| Chemical Properties | Back Directory | [storage temp. ]
Store at -20°C, stored under nitrogen | [solubility ]
|DMSO : < 1 mg/mL (insoluble or slightly soluble) | [form ]
Solid | [color ]
White to light yellow | [Water Solubility ]
Water : 100 mg/mL (147.34 mM; Need ultrasonic) |
| Hazard Information | Back Directory | [Uses]
Exaluren (ELX-02) disulfate is an investigational, advanced synthetic eukaryotic ribosome selective glycoside (ERSG). Exaluren disulfate is being developed as a therapy for genetic diseases caused by nonsense mutations[1]. | [Biological Activity]
ELX-02 disulfate (NB-124 disulfate) is an investigational, advanced synthetic eukaryotic ribosome selective glycoside (ERSG). ELX-02 disulfate is being developed as a therapy for genetic diseases caused by nonsense mutations[1].
ELX-02 (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells[1].
ELX-02 (10 and 30 mg/kg; repeat subcutaneous administration; twice weekly, total of 8 doses) shows accumulation in tissues that is dose dependent without gender difference[1].In plasma ELX-02 is rapidly absorbed with a Tmax of 0.25 h after both single (a single subcutaneous injection at 10 mg/kg at dose volume of 5 mL/kg) and repeated administration (twice weekly with 10 mg/kg/dose for 21 days; total of 7 administrations). ELX-02 is rapidly eliminated from plasma in a biphasic manner with the terminal half-life (T1/2) of 0.5 h[1]. In a CtnsY226X nonsense mutant mouse, subcutaneous ELX-02 accumulates in kidney tissue without overt renal toxicity and that ELX-02 (10 mg/kg X2/week for 3 weeks) reduces renal cystine accumulation in vivo[1]. | [in vivo]
Exaluren (ELX-02) disulfate (10 and 30 mg/kg; repeat subcutaneous administration; twice weekly, total of 8 doses) shows accumulation in tissues that is dose dependent without gender difference[1].
In plasma Exaluren (ELX-02) disulfate is rapidly absorbed with a Tmax of 0.25 h after both single (a single subcutaneous injection at 10 mg/kg at dose volume of 5 mL/kg) and repeated administration (twice weekly with 10 mg/kg/dose for 21 days; total of 7 administrations). Exaluren (ELX-02) disulfate is rapidly eliminated from plasma in a biphasic manner with the terminal half-life (T1/2) of 0.5 h[1].
In a CtnsY226X nonsense mutant mouse, subcutaneous Exaluren (ELX-02) disulfate accumulates in kidney tissue without overt renal toxicity and that Exaluren (ELX-02) disulfate (10 mg/kg X2/week for 3 weeks) reduces renal cystine accumulation in vivo[1]. | Animal Model: | Twenty-nine CtnsY226X/Y226X mice 5-7 month old[1] | | Dosage: | 10 and 30 mg/kg | | Administration: | Subcutaneous injection, at dose volume of 5 mL/kg, twice weekly for a period of 28 days (total of 8 doses) | | Result: | Highest levels were measured in the kidney, followed by spleen and liver, with lower levels in other tissues (lung, heart, cochlea and brain). |
| [storage]
Store at -20°C, stored under nitrogen | [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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