GlobeNewswire Inc | Oct 15, 2020 07:01AM EDT

October 15, 2020

Clinically meaningful, dose-dependent, and statistically significant improvements in theCognitive Drug Research(CDR) computerized assessmentsystem analysis

Company to host conference call today at8:30 AM ET

NEW YORK, Oct. 15, 2020 (GLOBE NEWSWIRE) -- 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 Alzheimers disease (AD), Parkinsons 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 ANAVEX2-73 (blarcamesine) in patients with Parkinsons disease dementia (PDD).

The study found that ANAVEX2-73 (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 ANAVEX2-73 (blarcamesine).

The ANAVEX2-73-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 ANAVEX2-73 (blarcamesine) or placebo, respectively. In addition to safety and cognitive efficacy, sleep function was assessed during the study at week 8 and week 14.

ANAVEX2-73-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 ANAVEX2-73 (blarcamesine) in Parkinsons 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 ANAVEX2-73 (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 ANAVEX2-73-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, Kings College London, UK. Cognitive impairment of patients with Parkinsons 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 Parkinsons disease dementia, and the safety concerns and modest or uncertain efficacy of currently used off-label treatments, the ANAVEX2-73 (blarcamesine) study results represent a meaningful step forward toward urgently needed treatment for this serious complication of Parkinsons 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 stud, said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. "Our strategy to advance ANAVEX2-73 (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 ANAVEX2-73 (blarcamesine) in Rett syndrome and Alzheimers 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, ANAVEX2-73-PDD-EP-001, which continues to assess safety, long term efficacy and changes in gut microbiota.1

ANAVEX2-73 (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 ANAVEX2-73-PDD-001 trial of ANAVEX2-73 (blarcamesine) in Parkinsons 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 Companys website at www.anavex.com. A replay of the webcast will be available for approximately 30 days following the live event.

About Parkinsons Disease Dementia (PDD)

Parkinsons disease is a fairly common neurological disorder in older adults, estimated to affect nearly 2 percent of those older than age 65. The Parkinsons Foundation estimates that 1 million Americans have Parkinsons disease. It is estimated that up to 80 percent of those with Parkinsons disease eventually experience Parkinsons disease dementia. The brain changes caused by Parkinsons disease begin in a region that plays a key role in movement. As Parkinsons 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 ANAVEX2-73(blarcamesine)

ANAVEX2-73 (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 Alzheimers disease (AD) study, ANAVEX2-73 (blarcamesine) has shown dose dependent improvement in exploratory endpoints of cognition (MMSE) and activities of daily living (ADCS-ADL). Full genomic analysis of ANAVEX2-73 (blarcamesine) Phase 2a AD patients was performed. The ANAVEX2-73 (blarcamesine) Phase 2 PDD study design includes genomic biomarkers identified in the ANAVEX2-73 (blarcamesine) Phase 2a AD study. Studies of ANAVEX2-73 (blarcamesine) in a disease modifying model of Parkinsons disease indicates that ANAVEX2-73 (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 Parkinsons 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 Parkinsons Research.

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 Alzheimers disease, Parkinsons disease, Rett syndrome and other central nervous system (CNS) diseases, pain and various types of cancer. Anavexs lead drug candidate, ANAVEX2-73 (blarcamesine), recently completed a successful Phase 2a clinical trial for Alzheimers disease. ANAVEX2-73 (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 Alzheimers disease. ANAVEX2-73 (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 Parkinsons Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX2-73 (blarcamesine) for the treatment of Parkinsons disease. ANAVEX3-71, which targets sigma-1 and muscarinic receptors, is a promising preclinical drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimers disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies. In preclinical trials, ANAVEX3-71 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 Companys 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 & Media:Email: ir@anavex.com

1 ClinicalTrials.gov Identifier: NCT045752592 Advances in Experimental Medicine and Biology Volume 964 (2017) Sigma Receptors: Their Role in Disease and as Therapeutic Targets.3 Source: www.alz.org/dementia/parkinsons-disease-symptoms