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 the publication of a relevant new
peer-reviewed study in the American Journal on Intellectual and
Developmental Disabilities, entitled ‘Rett Syndrome Behaviour
Questionnaire in Children and Adults With Rett Syndrome:
Psychometric Characterization and Revised Factor Structure’.1
The study reports distribution of scores and
other statistical features of the Rett Syndrome Behaviour
Questionnaire (RSBQ) in more than 600 children and adults with Rett
syndrome. It also re-examines the structure of the questionnaire,
specifically the grouping of questions (items) into different
subscales. This evaluation led authors to propose some changes to
the organization of the questions (i.e., revised subscales). The
study, which represents the largest ever analysis of the RSBQ and
the first report of the instrument in adults with Rett Syndrome,
was the result of an international collaboration including
anonymized databases from the United States, Australia, United
Kingdom, and Denmark. The ultimate goal of the project was to
provide reference values and other metrics of the RSBQ for its
clinical application and implementation in research studies. The
study was partially funded by a grant from the International Rett
Syndrome Foundation (IRSF). All analyses performed in the study are
available to the community through a website hosted by IRSF.
The RSBQ was developed by a British team of
clinicians and researchers to better delineate behavioral problems
in children with Rett syndrome. Over the course of the following
two decades, the use of the RSBQ has been expanded to observational
studies in adults with Rett syndrome and efficacy assessments. The
RSBQ’s implementation as global outcome measures in FDA-regulated
clinical trials is the result of its comprehensive nature since the
questionnaire includes not only behaviors but also behavior-related
symptoms. Indeed, most regulated trials include as co-primary
efficacy endpoints the RSBQ, as a caregiver measure, and the
Clinical Global Impression of Improvement (CGI-I) as an
investigator assessment. For all these reasons, this psychometric
study is timely and significant, providing additional support for
the use of the RSBQ in children and adults as well as reference
values and revised subscales for its improved use.
In the EXCELLENCE Phase 2/3 ANAVEX®2-73-RS-003
Rett syndrome pediatric clinical trial, the characterized Rett
Syndrome Behaviour Questionnaire (RBSQ), together with the Clinical
Global Impression Improvement Scale (CGI-I), represent the
co-primary efficacy endpoints of the study.
“This paper is a further demonstration of
Anavex’s involvement as part of the team of clinicians and
commitment to the rare disease and especially to the Rett syndrome
community,” said Christopher U Missling, PhD, President and Chief
Executive Officer of Anavex. "We are looking forward to the topline
data from the EXCELLENCE Phase 2/3 ANAVEX®2-73-RS-003 Rett syndrome
pediatric clinical trial in the second half of 2023.”
The paper can be accessed online at:
https://pubmed.ncbi.nlm.nih.gov/37104862/.
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.
1. Oberman LM, Leonard H, Downs J, Cianfaglione
R, Stahlhut M, Larsen JL, Madden KV, Kaufmann WE. Rett Syndrome
Behaviour Questionnaire in Children and Adults With Rett Syndrome:
Psychometric Characterization and Revised Factor Structure. Am J
Intellect Dev Disabil. 2023 May 1;128(3):237-253. doi:
10.1352/1944-7558-128.3.237.PMID: 37104862.
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 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),
has successfully completed a Phase 2a and recently 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 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 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: 516-662-9461Email: andrew@barwicki.com
1 Oberman LM, Leonard H, Downs J, Cianfaglione R, Stahlhut M,
Larsen JL, Madden KV, Kaufmann WE. Rett Syndrome Behaviour
Questionnaire in Children and Adults With Rett Syndrome:
Psychometric Characterization and Revised Factor Structure. Am J
Intellect Dev Disabil. 2023 May 1;128(3):237-253. doi:
10.1352/1944-7558-128.3.237.PMID: 37104862.
Anavex Life Sciences (NASDAQ:AVXL)
Historical Stock Chart
From Sep 2024 to Oct 2024
Anavex Life Sciences (NASDAQ:AVXL)
Historical Stock Chart
From Oct 2023 to Oct 2024