Drug intelligence / Profile preview

bazlitoran

Development stage
Phase 2
Lead developer
Aceragen
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules
Administration
Subcutaneous[2]
01

Overview

Bazlitoran is a first-in-class antisense oligonucleotide and investigational drug that acts as an antagonist of endosomal Toll-like receptors (TLRs) 7, 8, and 9. It was originally developed by Idera Pharmaceuticals (now Aceragen) for the treatment of rare hematologic malignancies and autoimmune diseases. Its mechanism involves blocking TLR7/8/9 signaling to reduce inflammatory responses implicated in diseases such as Waldenstrom's macroglobulinemia and diffuse large B cell lymphoma. Bazlitoran has orphan drug status for these indications but development has been suspended or discontinued in several disease areas[1][2][4][7].

Other names
ALL-P-AMBO-2'-DEOXY-P-THIOCYTIDYLYL-(3'->5')-P-THIOTHYMIDYLYL-(3'->5')-2'-DEOXY-P-THIOADENYLYL-(3'->5')-P-THIOTHYMIDYLYL-(3'->5')-2'-DEOXY-P-THIOCYTIDYLYL-(3'->5')-P-THIOTHYMIDYLYL-(3'->5')-2'-O-METHYL-P-THIOGUANYLYL-(3'->5')-2'-O-METHYL-P-THIOURIDYLYL-UNII 2U46M95B5MUNII2U46M95B5MUNII-2U46M95B5MCAS 1378549‑07‑5Bazlitoran sodium
02

Targets

TLR7 (Toll-like receptor 7)TLR8 (Toll-like receptor 8)TLR9 (Toll-like receptor 9)

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