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Blarcamesine: A New Oral Treatment Approach in Alzheimer's Disease*
Authors:
Audrey Gabelle, MD, PhD'; Stephen MacFarlane, MD; Timo Grimmer, MD3; Luca M Villa, PhD;
Elizabeth Gordon, PhD; Thomas Jubault; Nicolas Guizard, PhD; David Gould, MD5; Wolfgang Liedtke, MDS; Kun Jin, PhD5; William R Chezem, PhD°; Juan Carlos Lopez-Talavera, MD, PhD;
Olivier Courrèges; Christopher U Missling, PhDs;
Marwan N Sabbagh, MD®
' University Hospital of Montpellier, Montpellier (France)
2 The Dementia Centre, Hammond Care, Melbourne, Victoria (Australia)
3 Technical University of Munich, School of Medicine and Health, Klinikum rechts der Isar, Munich (Germany)
* QYNAPSE, Paris (France)
5 Anavex Life Sciences, New York, New York (USA)
6 Barrow Neurological Institute, St. Joseph's Hospital and Medical Center, Phoenix, Arizona (USA)
* Drug under evaluation by EMA, not authorized; information for scientific purposes only and enabling patient self-management.
• Patient-centered, evolving approach Connected devices, digital cognitive tests, and automated imaging reduce patient burden (consultation numbers and costs, fatigue, etc. while improving engagement.
• Accelerating data-driven decision-making
Facilitates predictive model development and decision-support tools based on large-scale patient data-for clinicians, health authorities, and payers.
A Real-World Data Platform for Personalized Patient Monitoring
References:
1. Christ MG et al. Sigma-1 receptor activation induces autophagy and increases protestasis capacity in vitro and in vivo.
Cells. 2019; 8(3):211.
2. Macfarlane S. et al. Blarcamesine for the treatment of Early Alzheimer's Disease. J Prev Alzheimers Dis. 2025; 12(1):100016.
3. This document 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.
Personalized Medicine &
Blarcamesine: Unique Results
Currently, no oral Disease-Modifying Therapy (DMT) is approved for Alzheimer's disease.
Blarcamesine is a small oral molecule, a selective sigma-1 receptor (SIGMAR1) agonist, involved in regulating autophagy and cellular homeostasis.
It is under EMA regulatory review, with promising clinical results in a randomized, double-blind, placebo-controlled 48-week Phase ||b/II trial (ANAVEX2-73-AD-004; NCT03790709).
• Doses studied: 30 mg and 50 mg, orally, once daily.
• Population: ITT patients with early Alzheimer's disease confirmed by biomarkers (CSF amyloid and tau/p-tau), amyloid or functional PET, or MRI.
• Endpoints analyzed: ADAS-Cog13, CDR-SB, ADCS-ADL.
• Genetic subgroup analysis: Non-mutated
SIGMAR1 (ABCLEAR1), representing >70% of early AD patients.
Results at 48 Weeks & 192 Weeks (~4 years)
• Change from baseline analyzed by MMRM (*: p < 0.05; **: p < 0.01; ***: p < 0.001; ****: p< 0.0001).
• Side effects analyzed across all patients (n=503).
• Efficacy studied in patients with =2 visits for primary endpoint (n=462).
Subgroup Results
Mechanism-related analysis:
• The wild-type SIGMAR1 population
(ABCLEAR1) was pre-specified.
• Compared to ITT, ABCLEAR1 patients showed greater improvements at 48 weeks:
• +49.8% for ADAS-Cog13
• +33.7% for CDR-SB
ABCLEAR analysis (30 mg vs placebo):
• Improvements across all cognitive & clinical scores:
• +84.7% ADAS-Cog13 (p<0.001)
• +53.2% ADCS-ADL (p<0.01)
• +75.2% CDR-SB (p<0.0001)
• Indicating near-stability of cognitive profile.
• Confirmed by imaging & biological biomarkers.
Long-Term Data
From Open-Label Extension Phase I|/III ATTENTION-AD (NCT03790709):
• 192 weeks (~4 years) follow-up confirms clinical efficacy and safety:
• ADAS-Cog13: -3.83 (p<0.05)
• ADCS-ADL: +4.30 (p<0.05)
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