Synonyms: AKCEA-TTR-LRx | Ionis-TTR-LRx | Wainua® | Wainzua®

eplontersen is an approved drug (FDA (2023), EMA & UK MHRA (2024))

Compound class: Nucleic acid

Comment: Eplontersen is a ligand-conjugated transthyretin (TTR)-directed antisense oligonucleotide. It was designed to reduce the hepatic expression of transthyretin protein and pathological TTR amyloid accumulation in patients with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN).

Full sequence as provided in the agent's INN record is: all-P-ambo-5'-O-(28-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]-16,16-bis{[3-({6-[(2-acetamido-2-deoxy-β-D -galactopyranosyl)oxy]hexyl}amino)-3-oxopropoxy]methyl}- 1-hydroxy-1,10,14,21-tetraoxo-2,18-dioxa-9,15,22-triaza-1λ 5-phosphaoctacosan-1-yl)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methylcytidylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyluridylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyluridylyl-(3'→5')-2'-O-(2-methoxyethyl)guanylyl-(3'→5')-2'-deoxy-P-thioguanylyl-(3'→5')-P-thiothymidylyl-(3'→5')-P-thiothymidylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-2'-deoxy-5-methyl-P-thiocytidylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-P-thiothymidylyl-(3'→5')-2'-deoxy-P-thioguanylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-2'-O-(2-methoxyethyl)adenylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyluridylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'→5')-2'-O-(2- methoxyethyl)-5-methylcytidine, but we have been unable to resolve this to a SMILES or HELM notation.