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500mg |
ln Vitro |
In human cells, exaluren (ELX-02) sulfate (100–400 μg/mL) can read nonsense mutations and is non-toxic [1].
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ln Vivo |
There are no gender differences in the dose-dependent accumulation of exaluren (ELX-02) sulfate (10 and 30 mg/kg; repeated subcutaneous injections; twice weekly for a total of 8 doses) in tissues [1]. Exaluren (ELX-02) sulfate is rapidly absorbed in plasma, with a Tmax of 0.25 hours. It was provided as a single subcutaneous injection at a dose of 5 mL/kg, 10 mg/kg in a treatment volume of 5 mL/kg), and recurrent dosing (twice weekly, 10 mg/kg each time). kg/dose for 21 days, totaling 7 doses. Exaluren (ELX-02) sulfate has a biphasic terminal half-life (T1/2) of 0.5 hours and is rapidly removed from plasma [1]. Exaluren (ELX-02) sulfate (10 mg/kg X2/week, Lasts for 3 weeks) can minimize the buildup of cystine in the kidney and accumulated in renal tissue without evident nephrotoxicity in CtnsY226X nonsense mutant mice [1].
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Cell Assay |
Cytotoxicity assay[1]
Cell Types: Wild-type human proximal tubule cells (HK-2) Tested Concentrations: 0, 100 and 400 μg/mL Incubation Duration: 0, 24, 48 and 72 hrs (hours) Experimental Results: Wild-type human proximal tubule cells Cell cytotoxicity assay (HK-2) 400 μg/mL had no toxic effects at 0, 24, 48 and 72 hrs (hours). |
Animal Protocol |
Animal/Disease Models: 29 CtnsY226X/Y226X mice, 5-7 months old [1]
Doses: 10 and 30 mg/kg Route of Administration: subcutaneous injection, dose volume 5 mL/kg, twice a week for 28 days ( Total 8 doses) Experimental Results: The highest levels were measured in the kidneys, followed by the spleen and liver, with lower levels in other tissues (lungs, heart, cochlea and brain). |
References |
[1]. Leubitz A, et al. Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX-02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers. Clin Pharmacol Drug Dev. 2019 Jan 16.
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Molecular Formula |
C19H40N4O14S
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Molecular Weight |
580.6043
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CAS # |
1375073-94-1
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Related CAS # |
Exaluren disulfate;2244622-33-9;Exaluren;1375073-93-0
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Appearance |
Typically exists as solids (or liquids in special cases) at room temperature
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SMILES |
S(=O)(=O)(O[H])O[H].O([C@]1([H])[C@@]([H])([C@]([H])([C@@]([H])([C@@]([H])([C@@]([H])(C([H])([H])[H])O[H])O1)O[H])O[H])N([H])[H])[C@]1([H])[C@]([H])(C([H])([H])[C@]([H])([C@@]([H])([C@@]1([H])O[C@@]1([H])[C@@]([H])([C@@]([H])([C@@]([H])([C@]([H])(C([H])([H])[H])N([H])[H])O1)O[H])O[H])O[H])N([H])[H])N([H])[H]
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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) |
May dissolve in DMSO (in most cases), if not, try other solvents such as H2O, Ethanol, or DMF with a minute amount of products to avoid loss of samples
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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 | 1.7224 mL | 8.6118 mL | 17.2236 mL | |
5 mM | 0.3445 mL | 1.7224 mL | 3.4447 mL | |
10 mM | 0.1722 mL | 0.8612 mL | 1.7224 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.