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Compound Detail

CHEMBL1086997

LUCERASTAT
🧪 Phase III
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🧪 Phase III
CCCCN1C[C@H](O)[C@@H](O)[C@@H](O)[C@H]1CO

Basic Information

ChEMBL ID CHEMBL1086997
Name LUCERASTAT
Phase Phase III
Structure Type MOL
InChI Key UQRORFVVSGFNRO-XFWSIPNHSA-N

Known Names

ACT-434964 Lucerastat N-butyldeoxygalactonojirimycin N-n-butyl deoxygalactonojirimycin
6
Targets
11
Activities
1
Assay Types
0
PK Parameters
12
Publications
4
Known Names
1
Indications
1
Mechanisms

Molecular Properties

219.3
MW (Da)
-1.45
ALogP
5
HBA
4
HBD
84.2
PSA (Ų)
0.46
QED
0
Ro5 Violations
4
Rot. Bonds

Drug Information

Molecule Type Small molecule
Chirality Single Enantiomer

Flags

Natural Product
USAN Stem -stat
USAN Year 2018

Extended Properties

Molecular Formula C10H21NO4
MW 219.28
Aromatic Rings 0
Heavy Atoms 15
NP-Likeness 1.24

Mechanism of Action

Mechanism Action Type Target Direct Efficacy
Ceramide glucosyltransferase inhibitor INHIBITOR Ceramide glucosyltransferase

Indications

Fabry Disease

Activity Summary

IC50 11 meas. best 6.52

Target Activity Profile

Target Type Best pChEMBL Mean pChEMBL Best (nM) # Data
Beta-glucosidase
CHEMBL3761
IC50 6.52 6.52 300.0 2
Sucrase-isomaltase, intestinal
CHEMBL2748
IC50 6.34 6.34 460.0 1
Lysosomal alpha-glucosidase
CHEMBL2608
IC50 5.82 5.82 1500.0 1
Ceramide glucosyltransferase
CHEMBL2063
IC50 5.0 5.0 10000.0 2
Maltase-glucoamylase
CHEMBL2074
IC50 5.0 5.0 10000.0 1
Glycogen debranching enzyme
CHEMBL5272
IC50 5.0 5.0 10000.0 1

Activity Data Samples

Top measurements by pChEMBL value

Literature References

PubMed DOI Target Context # Activities
23363020 10.1021/jm301557k Beta-galactosidase 6
20000679 10.1021/jm901281m Ceramide glucosyltransferase 1
20000679 10.1021/jm901281m Sucrase-isomaltase, intestinal 1
20000679 10.1021/jm901281m Maltase-glucoamylase 1
20000679 10.1021/jm901281m Lactase/phlorizin hydrolase 1
20000679 10.1021/jm901281m Unchecked 1
20000679 10.1021/jm901281m Beta-glucosidase 1
20000679 10.1021/jm901281m Lysosomal alpha-glucosidase 1
20000679 10.1021/jm901281m Glycogen debranching enzyme 1
25250725 10.1021/jm501181z Ceramide glucosyltransferase 1
25250725 10.1021/jm501181z Lysosomal acid glucosylceramidase 1
25250725 10.1021/jm501181z Beta-glucosidase 1

Data from ChEMBL 36 via Core Engine