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 the Independent Data Safety
Monitoring Board (DSMB) for the Company's ongoing clinical trial
program, including the late stage AVATAR (ANAVEX®2-73-RS-002)1,
EXCELLENCE (ANAVEX®2-73-RS-003)2 and the U.S. Rett syndrome
extension study (ANAVEX®2-73-RS-EP-001) of its investigational
compound ANAVEX®2-73 (blarcamesine) has completed its recent
pre-planned review of the respective interim safety data for these
three separate clinical studies.
Upon review of the interim safety data, the DSMB
made the following recommendation for the randomized, double-blind,
placebo-controlled AVATAR (ANAVEX®2-73-RS-002) study in adult
patients with Rett syndrome:
- The DSMB recommendation is to
continue the studies without modification.
Upon review of the interim safety data, the DSMB
made the following recommendation for the randomized, double-blind,
placebo-controlled EXCELLENCE (ANAVEX®2-73-RS-003) study in
pediatric patients with Rett syndrome:
- The DSMB recommendation is to
continue the studies without modification.
Upon review of the interim safety data, the DSMB
made the following recommendation for the open-label extension U.S.
Rett syndrome (ANAVEX®2-73-RS-EP-001) study in adult patients with
Rett syndrome:
- The DSMB recommendation is to
continue the studies without modification.
DSMBs are committees commonly used in clinical
trials to protect the interests of the patients and the integrity
of the study data in ongoing trials.
Based on the previously reported results of the
first placebo-controlled U.S. Phase 2 (ANAVEX®2-73-RS-001)3 study
in adult patients with Rett syndrome, Anavex is planning to meet
with the FDA to discuss the approval pathway. There are no
FDA-approved drugs for Rett syndrome. ANAVEX®2-73 has Fast Track
designation, Rare Pediatric Disease designation and Orphan Drug
designation from the FDA for the treatment of Rett syndrome and may
be considered for accelerated approval.
The respective ongoing placebo-controlled Phase
2 and Phase 2/3 ANAVEX®2-73 clinical studies in adult (AVATAR) and
pediatric (EXCELLENCE) patients with Rett syndrome are expected to
be completed by mid 2021 and 2H 2021, respectively.
ANAVEX®2-73 activates the sigma-1 receptor
(SIGMAR1). Data suggests that activation of SIGMAR1 results in the
restoration of complete housekeeping function within the body and
is pivotal to restoring neural cell homeostasis and promoting
neuroplasticity.4
PET scan data previously confirmed
dose-dependent target engagement of SIGMAR1 with ANAVEX®2-73.5
Anavex Life Sciences’ product portfolio includes
small drug molecule lead candidate ANAVEX®2-73 for the treatment of
Alzheimer’s disease, Parkinson’s disease and Rett syndrome.
About ANAVEX®2-73-RS-002 AVATAR Clinical
Study (NCT03941444)
The Phase 2 trial is a randomized double-blind,
placebo-controlled safety, tolerability, pharmacokinetic and
efficacy study of oral liquid ANAVEX®2-73 to treat Rett syndrome in
a total of 33 adult patients with Rett syndrome over a 7-weeks
treatment period incorporating precision medicine biomarkers.
Preceding the placebo-controlled randomization of 30 patients (Part
B), a 3-patient cohort (Part A) underwent a pharmacokinetic (PK)
assessment with safety, tolerability, pharmacokinetic and efficacy
evaluation of ANAVEX®2-73. All patients who completed the study are
eligible to continue to receive ANAVEX®2-73 for additional 48 weeks
within the open label extension protocol.
About ANAVEX®2-73-RS-003 EXCELLENCE
Clinical Study (NCT04304482)
The Phase 2/3 trial is a randomized
double-blind, placebo-controlled safety, tolerability,
pharmacokinetic and efficacy study of oral liquid ANAVEX®2-73 to
treat Rett syndrome in at least 69 pediatric patients with Rett
syndrome over a 12-weeks treatment period incorporating precision
medicine biomarkers. All patients who completed the study are
eligible to continue to receive ANAVEX®2-73 for additional 48 weeks
within the open label extension protocol.
About ANAVEX®2-73-RS-001 U.S. 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 to treat Rett syndrome in
a total of 31 adult patients with Rett syndrome over a 7-weeks
treatment period were evaluated incorporating precision medicine
biomarkers. Preceding the placebo-controlled randomization of 25
patients (Part B), a 6-patient cohort (Part A) underwent a 7-weeks
pharmacokinetic (PK) assessment with safety, tolerability,
pharmacokinetic and efficacy evaluation of ANAVEX®2-73. All
patients who completed in the study were eligible to continue to
receive ANAVEX®2-73 under a 36-week open label extension protocol
(ANAVEX®2-73-RS-EP-001).
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. The population of patients with Rett syndrome is estimated
to be approximately 11,000 patients in the U.S. 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),
completed successfully a Phase 2a clinical trials for Alzheimer’s
disease and a Phase 2 proof-of-concept study in Parkinson’s disease
dementia and a Phase 2 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 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 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 ClinicalTrials.gov Identifier: NCT039414442
ClinicalTrials.gov Identifier: NCT043044823 ClinicalTrials.gov
Identifier: NCT037589244 Advances in Experimental Medicine and
Biology Volume 964 (2017) Sigma Receptors: Their Role in Disease
and as Therapeutic Targets.5
https://assets.researchsquare.com/files/rs-189177/v1/65385792-095a-4505-90c4-c0b85c76dbd1.pdf.
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