Nizubaglustat

DB21603InvestigationalSmall molecule

Nizubaglustat is a small molecule drug. The usage of the INN stem '-glustat' in the name indicates that Nizubaglustat is a ceramide glucosyltransferase inhibitor. Nizubaglustat is under investigation in clinical... Nizubaglustat is under investigation in clinical trial NCT07054515 (A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis).

Mechanism: Pharmacologically active on Ceramide glucosyltransferase and Non-lysosomal glucosylceramidase
Curator reviewed
Formula / weight: C₂₄H₃₂FNO₅ · 433.52 g/mol (avg)
Code names: AZ-3102

Resolves to

Compound

UNII2X6NHN9RRNCAS1633666-49-5ChEMBLCHEMBL3354637

Structure

InChIKeyIRIRASPSMMWZOM-CJRSTVEYSA-NSMILESOC[C@H]1[C@@H](O)[C@H](O)[C@@H](O)CN1CCCCCOCC1=CC(F)=C(C=C1)C1=CC=CC=C1

Targets

Q16739UGCGQ9HCG7GBA2

What you can answer from here — as of September 16, 2026

2Protein targetsEach mapped to UniProt, with action and pharmacological action

Listed above

6Clinical trialsPhase, status and sponsor resolved per trial

2+ReferencesStructured and connected to the statements they support

Which drugs share a target with Nizubaglustat, and which of those have an active Phase 3 trial?

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