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 presentations of Psychometric
Evaluation of the Rett Syndrome Behaviour Questionnaire (RSBQ) and
Burden of Illness (BOI) study in Rett syndrome by the respective
working groups, at the IRSF Rett Syndrome Scientific Meeting,
taking place in Nashville, TN, April 26–27, 2022.
The presentation titled, “A Psychometric
Evaluation of the Rett Syndrome Behaviour Questionnaire (RSBQ) in
children and adults”, will be presented by Dr. Walter E Kaufmann,
MD, Chief Scientific Officer of Anavex and is co-authored by
Lindsay M. Oberman, Helen Leonard, Jenny Downs, Rina Cianfaglione,
Michelle Stahlhut, Jane L. Larsen and Katherine V. Madden.
The analyses presented support research and
clinical use of the components of the RSBQ with better psychometric
properties, in pediatric and adult populations. The authors propose
a novel factor structure with strong empirical and analytical
bases. The data highlight the need for additional evaluations and
potential improvements of the RSBQ, considering its more widespread
use.
The presentation titled, “Burden of illness in
Rett syndrome: initial evaluation of a disorder-specific caregiver
survey” will be presented by the Chief Scientific Officer of
Anavex, Dr. Walter E Kaufmann, MD and is co-authored with Alan K.
Percy, Jeffrey L. Neul, Jenny Downs, Helen Leonard, Paige Nues,
Girish D. Sharma, Theresa E. Bartolotta, Gill S. Townend, Leopold
M.G. Curfs, Orietta Mariotti, Claude Buda, Heather M. O'Leary,
Vanessa Vogel-Farley, Katherine V. Madden, Edward R. Hammond and
Christopher U. Missling.
Previous studies have shown decreased patient
and caregiver quality of life (QoL); however, applied instruments
have not adequately quantified disease burden on affected
individuals and their families in Rett syndrome. Consequently, a
global collaboration of stakeholders in the USA, Europe, and
Australia conducted the Burden of Illness (BOI) project utilizing a
caregiver questionnaire involving interviews with parents of 756
children and adults with Rett syndrome. Profiles of clinical
severity, impact on the affected individual, and impact on the
caregiver were characterized and compared.
The presented data demonstrate that core
impairments in Rett syndrome have high burden impact on affected
individuals and caregivers. Moreover, problems considered
relatively mild in severity, can place a disproportionate burden on
affected individuals and their caregivers. Ongoing analyses are
examining the role of patient characteristics and other factors on
the impact of Rett Syndrome on individuals and caregivers.
“It is important to continue to understand the
strength and weaknesses of measures of disease assessment in Rett
Syndrome and further improve their utility for clinical consistency
and meaningfulness,” commented Walter E Kaufmann, MD, Chief
Scientific Officer of Anavex. He continued, “We are confident that
this work represents an advance in understanding the disease for
both children and adults with Rett syndrome.”
Christopher U Missling, PhD, President &
Chief Executive Officer of Anavex, remarked, "We are honored to
support the Rett syndrome community with one of the largest Burden
of Illness study to date and understanding which factors of Rett
syndrome pose the greatest threat to health and wellbeing, helps
public health practitioners and policy-makers decide how to
appropriately use resources for maximum benefit. This can help to
reduce health inequities and the human and economic costs that
result from Rett syndrome."
ANAVEX®2-73 (blarcamesine) has successfully
completed both a placebo-controlled Phase 2 and a
placebo-controlled Phase 3 study in adult patients with Rett
syndrome. The placebo-controlled EXCELLENCE Phase 2/3 pediatric
Rett syndrome study (ANAVEX®2-73-RS-003)1 is currently ongoing and
is evaluating ANAVEX®2-73 for Rett syndrome patients ages 5 to
17.
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 affects 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),
has successfully completed a Phase 2a 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 muscarinic M1 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 RelationsTel: 516-662-9461Email:
andrew@barwicki.com
1 ClinicalTrials.gov Identifier: NCT04304482
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