Anavex Life Sciences Reports Data Review by the Independent Data Safety Monitoring Board for its Phase 2b/3 Clinical Trial of ANAVEX®2-73 in Patients with Alzheimer’s Disease

Rhea-AI Summary

Anavex Life Sciences Corp. (AVXL) announced positive progress in its Phase 2b/3 Alzheimer’s disease study of ANAVEX®2-73 (blarcamesine). The Independent Data Safety Monitoring Board (DSMB) recommended continuing the trial without modifications after reviewing interim safety data. The study, which has fully enrolled 509 patients, is centered on assessing safety and cognitive function, using primary endpoints like ADAS-Cog. Topline data is expected in the second half of 2022, with potential implications for treating neurodegenerative disorders.

Positive

• DSMB recommended continuation of the Phase 2b/3 study without modifications.
• The study has completed full enrollment of 509 patients.
• Anticipation of topline data in the second half of 2022.

Negative

• None.

The Company anticipates topline data in the second half of 2022

NEW YORK, Oct. 13, 2021 (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 Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, today announced that the Independent Data Safety Monitoring Board (DSMB) for the Company's Phase 2b/3 Alzheimer’s disease study of its investigational compound ANAVEX^®2-73 (blarcamesine) has completed its recent pre-planned review of the preliminary Phase 2b/3 study data.

As specified in the protocol, the DSMB reviewed the interim safety data for the ANAVEX^®2-73 Phase 2b/3 Alzheimer’s disease clinical study ANAVEX^®2-73-AD-004 and its Open Label Extension (OLE) ANAVEX^®2-73-AD-EP-004 ATTENTION-AD study.

Upon review of the interim safety data, the DSMB made the following recommendation:

• The DSMB recommendation is to continue the studies without modification.
  

DSMBs are committees commonly used in clinical trials to protect the interests of the patients and the integrity of the study data in ongoing trials.

ANAVEX^®2-73 activates the sigma-1 receptor (SIGMAR1). Data suggests that activation of SIGMAR1 results in the restoration of complete housekeeping function within the body and is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.¹

PET scan data previously confirmed dose-dependent target engagement of SIGMAR1 with ANAVEX^®2-73.²

The placebo-controlled 509-patient Phase 2b/3 ANAVEX^®2-73 clinical study in patients with Alzheimer's disease achieved full enrollment and includes prespecified SIGMAR1 gene expression as endpoints using ADAS-Cog (cognition) and ADCS-ADL (activities of daily living and function) as primary endpoints.³ The Company anticipates topline data in the second half of 2022.

Anavex Life Sciences’ product portfolio includes small drug molecule lead candidate ANAVEX^®2-73 for the treatment of Alzheimer’s disease, Parkinson’s disease and Rett syndrome.

About ANAVEX^®2-73-AD-004 Clinical Study (ClinicalTrials.gov Identifier: NCT03790709) and OLE ANAVEX^®2-73-AD-EP-004 Clinical Study ATTENTION-AD (ClinicalTrials.gov Identifier: NCT04314934)

ANAVEX^®2-73-AD-004 clinical study is a Phase 2b/3 double-blind, randomized, placebo-controlled, 48-week safety and efficacy trial of ANAVEX^®2-73 for the treatment of early Alzheimer’s disease. The Phase 2b/3 study enrolled 509 patients, randomized 1:1:1 to two different ANAVEX^®2-73 doses or placebo. The ANAVEX^®2-73 Phase 2b/3 study design includes genomic precision medicine biomarkers identified in the previous ANAVEX^®2-73 Phase 2a study (ANAVEX2-73-002, NCT02244541). Primary and secondary endpoints will assess safety and both cognitive and functional efficacy, measured through ADAS-Cog, ADCS-ADL and CDR-SB. ANAVEX^®2-73 Phase 2a Alzheimer’s disease study previously demonstrated dose dependent improvement in exploratory endpoints of cognition (MMSE) and function (ADCS-ADL).⁴ ANAVEX^®2-73-AD-EP-004 is the 96-week open label extension of the placebo-controlled Phase 2b/3 ANAVEX^®2-73 study.

About Alzheimer’s Disease

Alzheimer’s disease is a progressive degenerative brain disorder that gradually destroys a person's memory and ability to learn, reason, make judgments, communicate and carry out daily activities. An estimated 5.7 million Americans have currently Alzheimer's dementia. Alzheimer’s is the most common cause of dementia among older adults and is estimated to rank as the third leading cause of death for older people in the United States, just behind heart disease and cancer. In 2020, Alzheimer's and other dementias cost the nation $305 billion. By 2050, these costs could rise as high as $1.1 trillion.⁵ There are currently over 50 million people living with dementia around the world, with numbers expected to increase to nearly 152 million by 2050.⁶ Almost 10 million new cases of dementia are diagnosed each year worldwide, implying one new case every 3 seconds, and a significant increase in the caregiving burden placed on society and families.⁷

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), successfully completed a Phase 2a clinical trial for Alzheimer’s disease and recently a Phase 2 proof-of-concept study in Parkinson’s disease dementia and a Phase 2 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 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 Development
Toll-free: 1-844-689-3939
Email: info@anavex.com

Investors:
Andrew J. Barwicki
Investor Relations
Tel: 516-662-9461
Email: andrew@barwicki.com

¹ Advances in Experimental Medicine and Biology Volume 964 (2017) Sigma Receptors: Their Role in Disease and as Therapeutic Targets.
² Reyes ST et al. Effects of the sigma-1 receptor agonist blarcamesine in a murine model of fragile X syndrome: neurobehavioral phenotypes and receptor occupancy. Sci Rep. 2021 Aug 25;11(1):17150. doi: 10.1038/s41598-021-94079-7. PMID: 34433831; PMCID: PMC8387417.
³ ClinicalTrials.gov Identifier: NCT03790709.
⁴ Hampel H. et al. (2020). A precision medicine framework using artificial intelligence for the identification and confirmation of genomic biomarkers of response to an Alzheimer's disease therapy: Analysis of the blarcamesine (ANAVEX2‐73) Phase 2a clinical study. Alzheimer's & Dementia: Translational Research & Clinical Interventions, 6(1), e12013.
⁵ https://www.nia.nih.gov/health/alzheimers; https://www.alz.org/alzheimers-dementia/facts-figures.
⁶ Alzheimer's Disease International. World Alzheimer Report 2019. https://www.alz.co.uk/research/WorldAlzheimerReport2019.pdf.
⁷ AARP. 2020 Report: Caregiving in the U.S. https://www.aarp.org/content/dam/aarp/ppi/2020/05/full-report-caregiving-in-the-united-states.doi.10.26419-2Fppi.00103.001.pdf.

FAQ

What is the latest update on Anavex Life Sciences Corp.'s Phase 2b/3 Alzheimer's study?

The Independent Data Safety Monitoring Board has recommended continuing the Phase 2b/3 study of ANAVEX®2-73 without modifications after reviewing interim safety data.

When can we expect topline data from the ANAVEX®2-73 study?

Topline data from the Phase 2b/3 study of ANAVEX®2-73 is anticipated in the second half of 2022.

How many patients are enrolled in the ANAVEX®2-73 study?

The Phase 2b/3 study has fully enrolled 509 patients.

What are the primary endpoints of the ANAVEX®2-73 study?

The primary endpoints of the study include ADAS-Cog for cognition and ADCS-ADL for activities of daily living and function.