| Description | Sinbaglustat (OGT2378) (OGT2378) is a dual inhibitor of glucose ceramide synthetase (GCS) and non-lysosomal glucose ceramidase (GBA2).Sinbaglustat is an orally administered N-alkyl iminosugar that crosses the blood-brain barrier.Sinbaglustat is used for the treatment of lysosomal storage disorders and for the study of central neurodegenerative disorders associated with lysosomal dysfunction. Sinbaglustat is used for the treatment of lysosomal storage disorders and for the study of lysosomal dysfunction-related central neurodegenerative diseases. |
| In vitro | Sinbaglustat (OGT2378) (20 μM) leads to a remarkable reduction in the synthesis of glucosylceramide by 93% and ganglioside by >95% when compared to untreated MEB4 cells. This reduction occurs without inducing any cytotoxicity or antiproliferative effects.[1] Sinbaglustat is 50-fold more potent in inhibiting GBA2 than GCS.[2] |
| In vivo | Sinbaglustat (OGT2378) (2500 mg/kg/day; p.o.; in the powdered chow; corresponding to 35-40 mg/mouse/day of Sinbaglustat; Female syngeneic C57BL/6 mice and bearing MEB4 melanoma tumor, 6-8 weeks old bearing MEB4 melanoma tumor) is highly effective in impeding melanoma tumor growth in vivo. The effectiveness of p.o. Sinbaglustat in this murine model suggests that inhibition of glycosphingolipid synthesis is a promising approach to inhibit tumor progression.[1] |
| Synonyms | ACT-519276, Sinbaglustat, OGT2378 |
| molecular weight | 233.3 |
| Molecular formula | C11H23NO4 |
| CAS | 441061-33-2 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |
| Solubility | DMSO: 46.66 mg/mL (200 mM) |
| References | 1. Michael Weiss, et al. Inhibition of melanoma tumor growth by a novel inhibitor of glucosylceramide synthase. Cancer Res. 2003 Jul 1;63(13):3654-8. 2. Martine Gehin, et al. Assessment of Target Engagement in a First-in-Human Trial with Sinbaglustat, an Iminosugar to Treat Lysosomal Storage Disorders. Clin Transl Sci. 2021 Mar;14(2):558-567. |