Full Press Release Details
Anavex Life Sciences Announces Positive
Results from Proof of Concept controlled Phase 2 Clinical Trial Evaluating ANAVEX 2-73 (blarcamesine) in Parkinson's
Clinically meaningful, dose-dependent,
and statistically significant improvements in the Cognitive Drug Research (CDR) computerized assessment system analysis
Company to host conference call today
NEW YORK - October 15, 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 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).
The study found that ANAVEX 2-73 (blarcamesine)
was safe and well tolerated in oral doses up to 50 mg once daily. The results show clinically meaningful, dose-dependent, and statistically
significant improvements in the Cognitive Drug Research (CDR) computerized assessment system analysis. The study confirmed the
precision medicine approach of targeting SIGMAR1 as a genetic biomarker of response to ANAVEX 2-73 (blarcamesine).
The ANAVEX 2-73-PDD-001 study was an
international, double-blind, multicenter, placebo-controlled Phase 2 clinical study and 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.
ANAVEX 2-73-PDD-001 study results will
be submitted for presentation at a medical conference and 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 guidance.
Dr. Jaime Kulisevsky Bojarski, 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, "As the first
double-blind trial of ANAVEX 2-73 (blarcamesine) in PDD, this proof-of-concept study provides extremely encouraging
data. PDD can be debilitating with significant co-morbidities. New therapies are urgently needed to alleviate this suffering and
disability. There has not been a mechanistically novel medication approved for PDD in over 20 years."
"I am very pleased 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. "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. Given the limited options of adequate treatments
for Parkinson's disease dementia, and the safety concerns and modest or uncertain efficacy of currently used off-label treatments,
the ANAVEX 2-73 (blarcamesine) study results represent a meaningful step forward toward urgently needed treatment for
this serious complication of Parkinson's disease."
"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",
said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. "Our strategy to advance ANAVEX 2-73
(blarcamesine) with focus on Precision Medicine has been validated in this study of patients with significant cognitive
impairment and we are looking forward to the next clinical data readout of ANAVEX 2-73 (blarcamesine) in Rett syndrome
and Alzheimer's disease, indications where cognitive impairment is also prevalent."
After 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.[1]
ANAVEX 2-73 (blarcamesine) is
an orally available, small-molecule activator of the sigma-1 receptor, which has been shown to be pivotal to restoring neural cell
homeostasis and promoting neuroplasticity.[2]
Conference Call Information
Anavex will host a conference call and
webcast today at 8:30 AM Eastern Time to discuss the topline results of the ANAVEX 2-73-PDD-001 trial of ANAVEX 2-73 (blarcamesine)
in Parkinson's disease dementia. To participate in the live conference call, please dial 1 (866) 939-3921 (toll-free domestic)
or 1 (678) 302-3550 (international) and use the passcode 49986272. The webcast can be accessed at https://wsw.com/webcast/cc/avxl15/1496358
and on the Company's website at www.anavex.com. A replay of the webcast will be available for approximately 30 days following
About Parkinson's Disease Dementia (PDD)
Parkinson's 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.[3]
About ANAVEX 2-73 (blarcamesine)
ANAVEX 2-73 (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 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 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.
1 ClinicalTrials.gov Identifier: NCT04575259
2 Advances in Experimental Medicine and Biology Volume
964 (2017) Sigma Receptors: Their Role in Disease and as Therapeutic Targets.
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), 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.
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