Anavex Life Sciences Announces Exceeding of Enrollment Target for the ANAVEX®2-73 (blarcamesine) U.S. Phase 2 Rett Syndrome ...
16 June 2020 - 9:00PM
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 that it has surpassed by 50% the
company’s original enrollment target for the ANAVEX®2-73
(blarcamesine) U.S. Phase 2 study in Rett syndrome. The Company
expects to announce topline results from this study in 2H 2020.
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 multi-center, double-blind clinical trial at
8 sites across U.S. is measuring safety, tolerability, and efficacy
of daily oral ANAVEX®2-73 (blarcamesine) doses or placebo. After
completing the trial, eligible participants are able to join a
voluntary open-label 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)
demonstrated significant improvements of 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 the RSBQ Breathing Abnormalities. 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.
“Since there is currently no approved agent to
treat this devastating disease, there is a significant unmet
medical need for patients with Rett syndrome,” said Christopher U.
Missling, PhD, President and Chief Executive Officer of Anavex. "We
are excited to complete enrollment in this carefully conducted,
multicenter study with ANAVEX®2-73 (blarcamesine) in Rett syndrome
and look forward to reporting the results later this year."
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
(blarcamesine)
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 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 ANAXEX®2-73
(blarcamesine) led to significant improvements in multiple motor,
sensory, and autonomic phenotypes of relevance to Rett syndrome,
including motor coordination and balance, acoustic and visual
responses, hindlimb clasping (resembling the characteristic hand
stereotypies), 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: NCT037589243
ClinicalTrials.gov Identifier: NCT039414444 ClinicalTrials.gov
Identifier: NCT04304482
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