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[CAS NO. 1629063-78-0] Ibiglustat(L-Malicacid)
| Ships within | Stock | Price | Qty | Total |
Please click "REQUEST A QUOTE" button if there is no stock, or you need other sizes or custom synthesis.
| Catalog: | HY-16743A |
| Brand: | MCE |
| CAS: | 1629063-78-0 |
Overview
| MDL | - |
|---|---|
| Molecular Weight | 523.57 |
| Molecular Formula | C24H30FN3O7S |
| SMILES | O=C(O[C@@H]1CN2CCC1CC2)NC(C)(C3=CSC(C4=CC=C(F)C=C4)=N3)C.OC(C[C@H](O)C(O)=O)=O |
1 Publications Citing Use of MCE
Summary
Ibiglustat (Venglustat) L-Malic acid is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. Ibiglustat L-Malic acid can be used for the research of Gaucher disease type 3, Parkinson's disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease [1] [2] .
IC50 & Target
Glucosylceramide synthase [1] .
In Vitro
Ibiglustat (SAR402671) (1 μM, 15 days) L-Malic acid treated Fabry disease (FD) cells are close to the physiological level in untreated WT cells in GL-3 levels, suggesting that Ibiglustat L-Malic acid can prevent additional GL-3 accumulation and could serve to ameliorate the abundant levels of this sphingolipid in FD cardiomyocytes [4] .
MCE has not independently confirmed the accuracy of these methods. They are for reference only.
Clinical Trial
| NCT Number | Sponsor | Condition | Start Date | Phase |
|---|---|---|---|---|
| NCT05222906 | Genzyme, a Sanofi Company|Sanofi |
Gaucher´s Disease Type III
|
April 18, 2022 | Phase 3 |
| NCT03523728 | Genzyme, a Sanofi Company|Sanofi |
Polycystic Kidney, Autosomal Dominant
|
October 4, 2018 | Phase 2|Phase 3 |
| NCT02843035 | Genzyme, a Sanofi Company|Sanofi |
Gaucher Disease Type 1|Gaucher Disease Type 3
|
January 4, 2017 | Phase 2 |
| NCT02906020 | Genzyme, a Sanofi Company|Sanofi |
Parkinson´s Disease
|
December 15, 2016 | Phase 2 |
| NCT04221451 | Genzyme, a Sanofi Company|Sanofi |
Tay-Sachs Disease Sandhoff Disease
|
June 29, 2020 | Phase 3 |
| NCT05238714 | Genzyme, a Sanofi Company|Sanofi |
Disorders of Sphingolipid Metabolism (Healthy Volunteers)
|
May 26, 2020 | Phase 1 |
| NCT04705051 | Sanofi |
Congenital Cystic Kidney Disease
|
February 9, 2021 | Phase 3 |
| NCT05280548 | Genzyme, a Sanofi Company|Sanofi |
Fabry´s Disease
|
May 3, 2022 | Phase 3 |
| NCT05206773 | Genzyme, a Sanofi Company|Sanofi |
Fabry Disease
|
March 11, 2022 | Phase 3 |
| NCT03687554 | Genzyme, a Sanofi Company|Sanofi |
Healthy Volunteers|Polycystic Kidney, Autosomal Dominant
|
October 5, 2018 | Phase 1 |
Appearance
Solid
Shipping
Room temperature in continental US; may vary elsewhere.
Storage
| Powder | -20°C | 3 years |
|---|---|---|
| 4°C | 2 years | |
| In solvent | -80°C | 6 months |
| -20°C | 1 month |
Solvent & Solubility
DMSO : ≥ 100 mg/mL ( 191.00 mM )
* "≥" means soluble, but saturation unknown.
Stock Solutions
| Concentration Solvent Mass | 1 mg | 5 mg | 10 mg |
|---|
| 1 mM | 1.9100 mL | 9.5498 mL | 19.0996 mL |
| 5 mM | 0.3820 mL | 1.9100 mL | 3.8199 mL |
| 10 mM | 0.1910 mL | 0.9550 mL | 1.9100 mL |
References
- [1] Viel C, et al. Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synucleinopathy model. Sci Rep. 2021;11(1):20945. Published 2021 Oct 22.
- [2] Peterschmitt MJ, et al. Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers. Clin Pharmacol Drug Dev. 2021;10(1):86-98.
- [3] Iva Stojkovska, et al. Molecular mechanisms of α-synuclein and GBA1 in Parkinson’s disease. Cell Tissue Res. 2017.
- [4] Itier JM, et al. Effective clearance of GL-3 in a human iPSC-derived cardiomyocyte model of Fabry disease. J Inherit Metab Dis. 2014 Nov;37(6):1013-22.
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