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nizubaglustat   Click here for help

GtoPdb Ligand ID: 13906

Synonyms: AZ-3102 | AZ3102 | compound 76 [PMID: 25250725]
Compound class: Synthetic organic
Comment: Nizubaglustat (AZ-3102) is an azosugar (formerly called iminosugars). It is orally bioavailable and can cross the blood-brain barrier. Mechanistically it acts as an inhibitor of lysosomal and non-lysosomal enzymes that regulate ceramide turnover [1]. This approach is proposed for disease-modifying potential in the treatment of GM1 or GM2 gangliosidoses (neurodegenerative lysosomal storage diseases), and is exemplified by the approval of miglustat to treat Gaucher disease.
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Ligand Activity Visualisation Charts

These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.

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View interactive charts of activity data across species
2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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Physico-chemical Properties
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Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/). All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

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Hydrogen bond acceptors 6
Hydrogen bond donors 4
Rotatable bonds 10
Topological polar surface area 93.39
Molecular weight 433.51
XLogP 1.54
No. Lipinski's rules broken 0

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)

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Canonical SMILES C1=CC=C(C=C1)C2=C(C=C(C=C2)COCCCCCN3C[C@@H]([C@H]([C@@H]([C@@H]3CO)O)O)O)F
Isomeric SMILES C1[C@@H]([C@H]([C@@H]([C@@H](N1CCCCCOCC2=CC(=C(C=C2)C3=CC=CC=C3)F)CO)O)O)O
InChI InChI=1S/C24H32FNO5/c25-20-13-17(9-10-19(20)18-7-3-1-4-8-18)16-31-12-6-2-5-11-26-14-22(28)24(30)23(29)21(26)15-27/h1,3-4,7-10,13,21-24,27-30H,2,5-6,11-12,14-16H2/t21-,22-,23+,24+/m0/s1
InChI Key IRIRASPSMMWZOM-CJRSTVEYSA-N

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
No information available.
Summary of Clinical Use Click here for help
Nizubaglustat is a clinical lead for the treatment of GM2 gangliosidoses (Tay-Sachs or Sandhoff diseases) and Niemann-Pick disease type C (NP-C). The agent has orphan drug designations dating from 2022/3, from the EU EMA and US FDA for the treatment of GM2 gangliosidoses and NP-C. Around the same time, the UK HMRA issued an "Innovation Passport" for the treatment of GM1 and GM2 gangliosidoses. Tolerability, PK and PD results from a first-in-(healthy)human study completed in Australia (registry number ACTRN12621000576820) were published in 2024 [2].
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT05758922 Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease Phase 2 Interventional Azafaros A.G.