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. (Nasdaq: AVXL) announced that the Independent Data Safety Monitoring Board (DSMB) has reviewed the preliminary data from its Phase 2b/3 Alzheimer’s study of ANAVEX®2-73 (blarcamesine). The DSMB recommended continuing the study without modifications. The ongoing clinical trial is over 92% enrolled, focusing on safety and efficacy, with endpoints assessing cognition and daily function. ANAVEX®2-73 targets the sigma-1 receptor and may restore neural homeostasis, signaling potential in neurodegenerative treatments.

Positive

• DSMB recommended continuing Phase 2b/3 study without modification.
• Over 92% enrollment in the Phase 2b/3 Alzheimer's study.
• Interim data suggests ANAVEX®2-73 effectively targets sigma-1 receptor.

Negative

• None.

NEW YORK, March 31, 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 ongoing placebo-controlled 450-patient Phase 2b/3 ANAVEX^®2-73 clinical study in patients with Alzheimer's disease is presently over 92% enrolled 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

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 is expected to enroll approximately 450 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), recently completed successfully a Phase 2a clinical trials for Alzheimer’s disease and 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 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

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_{¹ Advances in Experimental Medicine and Biology Volume 964 (2017) Sigma Receptors: Their Role in Disease and as Therapeutic Targets.
² https://assets.researchsquare.com/files/rs-189177/v1/65385792-095a-4505-90c4-c0b85c76dbd1.pdf.
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 current status of the ANAVEX®2-73 Phase 2b/3 clinical study?

The study is over 92% enrolled, with the DSMB recommending continuation without modifications.

What does the DSMB's recommendation mean for Anavex shareholders?

The recommendation to continue the study without changes may indicate confidence in the safety and efficacy of ANAVEX®2-73, positively affecting investor sentiment.

What is ANAVEX®2-73 and its significance in Alzheimer's treatment?

ANAVEX®2-73 is an investigational drug targeting the sigma-1 receptor, aimed at treating Alzheimer’s disease and potentially restoring neural functions.

When was the interim safety data for ANAVEX®2-73 reviewed?

The interim safety data was reviewed by the DSMB as part of the ongoing Phase 2b/3 study.

What are the key endpoints being evaluated in the ANAVEX®2-73 trial?

The trial evaluates cognition and daily living activities using ADAS-Cog and ADCS-ADL scales.