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 the completion of the ANAVEX®2-73
(blarcamesine) U.S. Phase 2 study in Rett syndrome. The Company
expects to announce topline results from this study in the next
quarter.
ANAVEX®2-73 (blarcamesine) is an orally
available, small-molecule activator of the sigma-1 receptor which,
data suggest, is pivotal to restoring neural cell homeostasis and
promoting neuroplasticity.1
The ANAVEX®2-73 (blarcamesine) U.S. Phase 2
study enrolled 31 patients with Rett syndrome and all patients
completed the 7-week trial.The multi-center, double-blind clinical
study at 8 sites across U.S. measured safety, tolerability, and
efficacy of daily oral ANAVEX®2-73 (blarcamesine) doses or placebo.
After completing the trial, eligible participants were able to join
a voluntary open-label 12-week extension study of ANAVEX®2-73
(blarcamesine).
Anavex previously reported interim data of the
PART A intensive PK sub-cohort in the first 6 patients of the U.S.
Phase 2 Rett syndrome study in which ANAVEX®2-73 (blarcamesine)
that demonstrated significant improvements in the two global
efficacy endpoints, the Rett Syndrome Behaviour Questionnaire
(RSBQ) Total score and the Clinical Global Impression – Improvement
(CGI-I). ANAVEX®2-73 (blarcamesine) also significantly improved the
RSBQ Hand Behaviours and RSBQ Breathing Abnormalities subscales. In
addition, efficacy signals on both caregiver- and clinician-based
measures of severity correlated with levels of glutamate, a key
biomarker of disease pathogenesis.
This study is one of three ongoing clinical
studies in Anavex’s Rett Syndrome Program: U.S. RTT
(ANAVEX®2-73-RS-001)2, AVATAR (ANAVEX®2-73-RS-002)3 and EXCELLENCE
(ANAVEX®2-73-RS-003)4. 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 first US-based clinical study
completed with ANAVEX®2-73 (blarcamesine),” said Christopher U.
Missling, PhD, President and Chief Executive Officer of Anavex.
"Since there is currently no approved agent to treat this
devastating disease, we continue our fast-paced development
programs 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
predominantly 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 relatively 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
non-pharmacological therapies, and drugs for seizure control, motor
difficulties, breathing irregularities, and abnormal behaviors.
Rett syndrome is mainly 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 (blarcamesine)
ANAVEX®2-73 (blarcamesine) activates the Sigma-1
receptor (S1R) protein, which serves as a molecular chaperone and
functional modulator involved in restoring cellular homeostasis. In
a Phase 2a Alzheimer’s disease (AD) study, ANAVEX®2-73 showed dose
dependent improvement in exploratory endpoints of cognition (MMSE)
and activities of daily living (ADCS-ADL). The ANAVEX®2-73 Phase 2
Rett syndrome study design includes genomic biomarkers of response
identified in the ANAVEX®2-73 Phase 2a AD study. Studies of
ANAVEX®2-73 (blarcamesine) in a mouse model of Rett syndrome,
carrying a heterozygous Mecp2-null mutation and presenting with
neurological impairments that mimic the human disorder,
demonstrated improvements at both young ages (~6 weeks) and
adulthood (7 months). Chronic oral administration of ANAVEX®2-73
(blarcamesine) led to significant improvements in multiple motor,
sensory, and autonomic phenotypes of relevance to Rett syndrome,
including motor coordination and balance, auditory and visual
responses, hindlimb clasping (resembling the characteristic
repetitive hand movements), and episodes of apnea. Notably, these
changes were dose related and also evident in animals at an
advanced stage of pathology. 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 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
1 Advances in Experimental Medicine and Biology Volume 964
(2017) Sigma Receptors: Their Role in Disease and as Therapeutic
Targets.
2 ClinicalTrials.gov Identifier: NCT03758924
3 ClinicalTrials.gov Identifier: NCT03941444
4 ClinicalTrials.gov Identifier: NCT04304482
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