Anavex Life Sciences Announces Publication of Foundational Data for ANAVEX®2-73 (blarcamesine) in Rett Syndrome
November 06 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 disorders including Alzheimer’s disease,
Parkinson’s disease, Rett syndrome and other central nervous system
(CNS) diseases, today announced that preclinical data of
ANAVEX®2-73 (blarcamesine) in Rett syndrome were published in the
peer-reviewed journal, Pharmacology, Biochemistry and Behavior.
Rett syndrome, a rare genetic neurological
disorder that afflicts mostly girls, is caused by a genetic defect
associated with severe physical and cognitive impairments. The
study evaluated doses of ANAVEX®2-73 (blarcamesine) in heterozygous
MECP2 mice, a validated animal model for the disease, which
resulted in improvements in motor, sensory, and autonomic
phenotypes related to the disease. These included motor
coordination and balance, acoustic and visual responses, and other
improvements. ANAVEX®2-73 (blarcamesine) also showed a good safety
profile.
"Testing novel drugs that can safely improve the
symptoms of Rett syndrome is a high priority,” said Walter E
Kaufmann, M.D., Chief Medical Officer of Anavex and corresponding
author of the paper. “ANAVEX®2-73 (blarcamesine) is a sigma-1
receptor agonist and muscarinic receptor modulator with a strong
safety record. The drug’s ability to enhance calcium homeostasis
and improve the mitochondrial and cellular functions that underlie
pathological processes and compensatory mechanisms in Rett
syndrome, make ANAVEX®2-73 (blarcamesine) a promising
candidate."
The study, “ANAVEX®2-73 (blarcamesine), a
Sigma-1 receptor agonist, ameliorates neurologic impairments in a
mouse model of Rett syndrome,” served as a proof-of-concept for the
ongoing Phase 2 ANAVEX2-73-RS-001 study (NCT03758924) as well as
for upcoming pediatric studies. Preliminary results from the first
cohort of this study were recently presented at the 6th Annual
European Rett Syndrome Conference. The data described significant
improvements in the two global efficacy endpoints: the Rett
Syndrome Behaviour Questionnaire (RSBQ) Total score and the
Clinical Global Impression – Improvement (CGI-I); as well as in the
RSBQ Hand Behaviours and the RSBQ Breathing Abnormalities subscale
scores. Efficacy signals on both caregiver- and clinician-based
measures of severity also correlated with plasma changes of key
biomarkers related to disease pathogenesis.
“We look forward to completing the double-blind,
placebo-controlled Phase 2 ANAVEX2-73-RS-001 study, followed by the
double-blind, placebo-controlled Phase 2 ANAVEX2-73-RS-002 (AVATAR)
study,” said Christopher U. Missling, Ph.D., Chief Executive
Officer of Anavex. “We also expect to initiate the double-blind,
placebo-controlled clinical trial, ANAVEX2-73-RS-003 (EXCELLENCE)
in pediatric patients with Rett syndrome. Anavex is excited about
our progress to date, as well as our pursuit of a much-needed
treatment option for patients with Rett syndrome."
The full paper can be accessed online at:
(https://www.sciencedirect.com/science/article/pii/S0091305719302515).
About Rett Syndrome 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: their ability to speak,
walk, eat and easily breathe. The hallmark of Rett syndrome is near
constant repetitive hand movements while awake. The disease 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, autistic features,
slowed brain and head growth, ataxia, seizures and intellectual
disability.
Rett syndrome is caused by mutations in the
MECP2 gene and strikes all racial and ethnic groups. The disease
occurs worldwide in approximately one in every 10,000 to 15,000
live births. There is currently no cure for Rett syndrome.
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 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),
recently completed a successful Phase 2a clinical trial for
Alzheimer’s disease. ANAVEX®2-73 (blarcamesine) 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 (blarcamesine) 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 (blarcamesine) for the treatment of Parkinson’s
disease. ANAVEX®3-71, which targets sigma-1 and 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 mitochondrial
dysfunction and neuroinflammation. 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
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