Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq:
AVXL), a clinical-stage biopharmaceutical company developing
differentiated therapeutics for the treatment of neurodegenerative,
neurodevelopmental, and neuropsychiatric disorders including
Alzheimer’s disease, Parkinson’s disease, Rett syndrome,
schizophrenia, and other central nervous system (CNS) diseases,
today presented new data from the Phase IIb/III study showing that
blarcamesine (ANAVEX®2-73), once daily orally, demonstrates
pre-specified clinical efficacy through upstream SIGMAR1
activation.
Clinical data confirmed the mechanism of action
(MoA) by pre-specified SIGMAR1 gene analysis in people with early
Alzheimer's disease (AD). The data were presented by Marwan Noel
Sabbagh, MD, Professor of Neurology at Barrow Neurological
Institute and Chairman of the Anavex Scientific Advisory Board at
the Clinical Trials on Alzheimer’s Disease (CTAD) conference, which
is taking place October 29 - November 1, 2024, in Madrid,
Spain.
SIGMAR1 is an integral membrane protein which
activates an upstream compensatory process: Blarcamesine induces
autophagy through SIGMAR1 activation resulting in restoring
cellular homeostasis. In Alzheimer's disease patients, mutations
(variants) of genes have generally been identified as disease risk
factors. Likewise, impaired SIGMAR1 function (gene mutation,
variants) leads to potential suboptimal function. Hence, patients
who carry the non-mutated, common SIGMAR1 wild type (WT)1 gene, are
expected to have stronger beneficial response to blarcamesine than
patients with a SIGMAR1 mutation (variant), who nevertheless also
benefited from treatment.2
This was confirmed in the Phase IIb/III study
analysis: Over 48 weeks, blarcamesine significantly slowed clinical
progression by 36.3% in the primary endpoint ADAS-Cog13 [LS mean
ADAS-Cog13 difference of -2.027; P=0.008] in the ITT analysis. This
signal was even stronger in the pre-specified common SIGMAR1 wild
type (WT) group with slowed clinical progression by 49.8% at 48
weeks in the active group vs. placebo, respectively [LS mean
ADAS-Cog13 difference of -2.317; P=0.015]. Equal analysis with
CDR-SB led to comparable consistent results.
“These data are very exciting, particularly
featuring blarcamesine’s novel upstream mechanism of action,
enhancing autophagy through SIGMAR1 activation, a key clearance
mechanism that removes protein aggregates and misfolded proteins
across the Alzheimer's disease continuum,” said Juan Carlos
Lopez-Talavera, MD, PhD, Head of Research and Development of
Anavex. “The advantage of blarcamesine is that it is a small oral
molecule that exerts clinical benefits on cognition and
neurodegeneration and could be appealing because of its route of
administration and good comparative safety profile. We are on track
for regulatory submission of blarcamesine in Europe (EMA) in the
current quarter 2024.”
Overall, blarcamesine, a small molecule
administered orally once daily, demonstrated clinically meaningful
improvement over 48 weeks with primary endpoint ADAS-Cog13 score
being larger than 2 points.3 This suggests superior numerical
clinical efficacy compared to approved therapies while also slowing
neurodegeneration in early AD patients. Blarcamesine’s safety
profile indicates not requiring routine MRI monitoring, and given
its differentiated mechanism of action, could represent a novel
treatment that could be complementary or an alternative to
anti-beta amyloid monoclonal antibody drugs.
“Alzheimer’s disease is such a devastating
disease that affects tens of millions worldwide. We believe, the
clinically meaningful study results provide the potential for
patients and their families to have a better and longer quality of
life,” said Christopher U. Missling, PhD, President and Chief
Executive Officer of Anavex. “We believe the scalable and
convenient features of blarcamesine could reduce crucial barriers
within the currently complex healthcare ecosystem for Alzheimer's
disease and provide broader access to a diverse population with
early Alzheimer's disease.”
The presentation is available on the Investors
section of the Company’s website at www.anavex.com.
This release discusses investigational uses of
an agent in development and is not intended to convey conclusions
about efficacy or safety. There is no guarantee that any
investigational uses of such product will successfully complete
clinical development or gain health authority approval.
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a
publicly traded biopharmaceutical company dedicated to the
development of novel therapeutics for the treatment of
neurodegenerative, neurodevelopmental, and neuropsychiatric
disorders, including Alzheimer's disease, Parkinson's disease,
schizophrenia, Rett syndrome, and other central nervous system
(CNS) diseases, pain, and various types of cancer. Anavex's lead
drug candidate, ANAVEX®2-73 (blarcamesine), has successfully
completed a Phase 2a and a Phase 2b/3 clinical trial for
Alzheimer's disease, a Phase 2 proof-of-concept study in
Parkinson's disease dementia, and both a Phase 2 and a Phase 3
study in adult patients and one Phase 2/3 study in pediatric
patients with Rett syndrome. ANAVEX®2-73 is an orally available
drug candidate designed to restore cellular homeostasis by
targeting SIGMAR1 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. We believe that ANAVEX®3-71,
which targets SIGMAR1 and M1 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, Instagram, 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 Relations Tel: 516-662-9461 Email:
andrew@barwicki.com
1 WT = homozygous dominant (TT)2 Hampel H, Williams C, Etcheto
A, et al. A precision medicine framework using artificial
intelligence for the identification and confirmation of genomic
biomarkers of response to an Alzheimer's disease therapy: Analysis
of the blarcamesine (ANAVEX2-73) Phase 2a clinical
study. Alzheimers Dement (N Y). 2020; 6(1):e12013.3 Muir RT,
Hill MD, Black SE, Smith EE. Minimal clinically important
difference in Alzheimer's disease: Rapid review. Alzheimers
Dement. 2024;20(5):3352-3363. doi:10.1002/alz.13770
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