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 presented preliminary clinical efficacy data
from the PK cohort (Part A) of the U.S. Phase 2 Rett syndrome
clinical trial ANAVEX®2-73-RS-001.
“The data from the PK cohort (Part A, n=6) is
very encouraging because of the (1) magnitude of change (large to
very large effect sizes: Cohen’s d: RSBQ Total 1.47; Cohen’s d:
Glutamate 1.11), (2) the efficacy signal on both caregiver- and
clinician-based measures of severity and correlations with key
biomarker related to disease pathogenesis (Glutamate), (3) the
short duration of treatment with ANAVEX®2-73 (blarcamesine), (4)
the relatively low dose, (5) the older age of participants, and (6)
the clinical significance (RSBQ-CGI-I correlations),” said Walter E
Kaufmann, MD, Principal Investigator of the study and Chief Medical
Officer of Anavex. “The randomized, double-blind,
placebo-controlled study (Part B, n=15) is currently ongoing.”
This is one of three clinical studies in
Anavex’s Rett Syndrome Program: U.S. RTT (ANAVEX®2-73-RS-001),
AVATAR (ANAVEX®2-73-RS-002) and EXCELLENCE
(ANAVEX®2-73-RS-003).
Principal Investigator of the study and Chief
Medical Officer of Anavex, Walter E Kaufmann, MD presented the
data. The presentation will be available at www.anavex.com.
PK cohort (Part A) ranging in age from 18 to 36
years, who completed the pharmacokinetic (PK) part of the study
received a low dose of approx. 5 mg daily oral liquid dose of
ANAVEX®2-73 (blarcamesine) for 7 weeks. Efficacy evaluations took
place at Baseline (Week 0), Week 4 and Week 7.
In addition to the significant improvements of
the two global efficacy endpoints, the Rett Syndrome Behaviour
Questionnaire (RSBQ) Total score and the Clinical Global Impression
– Improvement (CGI-I), the RSBQ Hand Behaviours and the RSBQ
Breathing Abnormalities improved during the trial. Efficacy signals
on both caregiver- & clinician-based measures of severity
correlated with a key biomarker related to disease pathogenesis
(Glutamate). The biomarker correlation was also significant with
the improvement in CSHQ (Children's Sleep Habits
Questionnaire).1
Christopher U Missling, PhD, President and Chief
Executive Officer of Anavex stated, “We are intrigued by the
biomarker correlation with the efficacy measures for ANAVEX®2-73
(blarcamesine) in patients with Rett syndrome and we are currently
proceeding with Part B of the study. In addition to Rett syndrome2,
Anavex has ongoing clinical development programs for ANAVEX®2-73
(blarcamesine) for the treatment of Alzheimer’s disease3 and
Parkinson’s disease dementia4.”
1 Excitatory-inhibitory imbalances postulated in many neurologic
disorders, including Rett syndrome, have been linked to imbalances
between Glutamate and GABA: Kaufmann et al. Expert Opin Orphan
Drugs 4:1043-1055, 2016; Banerjee et al. Brain 142:239-248, 2019.2
ClinicalTrials.gov Identifier: NCT03758924; NCT039414443
ClinicalTrials.gov Identifier: NCT037907094 ClinicalTrials.gov
Identifier: NCT03774459
About Rett Syndrome
Rett syndrome is a devastating, 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, autistic features,
slowed brain and head growth, ataxia, seizures and intellectual
disability. There is currently no cure for Rett syndrome. 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-RS-001 Clinical Study
(NCT03758924)
The Phase 2 trial is a randomized double-blind,
placebo-controlled safety, tolerability, pharmacokinetic and
efficacy study of oral liquid ANAVEX®2-73 (blarcamesine) to treat
Rett syndrome. Pharmacokinetic and dose-finding elements in a total
of 21 patients over a 7-week treatment period will be evaluated
incorporating precision medicine biomarkers. Preceding the
placebo-controlled randomization of 15 patients (Part B), a 6
patient cohort (Part A) underwent a 7-week pharmacokinetic (PK)
assessment with safety, tolerability, pharmacokinetic and efficacy
evaluation of ANAVEX®2-73 (blarcamesine). All patients who
participate in the study will be eligible to receive ANAVEX®2-73
(blarcamesine) under an open label extension protocol.
About ANAVEX®2-73
ANAVEX®2-73 (blarcamesine) 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 (blarcamesine) 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 (blarcamesine) Phase 2a AD patients
was performed. The ANAVEX®2-73 (blarcamesine) Phase 2 Rett syndrome
study design includes genomic biomarkers identified in the
ANAVEX®2-73 (blarcamesine) Phase 2a AD study. Studies of
ANAVEX®2-73 (blarcamesine) in a mouse model with a heterozygous
Mecp2-null mutation (HET) that causes neurological symptoms that
mimic Rett syndrome, ANAXEX®2-73 (blarcamesine) was evaluated in
automatic visual responses and breathing tests in 7-month old mice,
an age at which advanced pathology is evident. Vehicle-treated HET
mice demonstrated fewer automatic visual responses and more
frequent expiratory apneas than wild-type mice. Treatment with
ANAVEX®2-73 (blarcamesine) for four weeks significantly increased
these visual responses in the HET mice (p<0.05). Additionally,
chronic oral dosing daily for 3-6.5 weeks of ANAVEX®2-73
(blarcamesine) starting at ~5 weeks of age was also conducted in
the HET mouse model of Rett syndrome, and dose-dependent
improvements in a variety of sensory and motor deficits, including
those involving motor coordination, balance, and learning, were
also observed. Notably, one of the strongest effects was on
hindlimb clasping, a postural response that resembles the
characteristic hand stereotypies present in Rett syndrome. 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 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
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