VENGLUSTAT

Search structure
Synonyms:
Status: Phase 3
Entry Type: Small molecule
Molecule Category: UNKNOWN
UNII: BLP1XA3FZA

Structure
InChI Key YFHRCLAKZBDRHN-MRXNPFEDSA-N
Smile CC(C)(NC(=O)O[C@@H]1CN2CCC1CC2)c1csc(-c2ccc(F)cc2)n1
InChI
InChI=1S/C20H24FN3O2S/c1-20(2,17-12-27-18(22-17)14-3-5-15(21)6-4-14)23-19(25)26-16-11-24-9-7-13(16)8-10-24/h3-6,12-13,16H,7-11H2,1-2H3,(H,23,25)/t16-/m1/s1

Physicochemical Descriptors

Property Name Value
Molecular Formula C20H24FN3O2S
Molecular Weight 389.5
AlogP 4.0
Hydrogen Bond Acceptor 5.0
Hydrogen Bond Donor 1.0
Number of Rotational Bond 4.0
Polar Surface Area 54.46
Molecular species BASE
Aromatic Rings 2.0
Heavy Atoms 27.0

Pharmacology

Action Mechanism of Action Reference
INHIBITOR Ceramide glucosyltransferase inhibitor
Targets EC50(nM) IC50(nM) Kd(nM) Ki(nM) Inhibition(%)
Enzyme Transferase
- 300-560 32.5-160 - 50-90
Epigenetic regulator Reader Bromodomain
- - - - 50
Epigenetic regulator Reader Methyl-lysine/arginine binding protein Chromodomain
- - - - 50
Epigenetic regulator Reader Methyl-lysine/arginine binding protein PWWP domain
- - - - 50
Epigenetic regulator Reader Methyl-lysine/arginine binding protein WDR domain
- - - - 50
Epigenetic regulator Reader Plant homeodomain
- - - - 50
Epigenetic regulator Writer DNA methyltransferase
- - - - 50
Epigenetic regulator Writer Protein methyltransferase PR domain
- - - - 50
Epigenetic regulator Writer Protein methyltransferase Protein arginine methyltransferase
- - - - 50
Epigenetic regulator Writer Protein methyltransferase SET domain
- - - - 50
Epigenetic regulator Writer Protein methyltransferase
- - - - 50

Indications

Mesh Heading Maximum Phase Mesh ID Reference
Tay-Sachs Disease 3 D013661 ClinicalTrials
Gaucher Disease 3 D005776 ClinicalTrials
Sandhoff Disease 3 D012497 ClinicalTrials
Gaucher Disease 3 D005776 ClinicalTrials
Kidney Diseases, Cystic 3 D052177 ClinicalTrials
Fabry Disease 3 D000795 ClinicalTrials
Polycystic Kidney, Autosomal Dominant 2 D016891 ClinicalTrials
Parkinson Disease 2 D010300 ClinicalTrials

Related Entries

Scaffolds

Cross References

Resources Reference
ChEMBL CHEMBL4297611
DrugBank DB14966
FDA SRS BLP1XA3FZA
PubChem 60199242
SureChEMBL SCHEMBL12615329
ZINC ZINC000202143443
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