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Nizubaglustat

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Investigational substrate-modulating azasugar in Phase 3 for GM1 and GM2 gangliosidoses and Niemann-Pick disease type C.

Modality
oral brain-penetrant azasugar that inhibits glucosylceramide synthase and non-lysosomal glucosylceramidase GBA2
Availability (US)
Phase 3 NAVIGATE program; GM1/GM2 enrollment completed in July 2026 and the NPC study remained recruiting
Biomarkers
glycosphingolipids; glucosylceramide; lysosomal function; disease-specific neurologic and ataxia measures
Hallmark targeted
disabled autophagy, loss of proteostasis, intracellular lipid accumulation
Kind
investigational drug
Clinical trials
NCT07054515 — Phase 3 NAVIGATE master protocol; NCT07082543 — GM1/GM2 subprotocol; NCT07082725 — NPC subprotocol