ELX-02 (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells.

ELX-02 is rapidly eliminated from plasma in a biphasic manner with the terminal half-life (T1/2) of 0.5 h.??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.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.?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).