Anavex Life Sciences Announces Oral Blarcamesine Cognitive Resilience Results Approximating Normal Aging in New Precision Medicine Clinical Data from Phase IIb/III Alzheimer’s Disease Trial

New clinical Precision Medicine population 48-week data demonstrates unprecedented cognitive stabilization in early Alzheimer’s disease

Cognitive outcomes observed in the oral blarcamesine 30 mg Precision Medicine cohort move toward normal aging profiles across validated clinical scales, supporting its relevance in early-stage Alzheimer’s care

84.7% reduction in decline at 48 weeks of blarcamesine treatment vs placebo on the primary cognitive endpoint ADAS-Cog13

Blarcamesine could represent a novel treatment option for up to ~70% of early Alzheimer’s patients benefiting from further improved outcomes using directed Precision Medicine to alleviate significant medical and economic burden

NEW YORK, Sept. 09, 2025 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company focused on developing innovative treatments for Alzheimer's disease, Parkinson's disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome, and other central nervous system (CNS) disorders, announced today the latest findings for blarcamesine, an oral small molecule for the potential treatment of early Alzheimer’s disease (AD).

On all standard scales for measuring Alzheimer’s disease and cognitive decline, after 48 weeks, the defined Precision Medicine population ABCLEAR3¹ consisting of early AD patients with confirmed and progressed pathology taking 30 mg once-daily oral blarcamesine demonstrated barely detectable decline. This was comparable to minimally perceptible decline in prodromal (pre-dementia) aging adults.

• For ADAS-Cog13 (the standardized neuropsychological test used in Alzheimer's disease research to measure cognitive function and track disease progression), blarcamesine showed a 48-week change from baseline of 0.853 compared to ~1 point typical annual decline in prodromal (pre-dementia) aging adults.
• For CDR-SB (Clinical Dementia Rating, standard test that evaluates the severity of cognitive impairment and functional decline in individuals with AD), blarcamesine demonstrated a change from baseline of 0.465, aligning with the 0-0.5 point annual range seen in prodromal aging.

These data are similar to referenced barely detectable prodromal AD decline, in spite of the more advanced stage of AD impairment at baseline of the blarcamesine population.^2,3

Oral blarcamesine treatment over 48 weeks in a Precision Medicine population, including up to ~70% of the global population, indicate the ability to shift the cognitive decline of a MCI or mild AD patient population to that of a prodromal AD population.

In comparison, the respective placebo group in the ANAVEX2-73-AD-004 Phase IIb/III ABCLEAR3⁴ population, ADAS-Cog13 LS mean declined by 5.592 points resulting in an ADAS-Cog13 LS mean difference of -4.739 [95% CI -7.370, -2.108]; P=0.0004. This represents a 84.7% reduction in decline at 48 weeks of blarcamesine treatment vs placebo on the cognitive endpoint ADAS-Cog13.

"Given the strong interest in living a longer life without Alzheimer’s dementia, novel therapeutic directions are required. Blarcamesine’s mechanism of autophagy restoration via SIGMAR1 activation addresses a non-amyloid, upstream target, representing such a highly transformative clinical innovation,” said Marwan Noel Sabbagh, MD, Professor of Neurology, and Chairman of the Anavex Scientific Advisory Board. “We are thrilled that today's findings show the superior effect of blarcamesine Precision Medicine population shifting the previous cognitive decline of Alzheimer’s disease to barely detectable decline resembling prodromal older cognitive decline.”

“Today’s data exemplify how precision medicine is transforming Alzheimer’s care – oral blarcamesine enables a shift from fit-for-all approaches to individualized, tailored therapy. We are finally moving toward measurable individualized benefit with a high confidence of the meaningful clinical effectiveness of blarcamesine at 30 mg dose level and a favorable safety profile,” commented Professor of Neurology and Doctor in Neurosciences at the Memory Resources Research Center, the European Neurodegenerative Excellence Center of Montpellier University, Audrey Gabelle, MD, PhD and Member of the Anavex Scientific Advisory Board. “Ensuring equitable access to effective Alzheimer’s treatments is not just a scientific goal, but a societal responsibility. Blarcamesine combines scientific precision with a bold commitment to equity by offering easy-to-implement, safe, scalable and equitable access of care for people living with Alzheimer’s.”

The mechanistic confirmation that blarcamesine restores impaired autophagy through SIGMAR1 activation by acting upstream of amyloid and tau pathologies at the molecular level was previously established both in vitro and in vivo. Specifically, studies demonstrated enhanced autophagic flux in human cells and in C. elegans as well as increased proteostasis capacity, ultimately ameliorating paralysis caused by protein aggregation in C. elegans.^5,6

A clinical Precision Medicine approach confirmed that the prespecified SIGMAR1 non-mutated population, termed ABCLEAR1⁷, which represents up to ~70% of the global population, achieved deeper clinical responses to blarcamesine than the respective ITT population, hence also confirming clinical activation of SIGMAR1 through blarcamesine. Additional non-mutated populations with potentially enhanced responses could also be identified through Genome-Wide Association Study (GWAS) analyses. Data confirmed that within a heterogeneous Alzheimer’s disease population by targeting a prevalent genetic profile through Precision Medicine approach, the efficacy of blarcamesine may be further improved.

“Our company’s goal is to provide potential high-quality options for patients with Alzheimer’s disease. Today’s new clinical efficacy data update from our Phase IIb/III trial is critical, as it adds contemporary context to Precision Medicine data showing strong protection from Alzheimer’s disease with an oral once daily potential solution with the aim to alleviate significant medical and economic burden,” commented Juan Carlos Lopez-Talavera, MD, PhD, Head of Research and Development of Anavex. “We’re excited by the meaningful improvements seen with blarcamesine, confirmed by results of patients identified and confirmed through Precision Medicine.”

“We believe these data reinforce the opportunity to potentially transform the treatment paradigm for individuals living with Alzheimer’s disease. In this Precision Medicine population blarcamesine’s once-daily oral administration may support broader implementation across diverse care settings, offering a patient-friendly and scalable option for early-stage Alzheimer’s disease,” said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. “Blarcamesine’s combination of efficacy and safety may streamline treatment pathways in the complex Alzheimer’s ecosystem, paving the way for broader adoption and equitable access among patients.”

Anavex will continue to evaluate the Phase IIb/III early Alzheimer’s disease and ATTENTION-AD trial data, which will be published and presented at international AD conferences.

This release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that any investigational uses of such product will successfully complete clinical development or gain health authority approval.

About Anavex Life Sciences Corp.

Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of novel therapeutics for the treatment of neurodegenerative, neurodevelopmental, and neuropsychiatric disorders, including Alzheimer's disease, Parkinson's disease, schizophrenia, Rett syndrome, and other central nervous system (CNS) diseases, pain, and various types of cancer. Anavex's lead drug candidate, ANAVEX®2-73 (blarcamesine), has successfully completed a Phase 2a and a Phase 2b/3 clinical trial for Alzheimer's disease, a Phase 2 proof-of-concept study in Parkinson's disease dementia, and both a Phase 2 and a Phase 3 study in adult patients and one Phase 2/3 study in pediatric patients with Rett syndrome. ANAVEX®2-73 is an orally available drug candidate designed to restore cellular homeostasis by targeting SIGMAR1 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. We believe that ANAVEX®3-71, which targets SIGMAR1 and M1 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

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_{¹ ABCLEAR3 = Alzheimer’s Blarcamesine Cognition Efficacy and Resilience gene variants non-carrier population (SIGMAR1 wild type [WT]/COL24A1 wild type [WT]).
² MMSE, mean [SD] was 23.5 [3.21] for blarcamesine ABCLEAR3 population and 25.68 [2.2] for Prodromal AD population.
³ McDougall, F et al. “Psychometric Properties of the Clinical Dementia Rating - Sum of Boxes and Other Cognitive and Functional Outcomes in a Prodromal Alzheimer's Disease Population.” JPAD. vol. 8,2 (2021): 151-160.
⁴ ABCLEAR3 = Alzheimer’s Blarcamesine Cognition Efficacy and Resilience gene variants non-carrier population (SIGMAR1 wild type [WT]/COL24A1 wild type [WT]).
⁵ Christ, M G et al. “Sigma-1 Receptor Activation Induces Autophagy and Increases Proteostasis Capacity In Vitro and In Vivo.” Cells vol. 8,3 211. 2 Mar. 2019.
⁶ Baeken, M W et al. “Conserved LIR-specific interaction of Sigma-1 receptor and GABARAP.” iScience Volume 28, Issue 9, 2025, 113287.
⁷ ABCLEAR1 = Alzheimer’s Blarcamesine Cognition Efficacy and Resilience gene variant non-carrier population (SIGMAR1 wild type [WT])}

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