Drug intelligence / Profile preview

tilsotolimod

Development stage
Phase 2
Lead developer
Aceragen
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
Administration
Intratumoral
01

Overview

Tilsotolimod is a synthetic oligonucleotide and investigational immunotherapy drug that acts as an agonist of Toll-like receptor 9 (TLR9). It is designed to stimulate the innate and adaptive immune system by activating TLR9, which leads to dendritic cell activation, increased type I interferon production, enhanced cytotoxic T-cell responses, and modulation of the tumor microenvironment. The drug has been primarily developed for use in combination with checkpoint inhibitors for the treatment of advanced melanoma and other refractory solid tumors. Tilsotolimod has demonstrated antitumor activity in preclinical models and early clinical trials but did not show added benefit when combined with ipilimumab in advanced melanoma patients. It received Fast Track Designation from the FDA for anti–PD-1–refractory melanoma in combination with ipilimumab and Orphan Drug Designation for stage IIb-IV melanoma[1][3][5][6][7].

Other names
tilsotolimod sodium
02

Targets

TLR9 (Toll-like receptor 9)

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