Anavex Life Sciences Announces First Patient Dosed in EXCELLENCE Phase 2/3 Clinical Trial of ANAVEX®2-73 (Blarcamesine) in P...
July 01 2020 - 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 diseases including Alzheimer’s disease,
Parkinson’s disease, Rett syndrome and other central nervous system
(CNS) diseases, today announced that the first patient was dosed in
the EXCELLENCE Phase 2/3 ANAVEX®2-73-RS-003 clinical trial for the
treatment of Rett syndrome with ANAVEX®2-73 (blarcamesine).
The Phase 2/3 study will use a convenient
once-daily oral liquid ANAVEX®2-73 (blarcamesine) formulation to
treat Rett syndrome. Safety and efficacy will be investigated in at
least 69 patients over a 12-week treatment period including
ANAVEX®2-73-specific precision medicine biomarkers. All patients
who participate in the study will be eligible to receive
ANAVEX®2-73 (blarcamesine) under a voluntary open label extension
protocol.
This represents the 3rd ongoing clinical study
in Anavex’s Rett Syndrome Program: U.S. RTT (ANAVEX®2-73-RS-001)1,
AVATAR (ANAVEX®2-73-RS-002)2 and EXCELLENCE
(ANAVEX®2-73-RS-003)3.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.
For parents who wish to learn more about
the EXCELLENCE pediatric Phase 2/3 Rett syndrome clinical study,
please visit the website,
www.rettsyndrometrial.com.
Rett Syndrome Association of Australia (RSAA)
will make the necessary bookings and pay for them directly so as
not to burden and leave participants and their families out of
pocket for major expenses whilst directly participating in the
EXCELLENCE trial. Please contact Claude Buda, RSAA President,
directly by phone or email for any inquiries:
Claude Buda, RSAA PresidentPhone: +61
(0)411 089 156Email: claudebuda@rettaustralia.org.au
“We are delighted to expand the ANAVEX®2-73
(blarcamesine) treatment program for Rett syndrome to include
pediatric patients. This is the 3rd ongoing clinical study within
the Anavex Rett syndrome program,” said Christopher U Missling,
PhD, President and Chief Executive Officer of Anavex. “In addition
to Rett syndrome, Anavex has ongoing clinical development programs
for ANAVEX®2-73 (blarcamesine) for the treatment of Alzheimer’s
disease4 and Parkinson’s disease dementia5.”
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.
___________________________1 ClinicalTrials.gov
Identifier: NCT037589242 ClinicalTrials.gov Identifier:
NCT039414443 ClinicalTrials.gov Identifier: NCT043044824
ClinicalTrials.gov Identifier: NCT037907095 ClinicalTrials.gov
Identifier: NCT03774459
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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