Drug intelligence / Profile preview

lefitolimod

Development stage
Phase 3
Lead developer
Mologen
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Vaccines & Immunotherapeutics
Administration
Subcutaneous
01

Overview

Lefitolimod is a synthetic oligonucleotide and investigational immunotherapy that acts as a toll-like receptor 9 (TLR9) agonist. It belongs to the family of double-stem loop immunomodulators (dSLIM). Lefitolimod binds to and activates TLR9 on plasmacytoid dendritic cells and B cells, triggering innate immune responses including secretion of type I interferons such as IFN-alpha. This activation leads to downstream stimulation of natural killer cells, cytotoxic T lymphocytes, monocytes, and B cells. The drug has been studied as a latency-reversing agent in HIV infection—aiming to reactivate latent virus reservoirs—and as an immunotherapeutic agent in various cancers including small cell lung cancer and colorectal cancer. Lefitolimod is being developed by Mologen for these indications[1][2][5][7].

Other names
TLR9 agonist MGN1703TLR-9 agonist MGN1703TLR 9 agonist MGN1703double-stem loop immunomodulator (dSLIM)
02

Targets

TLR9 (Toll-like receptor 9)

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