Drug intelligence / Profile preview

vidutolimod + nivolumab

Development stage
Phase 3
Lead developer
Regeneron Pharmaceuticals
Modality
Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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, Monoclonal Antibodies → Antibody-Based Therapeutics
Administration
Intratumoral, Intravenous, Subcutaneous
01

Overview

**Vidutolimod + nivolumab** is a combination investigational immunotherapy for cancer. Vidutolimod is a synthetic DNA-based agonist targeting the Toll-Like Receptor 9 (TLR9), designed to activate plasmacytoid dendritic cells and stimulate innate and adaptive anti-tumor immunity. Nivolumab is a fully human monoclonal antibody (brand name: Opdivo) that blocks the PD-1 receptor on T cells, thus preventing immune evasion by tumor cells via the PD-1/PD-L1 axis. In neoadjuvant clinical trials, particularly for high-risk, resectable stage III cutaneous melanoma, this combination has demonstrated robust anti-tumor immune responses, including a 55% major pathologic response rate. Mechanistically, their synergy is attributed to enhanced infiltration and activation of myeloid cells, plasmacytoid dendritic cells, and CD8+ tumor-infiltrating lymphocytes, setting this combination apart from nivolumab monotherapy. Vidutolimod is not FDA-approved and remains investigational; nivolumab is FDA-approved for various cancers.

Other names
PS20PS-20PS 20
02

Targets

PDCD1 (Programmed cell death protein 1 receptor)TLR9 (Toll-like receptor 9)

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