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Proof of Concept Controlled Phase 2 Clinical Trial Data Evaluating ANAVEX 2-73 ( blarcamesine ) in Parkinson's Disease Dementia Presented at CTAD 2020 Conference Statistically significant improvements in CDR system Cogni

Key Takeaway: of Concept Controlled Phase 2 Clinical Trial Data Evaluating ANAVEX 2-73 (blarcamesine) in Parkinson's Disease Dementia Presented at CTAD 2020 Conference significant improvements in CDR system Cognitive Domain of Attention assessed by Choice Reaction Time (p = 0.039) and Digi

Full Press Release Details

of Concept Controlled Phase 2 Clinical Trial Data Evaluating
ANAVEX 2-73 (blarcamesine) in Parkinson's Disease Dementia Presented
at CTAD 2020 Conference
significant improvements in CDR system Cognitive Domain of Attention
assessed by Choice Reaction Time (p = 0.039) and Digital
Vigilance (p = 0.008)
significant dose-dependent improvements in Episodic Memory (p = 0.003)
(blarcamesine) prevented the on-going cognitive decline in treated
patients compared to placebo
breaking abstract of cognitive outcome measures relevant to Alzheimer's disease
selected for oral presentation at CTAD
YORK - November 6, 2020 - 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 (AD), Parkinson's disease (PD), Rett syndrome and other
central nervous system (CNS) diseases, today announced additional details on and presented the results from the proof of concept
Phase 2 controlled trial evaluating the safety, tolerability, and efficacy of ANAVEX 2-73 (blarcamesine) in patients
with Parkinson's disease dementia (PDD) at the 13th international conference on Clinical Trials on Alzheimer's
Disease (CTAD). CTAD is an annual conference focused on Alzheimer's research and development and takes place this year as
a virtual event on November 4-7th, 2020.
of the Late-breaking Presentation:
"ANAVEX 2-73 (blarcamesine) Currently in Phase 2b/3 Early Alzheimer's Disease (AD): Analysis of Cognitive
Outcome Measures Relevant to AD of Double-blind, Multicenter, Placebo-controlled Phase 2 Clinical Trial in 132 Patients with Parkinson's
Presentation Type: Late-Breaking, Oral Presentation (LB25)
Presenter: Dag Aarsland, MD, PhD - King's College London, UK
Date/Time: November 6, 2020, 10:45 am EST
study found that ANAVEX 2-73 (blarcamesine) was well tolerated in oral doses up to 50 mg once daily. The results showed
clinically meaningful, dose-dependent, and statistically significant improvements in the Cognitive Drug Research (CDR) computerized
assessment system analysis. The study validated the precision medicine approach of targeting SIGMAR1 as a genetic biomarker of
response to ANAVEX 2-73 (blarcamesine), confirming that ANAVEX 2-73 (blarcamesine) acts through SIGMAR1
activation. These results support continued development in PDD / PD as well as the currently ongoing Phase 2 and Phase 2/3 clinical
studies with ANAVEX 2-73 (blarcamesine) in Rett syndrome1 and Alzheimer's disease2.
ClinicalTrials.gov Identifiers: NCT03758924, NCT03941444, NCT04304482
ClinicalTrials.gov Identifiers: NCT03790709, NCT02756858
of the study results:
presentation is available on the Anavex website (www.anavex.com).
ANAVEX 2-73-PDD-001 study was an international, double-blind, multicenter, placebo-controlled proof of concept Phase 2 clinical
study that randomized 132 patients with PDD equally to target doses of 30mg, 50mg ANAVEX 2-73 (blarcamesine) or placebo,
respectively. In addition to safety and cognitive efficacy, sleep function was assessed during the study at week 8 and week 14.
study results will be submitted for publication in a peer-reviewed medical journal. Anavex is planning a pivotal trial of ANAVEX 2-73
(blarcamesine) in Parkinson's disease dementia after submitting the results of the study to the FDA to obtain regulatory
am very intrigued to see the promising results of the ANAVEX 2-73-PDD-001 trial, providing significant improvements in cognitive
function accompanied by a favorable safety and tolerability profile," said Dag Aarsland, MD, PhD, Professor and the Head
of Department of Old Age Psychiatry at the Institute of Psychiatry, Psychology & Neuroscience, King's College London,
UK. "The ANAVEX 2-73 (blarcamesine) study results represent a meaningful step forward toward urgently needed
treatment for this serious complication of Parkinson's disease given that cognitive impairment of patients with Parkinson's
disease dementia is very distressing to patients and their families and is associated with greater risk of institutionalization
and accelerated progression to severe dementia and death."
Jaime Kulisevsky, MD, PhD, Full Professor of Neurology & Vice-Dean Faculty of Medicine Autonomous University of Barcelona
and Director of the Movement Disorders Unit, Department of Neurology, Sant Pau Hospital and Principal Investigator in the trial,
commented, "PDD is a debilitating disorder with significant co-morbidities and there has not been a mechanistically novel
medication approved for PDD in over 20 years. Hence, new therapies are urgently needed to alleviate this suffering and disability.
As the first double-blind trial of ANAVEX 2-73 (blarcamesine) in PDD, this proof-of-concept study provides very encouraging
and clinically relevant data."
Mahurin, R. K., & Pirozzolo, F. J. (1993). Application of Hick's law of response speed in Alzheimer and Parkinson
diseases. Perceptual and Motor Skills, 77(1), 107-113
Wesnes K, Edgar C, Andreasen N, Annas P, Basun H, Lannfelt L, et al. Computerized cognition assessment during
acetylcholinesterase inhibitor treatment in Alzheimer's disease. Acta Neurol Scand 2010; 122:270-7
U Missling, PhD, President & Chief Executive Officer of Anavex, "Our strategy has been consistently to advance ANAVEX 2-73
(blarcamesine) with focus on Precision Medicine and to validate this approach in clinical studies in patients with significant
cognitive impairments. We are pleased with these PDD study results that will be further supplemented by actigraphy movement data
and whole genome exome DNA and RNA data. Finally, we would like to thank all the patients and participating families as well the
investigators and clinical site coordinators for their dedication to this study."
completing the trial, participants were able to enroll in a voluntary 48-week open-label extension study, ANAVEX 2-73-PDD-EP-001,
which continues to assess safety, long term efficacy and changes in gut microbiota.5
(blarcamesine) is an orally available, small-molecule activator of the sigma-1 receptor (SIGMAR1), which has been shown
to be pivotal to restoring neural cell homeostasis and promoting neuroplasticity.6
Parkinson's Disease Dementia (PDD)
disease is a fairly common neurological disorder in older adults, estimated to affect nearly 2 percent of those older than age
65. The Parkinson's Foundation estimates that 1 million Americans have Parkinson's disease. It is estimated that up
to 80 percent of those with Parkinson's disease eventually experience Parkinson's disease dementia. The brain changes
caused by Parkinson's disease begin in a region that plays a key role in movement. As Parkinson's brain changes gradually
spread, they often begin to affect mental functions, including memory and the ability to pay attention, make sound judgments and
plan the steps needed to complete a task.7
ANAVEX 2-73 (blarcamesine)
(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 (blarcamesine) has
shown dose dependent improvement in exploratory endpoints of cognition (MMSE) and activities of daily living (ADCS-ADL). Full
genomic analysis of ANAVEX 2-73 (blarcamesine) Phase 2a study in AD patients was performed. The ANAVEX 2-73 (blarcamesine)
Phase 2 PDD study design includes genomic biomarkers identified in the ANAVEX 2-73 (blarcamesine) Phase 2a AD study.
Studies of ANAVEX 2-73 (blarcamesine) in a disease modifying animal model of Parkinson's disease indicates that
ANAVEX 2-73 (blarcamesine) is well tolerated, induces significant motor recovery (p<0.05), induces neurohistological
restoration (p<0.05) and reduces microglial activation (p<0.05), a potential biomarker of Parkinson's disease. Behavioral
patterns were completely normal, meaning no signs of either dystonia or stereotypic behaviors were detected in animals receiving
the treatment. These studies were funded by The Michael J. Fox Foundation for Parkinson's Research.
ClinicalTrials.gov Identifier: NCT04575259
Advances in Experimental Medicine and Biology Volume 964 (2017) Sigma Receptors: Their Role in Disease and as Therapeutic
Anavex Life Sciences Corp.
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.
Last updated: Nov 6, 2020