| Size | Price | Stock | Qty |
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| 5mg |
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| 10mg |
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| 100mg | |||
| Other Sizes |
| Targets |
ML198 targets glucocerebrosidase (GCase), a lysosomal enzyme that catalyzes the breakdown of glucocerebroside. Mutations in the GCase gene cause Gaucher disease, leading to the accumulation of glucocerebroside in lysosomes. ML198 acts as a non-inhibitory chaperone, binding to GCase and facilitating its proper folding and translocation to the lysosome, without inhibiting its enzymatic activity.
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| ln Vitro |
For more than 48 hours, ML198 remains stable in D-PBS pH 7.4 at room temperature [1]. In human fibroblasts, ML198 enhances GCase translocation to lysosomes (20% cellular translocation at 5 µM vs. 5% in DMSO) [1]. ML198 has low water solubility, high microsomal stability, and Caco-2 permeability [1].
In vitro, ML198 activates GCase with an EC50 of 0.4 μM. In human fibroblasts, ML198 enhances GCase translocation to lysosomes, with 20% cellular translocation at 5 µM compared to 5% in DMSO. The compound shows promising microsomal stability and Caco-2 permeability, indicating good drug-like properties. However, it has low water solubility. |
| ln Vivo |
Detailed in vivo studies for ML198 are limited. As a research tool, it is primarily used in cell-based assays to study GCase function and Gaucher disease.
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| Enzyme Assay |
The in vitro enzyme activation assay for ML198 involves measuring GCase activity in the presence of the compound. The EC50 is determined from dose-response curves.
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| Cell Assay |
Cell-based assays for ML198 involve treating Gaucher disease patient fibroblasts with the compound and measuring GCase activity and lysosomal translocation. The compound's ability to enhance GCase translocation to lysosomes is assessed by immunofluorescence or subcellular fractionation.
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| Animal Protocol |
In vivo animal studies for ML198 are not well-documented.
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| ADME/Pharmacokinetics |
ML198 has a molecular weight of 290.32 g/mol and a molecular formula of C17H14N4O. It is soluble in DMSO (4 mg/mL) and practically insoluble in water and ethanol. The compound should be stored at -20°C for long-term stability.
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| Toxicity/Toxicokinetics |
The toxicity profile of ML198 is limited. As a research compound, comprehensive toxicological evaluations have not been published.
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| References | |
| Additional Infomation |
ML198 is a research compound for studying GCase and Gaucher disease. It has not entered clinical trials. Its mechanism involves GCase activation and chaperone activity.
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| Molecular Formula |
C17H14N4O
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|---|---|
| Molecular Weight |
290.319262981415
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| Exact Mass |
290.116
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| CAS # |
1380716-06-2
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| PubChem CID |
46907762
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| Appearance |
Light yellow to yellow solid powder
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| LogP |
2.2
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| Hydrogen Bond Donor Count |
1
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| Hydrogen Bond Acceptor Count |
3
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| Rotatable Bond Count |
3
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| Heavy Atom Count |
22
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| Complexity |
463
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| Defined Atom Stereocenter Count |
0
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| SMILES |
O=C(C1C=NN2C(C)=CC(C)=NC2=1)NC1C=CC(C#C)=CC=1
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| InChi Key |
HVZPJBKUFRREQC-UHFFFAOYSA-N
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| InChi Code |
InChI=1S/C17H14N4O/c1-4-13-5-7-14(8-6-13)20-17(22)15-10-18-21-12(3)9-11(2)19-16(15)21/h1,5-10H,2-3H3,(H,20,22)
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| Chemical Name |
N-(4-ethynylphenyl)-5,7-dimethylpyrazolo[1,5-a]pyrimidine-3-carboxamide
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| Synonyms |
ML 198; ML-198; ML198
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| HS Tariff Code |
2934.99.9001
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| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
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| Solubility (In Vitro) |
DMSO : ~5 mg/mL (~17.22 mM)
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|---|---|
| Solubility (In Vivo) |
Note: Listed below are some common formulations that may be used to formulate products with low water solubility (e.g. < 1 mg/mL), you may test these formulations using a minute amount of products to avoid loss of samples.
Injection Formulations
Injection Formulation 1: DMSO : Tween 80： Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution → 50 μL Tween 80 → 850 μL Saline)(e.g. IP/IV/IM/SC) *Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH ₂ O to obtain a clear solution. Injection Formulation 2: DMSO : PEG300 ：Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL DMSO → 400 μLPEG300 → 50 μL Tween 80 → 450 μL Saline) Injection Formulation 3: DMSO : Corn oil = 10 : 90 (i.e. 100 μL DMSO → 900 μL Corn oil) Example: Take the Injection Formulation 3 (DMSO : Corn oil = 10 : 90) as an example, if 1 mL of 2.5 mg/mL working solution is to be prepared, you can take 100 μL 25 mg/mL DMSO stock solution and add to 900 μL corn oil, mix well to obtain a clear or suspension solution (2.5 mg/mL, ready for use in animals). View More
Injection Formulation 4: DMSO : 20% SBE-β-CD in saline = 10 : 90 [i.e. 100 μL DMSO → 900 μL (20% SBE-β-CD in saline)] Oral Formulations
Oral Formulation 1: Suspend in 0.5% CMC Na (carboxymethylcellulose sodium) Oral Formulation 2: Suspend in 0.5% Carboxymethyl cellulose Example: Take the Oral Formulation 1 (Suspend in 0.5% CMC Na) as an example, if 100 mL of 2.5 mg/mL working solution is to be prepared, you can first prepare 0.5% CMC Na solution by measuring 0.5 g CMC Na and dissolve it in 100 mL ddH2O to obtain a clear solution; then add 250 mg of the product to 100 mL 0.5% CMC Na solution, to make the suspension solution (2.5 mg/mL, ready for use in animals). View More
Oral Formulation 3: Dissolved in PEG400 (Please use freshly prepared in vivo formulations for optimal results.) |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 3.4445 mL | 17.2224 mL | 34.4448 mL | |
| 5 mM | 0.6889 mL | 3.4445 mL | 6.8890 mL | |
| 10 mM | 0.3444 mL | 1.7222 mL | 3.4445 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation:：Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
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