Drug intelligence / Profile preview

blarcamesine

Development stage
Phase 3
Lead developer
Anavex Life Sciences
Modality
Classical Binding Small Molecules → Small Molecules
Administration
Oral
01

Overview

Blarcamesine is an orally administered small molecule drug developed primarily for the treatment of neurodegenerative and neurodevelopmental disorders, including Alzheimer's disease, Parkinson's disease, Rett syndrome, and Fragile X syndrome. Its mechanism of action centers on agonism of the sigma-1 receptor (SIGMAR1), an intracellular chaperone protein that modulates multiple cellular pathways related to neuroprotection and autophagy. Blarcamesine also acts as a muscarinic acetylcholine M1 receptor agonist and has some activity at the NMDA receptor. By activating SIGMAR1, blarcamesine induces autophagy and restores cellular homeostasis—processes believed to be impaired early in Alzheimer’s pathology. The drug has demonstrated memory-preserving and neuroprotective effects in preclinical models by blocking tau hyperphosphorylation, protecting mitochondria, reducing β-amyloid accumulation, and facilitating clearance of pathogenic proteins. Clinical trials have shown efficacy signals in Alzheimer's disease with a favorable safety profile[1][3][5][6][7][8].

Brand names
ANAVEX2-73ANAVEX-2-73ANAVEX 2-73
Other names
blarcamesine hydrochloride
02

Targets

SIGMAR1 (Sigma non-opioid intracellular receptor 1)NMDAR (Glutamate receptor ionotropic, NMDA)M1 (Muscarinic acetylcholine receptor M1)CHRM2 (M2)

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