Presentations cover the Psychometric Evaluation of the Rett Syndrome Behaviour Questionnaire (RSBQ) and the Burden of Illness (BOI) Study in Rett Syndrome
NEW YORK – April 27, 2022
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) 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.
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