation
and Orphan Drug designation from the FDA for the treatment of Rett
syndrome.
“We would like to thank the investigators and
clinical site coordinators as well as all the participating
families for their dedication to this clinical study completed with
ANAVEX®2-73 (blarcamesine),” said Christopher U. Missling, PhD,
President and Chief Executive Officer of Anavex. “Rett syndrome is
a devastating, non-inherited genetic post-natal progressive
neurodevelopmental disorder that occurs almost exclusively in girls
and leads to severe impairments, affecting nearly every aspect of
the child’s life, hence we continue our fast-paced development
program of ANAVEX®2-73 (blarcamesine) in Rett syndrome.”
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 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 and recently a Phase 2b/3
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 Advances in Experimental Medicine and Biology
Volume 964 (2017) Sigma Receptors: Their Role in Disease and as
Therapeutic Targets.2 ClinicalTrials.gov Identifier: NCT037589243
ClinicalTrials.gov Identifier: NCT039414444 ClinicalTrials.gov
Identifier: NCT04304482
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