New clinical Precision Medicine population data demonstrates up to 84.6 Weeks (19.5 Months) ‘time saved’ by early-start
ADAS-Cog13 difference: −5.43 (P = 0.0035), ADCS-ADL difference: +9.50 (P < 0.0001)
Restoring impaired autophagy as early event, preceding amyloid-beta and tau
Oral presentation at the Alzheimer’s Association International Conference (AAIC) 2025
NEW YORK – July 31, 2025
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.
The data were presented by Marwan Noel Sabbagh, MD, Professor of Neurology, and Chairman of the Anavex Scientific Advisory Board at the 2025 Alzheimer’s Association International Conference (AAIC), held in Toronto.
The ATTENTION-AD (ANAVEX®2-73-AD-EP-004) open-label extension (OLE) Phase IIb/III treatment trial followed the 48-week ANAVEX®2-73-AD-004 double-blind (DB) clinical trial, with a combined duration of up to 192 weeks. The trial was designed to evaluate the safety and tolerability of blarcamesine as well as its long-term effects on cognition (ADAS-Cog13) and function (ADCS-ADL) in participants with early Alzheimer’s disease. [1]
Blarcamesine-treated patients continue to accrue benefit through up to 4 years, as measured by the pre-specified clinical endpoints ADAS-Cog13 and ADCS-ADL, respectively. In the intent-to-treat (ITT) population, delayed-start analysis of treatment with oral blarcamesine was significant for both cognition and function, reflecting the importance of early treatment initiation. For ADAS-Cog13 a significant difference between the early-start and late-start treatment groups at Week 192 (LS mean difference −3.83, P = 0.0165) was observed. [2] Similarly, for ADCS-ADL at Week 192 statistical significance (LS mean difference +4.30, P = 0.0206) was reached. Additionally, the GWAS-identified [3] population ABCLEAR2 [4], with a global frequency of ~71.7% [5], showed further improvement in both cognition (LS mean difference −5.43, P = 0.0035) and function (LS mean difference +9.50, P < 0.0001). This clinical Precision Medicine population data demonstrates up to 84.6 Weeks (19.5 Months) of ‘time saved’ by the early-start analysis.
In Alzheimer’s disease clinical trials, ‘time saved’ refers to the estimated amount of time a treatment delays the progression of the disease, allowing patients to maintain functionality and independence longer—an inherently meaningful measurement for real-world outcomes. [6][7] Additionally, blarcamesine exhibited a favorable safety profile with no treatment-related deaths.
“This Precision Medicine data provide potentially continued persuasive evidence that earlier initiation of treatment with blarcamesine may have a significant positive impact on disease progression and may provide sustained clinically meaningful benefits to patients with early Alzheimer’s disease over the long-term,” said Dr. Sabbagh. “Prespecified delayed-start analysis indicate disease-modifying effect of oral blarcamesine and highlight the importance of early and continued long-term treatment.”
Further presentations at AAIC 2025 featured prespecified Precision Medicine data from the 48-week ANAVEX®2-73-AD-004 double-blind trial, confirming that blarcamesine restores impaired autophagy as an early event preceding amyloid-beta and tau pathology.
The mechanistic confirmation that blarcamesine restores impaired autophagy through SIGMAR1 activation and acts upstream of amyloid and tau was previously established in vitro and in vivo. Studies demonstrated enhanced autophagic flux and increased proteostasis capacity, ameliorating paralysis caused by protein aggregation in C. elegans. [8]
A clinical Precision Medicine approach also confirmed that the SIGMAR1 wild-type (WT) population, ABCLEAR1 [9], which represents up to 70% of the global population, achieved deeper clinical responses to blarcamesine. Additional GWAS-defined responder groups may further enhance treatment efficacy within heterogeneous Alzheimer’s disease populations.
While the ITT population showed significant improvement versus placebo after 48 weeks (36.3% for ADAS-Cog13 and 27.6% for CDR-SB), the ABCLEAR1 population demonstrated even greater improvement. [10] In the 30 mg blarcamesine group, serious TEAEs occurred in 12.7% receiving drug and 9.1% on placebo; no deaths occurred in the blarcamesine group.
“We remain excited about these enhanced clinically meaningful improvements which were further confirmed by the observed efficacy treatment effects from the two blarcamesine clinical trials with identified Precision Medicine patient populations,” said Juan Carlos Lopez-Talavera, MD, PhD, Head of R&D. “Convenient once-daily oral dosing may allow us to offer a scalable and patient-friendly option for early Alzheimer’s.”
“We are motivated by the Anavex team’s continued contributions to advancing science across this devastating chronic disease,” said Christopher U Missling, PhD, President & CEO. “Additionally, the comprehensive data from the blarcamesine Alzheimer’s program represents a solid foundation for continuous engagement with the Alzheimer’s disease community.”
The respective presentations are available on the Investors section of the Company’s website at www.anavex.com.
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 investigational uses will successfully complete development or gain regulatory approval.
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company developing novel therapeutics for neurodegenerative, neurodevelopmental, and neuropsychiatric disorders. Its lead candidate, ANAVEX®2-73 (blarcamesine), has completed multiple trials across Alzheimer’s disease, Parkinson’s disease dementia, and Rett syndrome. Further information is available at www.anavex.com. Connect with the Company on Twitter, Facebook, Instagram, and LinkedIn.
Forward-Looking Statements
Statements in this press release that are not historical are forward-looking and involve risks and uncertainties. Actual results may differ materially. Readers should not place undue reliance on these statements. Anavex undertakes no obligation to update forward-looking statements.
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
References
[1] Scheduled visits mapping for DB + OLE structure.
[2] Minimal clinically important differences reference: Muir RT et al., 2024.
[3] GWAS = Genome-Wide Association Study.
[4] ABCLEAR2 definition.
[5] SNP Reference: https://www.ncbi.nlm.nih.gov/snp/
[6] Petersen RC et al., 2023.
[7] Dickson SP et al., 2024.
[8] Christ MG et al., 2019.
[9] ABCLEAR1 definition.
[10] Macfarlane S et al., 2025.