Drug General Information
Drug ID
D04BQV
Former ID
DCL000138
Drug Name
Isofagomine tartrate
Synonyms
Plicera; Afegostat tartrate; AT-2101; HGT-3410; Plicera (TN); NN-42-1007
Drug Type
Small molecular drug
Indication Metabolic disease [ICD9: 270-279; ICD10:E70-E89] Phase 2 [542434], [548215]
Company
Amicus Therape.
Structure
Download
2D MOL

3D MOL

Formula
C6H13NO3
InChI
InChI=1S/C6H13NO3/c8-3-4-1-7-2-5(9)6(4)10/h4-10H,1-3H2/t4-,5-,6-/m1/s1
InChIKey
QPYJXFZUIJOGNX-HSUXUTPPSA-N
CAS Number
CAS 957230-65-8
PubChem Compound ID
PubChem Substance ID
Target and Pathway
Target(s) Glucosylceramidase Target Info Inhibitor [536895], [536909]
KEGG Pathway Other glycan degradation
Sphingolipid metabolism
Metabolic pathways
Lysosome
PathWhiz Pathway Sphingolipid Metabolism
Reactome Glycosphingolipid metabolism
WikiPathways Sphingolipid metabolism
References
Ref 542434(http://www.guidetopharmacology.org/) Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 7410).
Ref 548215Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800023168)
Ref 536895Isofagomine induced stabilization of glucocerebrosidase. Chembiochem. 2008 Nov 3;9(16):2643-9.
Ref 536909Identification of pharmacological chaperones for Gaucher disease and characterization of their effects on beta-glucocerebrosidase by hydrogen/deuterium exchange mass spectrometry. Chembiochem. 2008 Nov 3;9(16):2650-62.

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