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nizubaglustat
- Development stage
- Phase 3
- Lead developer
- Azafaros
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
- Administration
- Oral
01
Overview
Nizubaglustat is a novel, orally available, brain-penetrant small molecule azasugar developed as a potential disease-modifying therapy for rare lysosomal storage disorders with neurological involvement, including GM1 gangliosidosis, GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases), and Niemann-Pick disease type C. It acts as a dual inhibitor of two key enzymes in the impaired metabolic pathway—ceramide glucosyltransferase (glucosylceramide synthase) and non-lysosomal neutral glucosylceramidase (GBA2 protein)—to reduce the harmful accumulation of waste lipids in cells. This mechanism aims to restore more normal cell function by addressing the underlying cause of these neurodegenerative disorders. Nizubaglustat is being developed by Azafaros, based on discoveries from scientists at Amsterdam UMC and Leiden University[1][2][3][4][5][7][8].
- Other names
- nizubaglustat
02
Targets
UGCG (UDP-glucose ceramide glucosyltransferase)GBA2 (Beta-glucosidase 2)
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