Details of the Drug

General Information of Drug (ID: DMK3C4L)

Drug Name

ELX-02

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

Indication

Disease Entry	ICD 11	Status	REF
Cystic fibrosis	CA25	Phase 2	[1]
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Drug Type

Small molecule

Structure

3D MOL

2D MOL

Chemical Identifiers

Formula: C19H38N4O10
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
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
InChIKey: KJBRSTPUILEBDR-DBMIJKFDSA-N

Cross-matching ID

PubChem CID: 71461382
TTD ID: D18ATS

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Ribosome (hRBS)	TTJ073B	NOUNIPROTAC	Modulator	[2]

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Molecular Interaction Atlas (MIA)

MIA Detail

Jump to Detail Molecular Interaction Atlas of This Drug

References

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.
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.