ELX-02 disulfate (C09-1130-680)

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.In VitroELX-02 (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells. MCE has not independently confirmed the accuracy of these methods. They are for reference only. Cell Cytotoxicity AssayCell Line: Wildtype human proximal tubule cells (HK-2) Concentration: 0, 100 and 400 μg/mL Incubation Time: 0, 24, 48 and 72 hours Result: Cytotoxicity assay in wildtype human proximal tubule cells (HK-2) showing no toxic effect of 400 μg/mL at 0, 24, 48 and 72 h.In VivoELX-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 . ?/nIn plasma ELX-02 is rapidly absorbed with a T max 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 (T 1/2 ) of 0.5 h . ?/nIn a Ctns Y226X 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 . MCE has not independently confirmed the accuracy of these methods. They are for reference only. Animal Model: Twenty-nine Ctns Y226X/Y226X mice 5-7 month old 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).Form:Solid. Specification: ≥98.0% Molecular Formula: C19H42N4O18S2 Molecular Weight: 678.68 PubChem CID: 138454758