Anavex Life Sciences Reports Recent Data Review by the Independent Data Safety Monitoring Board for its U.S. Phase 2 Clinical...
July 31 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 the Independent Data Safety
Monitoring Board (DSMB) for the Company's U.S. Phase 2 Rett
syndrome study of its investigational compound ANAVEX®2-73
(blarcamesine) has completed its recent pre-planned review of the
preliminary Phase 2 study data.
The DSMB reviewed the preliminary efficacy and
safety data for the ANAVEX®2-73 Phase 2 Rett syndrome clinical
study ANAVEX®2-73-RS-001.
Upon review of the most recent data, the DSMB
made the following recommendation:
- The DSMB recommendation is to continue the study 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.
ANAVEX®2-73 has already received orphan drug
designation from the FDA as well as a positive opinion for orphan
designation from the European Medicines Agency (EMA) for the
treatment of Rett syndrome.
About ANAVEX®2-73-RS-001 Clinical Study
(ClinicalTrials.gov Identifier: 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.
Pharmacokinetic and dose-finding elements in a total of 15 patients
over a 7-week treatment period will be evaluated incorporating
ANAVEX®2-73-specific genomic precision medicine and other
biomarkers. Preceding the placebo-controlled randomization of 15
patients, a 6 patient cohort underwent a 7-week pharmacokinetic
(PK) assessment with safety, tolerability, pharmacokinetic and
efficacy evaluation of ANAVEX®2-73. All patients who participate in
the study will be eligible to receive ANAVEX®2-73 under an open
label extension protocol.
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
ANAVEX®2-73 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 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 Phase 2a AD patients was performed. The ANAVEX®2-73
Phase 2 Rett syndrome study design includes genomic biomarkers
identified in the ANAVEX®2-73 Phase 2a AD study. Studies of
ANAVEX®2-73 in a mouse model with a heterozygous Mecp2-null
mutation (HET) that causes neurological symptoms that mimic Rett
syndrome, ANAXEX®2-73 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 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 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 diseases 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, recently
completed a successful Phase 2a clinical trial for Alzheimer’s
disease. 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 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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