Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D18ATS
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| Drug Name |
ELX-02
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| Synonyms |
exaluren; ELX-02; Exaluren [INN]; TP732M43NA; 1375073-93-0; NB-124; HY-114231B; CS-0095334; ELX-02free; ELX-02 free; UNII-TP732M43NA; CHEMBL2220350; GTPL10944; EX-A7401; HY-114231; CS-0080240; (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; 4-O-(2-AMINO-2,7-DIDEOXY-D-GLYCERO-.ALPHA.-D-GLUCOHEPTOPYRANOSYL)-5-O-(5-AMINO-5,6-DIDEOXY-.ALPHA.-L-TALOFURANOSYL)-2-DEOXY-D-STREPTAMINE; D-STREPTAMINE, O-2-AMINO-2,7-DIDEOXY-D-GLYCERO-.ALPHA.-D-GLUCO-HEPTOPYRANOSYL-(1->4)-O-(5-AMINO-5,6-DIDEOXY-.ALPHA.-L-TALOFURANOSYL-(1->5))-2-DEOXY-; D-Streptamine, O-2-amino-2,7-dideoxy-D-glycero-alpha-D-gluco-heptopyranosyl-(1->4)-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl-(1->5))-2-deoxy-; O-2-Amino-2,7-dideoxy-D-glycero-alpha-D-gluco-heptopyranosyl-(1->4)-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl-(1->5))-2-deoxy-D-streptamine
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| Drug Type |
Small molecule
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| Indication | Cystic fibrosis [ICD-11: CA25; ICD-10: E84, E84.9] | Phase 2 | [1] | |
| Company |
Eloxx Pharmaceuticals
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| Structure |
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Download2D MOL |
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| Formula |
C19H38N4O10
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| Canonical 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
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| InChI |
InChI=1S/C19H38N4O10/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/h4-19,24-29H,3,20-23H2,1-2H3/t4-,5+,6+,7-,8+,9-,10+,11-,12-,13+,14+,15+,16+,17+,18+,19-/m0/s1
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| InChIKey |
KJBRSTPUILEBDR-DBMIJKFDSA-N
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| PubChem Compound ID | ||||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Ribosome (hRBS) | Target Info | Modulator | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04135495) A Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients With Cystic Fibrosis With at Least One G542X Allele. U.S.National Institutes of Health. | |||
| REF 2 | A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Escalation Study to Evaluate the Safety and Pharmacokinetics of ELX-02 in Healthy Subjects. Clin Pharmacol Drug Dev. 2021 Aug;10(8):859-869. | |||
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