Anavex Life Sciences Announces Exceeding of Enrollment Target for the Precision Medicine ANAVEX®2-73 (blarcamesine) Phase 2b/3 Clinical Trial in Patients with Alzheimer’s Disease

Rhea-AI Summary

Anavex Life Sciences Corp. (Nasdaq: AVXL) has successfully exceeded its enrollment target for the ANAVEX®2-73 Phase 2b/3 study in Alzheimer’s disease, enrolling over 450 patients across 52 sites in North America, Europe, and Australia. The study focuses on the efficacy, tolerability, and safety of two doses of ANAVEX®2-73 (blarcamesine) compared to a placebo, utilizing SIGMAR1 gene expression as a precision medicine biomarker. Topline results are anticipated by mid-2022, highlighting the significant need for new therapies in Alzheimer’s treatment.

Positive

• Exceeded enrollment target for Phase 2b/3 study with over 450 patients.
• Incorporates precision medicine biomarker (SIGMAR1 gene expression) related to clinical benefits.
• Anticipated topline results by mid-2022, potentially advancing Alzheimer's treatment options.

Negative

• None.

NEW YORK, June 08, 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 it has exceeded its enrollment target for the ANAVEX^®2-73 (blarcamesine) Phase 2b/3 study in Alzheimer’s disease. The Company expects to announce topline results from this study by mid-2022.

The study includes a prespecified precision medicine biomarker, SIGMAR1 gene expression, which demonstrated correlation with direct measures of clinical benefit, cognition and activities of daily living and function in a previous Phase 2a Alzheimer’s disease study.¹

The double-blind, placebo-controlled 450-patient Phase 2b/3 ANAVEX^®2-73 clinical study in patients with Alzheimer's disease exceeded enrollment beyond 450 patients at 52 sites across North America, Europe and Australia using ADAS-Cog (cognition) and ADCS-ADL (activities of daily living and function) as primary endpoints.²

This multi-center, double-blind clinical trial is measuring efficacy, tolerability, and safety of two different once daily oral ANAVEX^®2-73 (blarcamesine) doses or placebo.

ANAVEX^®2-73 is an orally available, small-molecule activator of 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.³

PET scan data previously confirmed dose-dependent target engagement of SIGMAR1 with ANAVEX^®2-73.⁴

“A significant unmet need exists currently for Alzheimer’s disease patients worldwide given the lack of available adequate therapeutic interventions, and we are excited to have exceeded enrollment of this study for ANAVEX^®2-73," said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. "Given ANAVEX^®2-73’s convenient oral route of administration we believe it has the potential to deliver broad clinical utility."

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).⁵ 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 currently have 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 approximately $305 billion. By 2050, these costs could rise as high as $1.1 trillion.⁶ There are currently over 50 million people living with dementia around the world, with numbers expected to increase to nearly 152 million by 2050.⁷ 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.⁸

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 trials 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 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 Hampel et al. 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 Dement. 2020;00:1–14
² ClinicalTrials.gov Identifier: NCT03790709.
³ Advances in Experimental Medicine and Biology Volume 964 (2017) Sigma Receptors: Their Role in Disease and as Therapeutic Targets.
⁴ https://assets.researchsquare.com/files/rs-189177/v1/65385792-095a-4505-90c4-c0b85c76dbd1.pdf.
⁵ 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.
⁶ https://www.nia.nih.gov/health/alzheimers; https://www.alz.org/alzheimers-dementia/facts-figures.
⁷ Alzheimer's Disease International. World Alzheimer Report 2019. https://www.alz.co.uk/research/WorldAlzheimerReport2019.pdf.
⁸ 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 ANAVEX®2-73 Phase 2b/3 study about?

The ANAVEX®2-73 Phase 2b/3 study is a clinical trial evaluating the efficacy, tolerability, and safety of ANAVEX®2-73 in Alzheimer's disease, utilizing over 450 patients and measuring outcomes through cognitive and daily living activity assessments.

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

Topline results from the ANAVEX®2-73 Phase 2b/3 study are expected to be announced by mid-2022.

How does the SIGMAR1 gene expression relate to Alzheimer's treatment?

The SIGMAR1 gene expression serves as a precision medicine biomarker that previously showed a correlation with clinical benefits in Alzheimer's patients, suggesting its potential role in predicting treatment outcomes.

How many sites are involved in the ANAVEX®2-73 study?

The ANAVEX®2-73 Phase 2b/3 study involves 52 sites across North America, Europe, and Australia.

What is the significance of the ANAVEX®2-73 study for Alzheimer's patients?

This study addresses a significant unmet need for effective Alzheimer's therapies, as currently available treatments are inadequate for many patients.