Exaluren (sulfate)

Exaluren (ELX-02) sulfate is an investigational, advanced synthetic eukaryotic ribosome selective glycoside (ERSG). Exaluren sulfate is being developed as a therapy for genetic diseases caused by nonsense mutations^[1]. In Vitro:Exaluren (ELX-02) sulfate (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells^[1]. In Vivo:Exaluren (ELX-02) sulfate (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) sulfate 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). Exaluren (ELX-02) sulfate is rapidly eliminated from plasma in a biphasic manner with the terminal half-life (T_1/2) of 0.5 h^[1].
In a Ctns^Y226X nonsense mutant mouse, subcutaneous Exaluren (ELX-02) sulfate accumulates in kidney tissue without overt renal toxicity and that Exaluren (ELX-02) sulfate (10 mg/kg X2/week for 3 weeks) reduces renal cystine accumulation in vivo^[1].