For research use only. Not for therapeutic Use.
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].
Exaluren (ELX-02) disulfate (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells[1].
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].
| Catalog Number | I019080 |
| CAS Number | 2244622-33-9 |
| Synonyms | (2R,3S,4R,5R,6S)-5-amino-6-[(1R,2R,3S,4R,6S)-4,6-diamino-2-[(2S,3R,4S,5R)-5-[(1S)-1-aminoethyl]-3,4-dihydroxyoxolan-2-yl]oxy-3-hydroxycyclohexyl]oxy-2-[(1R)-1-hydroxyethyl]oxane-3,4-diol;sulfuric acid |
| Molecular Formula | C19H42N4O18S2 |
| Purity | ≥95% |
| InChI | InChI=1S/C19H38N4O10.2H2O4S/c1-4(20)14-12(28)13(29)19(30-14)33-17-9(25)6(21)3-7(22)16(17)32-18-8(23)10(26)11(27)15(31-18)5(2)24;2*1-5(2,3)4/h4-19,24-29H,3,20-23H2,1-2H3;2*(H2,1,2,3,4)/t4-,5+,6+,7-,8+,9-,10+,11-,12-,13+,14+,15+,16+,17+,18+,19-;;/m0../s1 |
| InChIKey | WYGVCSNIMZZOIT-BPQPGTHUSA-N |
| SMILES | CC(C1C(C(C(O1)OC2C(C(CC(C2OC3C(C(C(C(O3)C(C)O)O)O)N)N)N)O)O)O)N.OS(=O)(=O)O.OS(=O)(=O)O |
| Reference | [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. |
