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Vidutolimod (CMP-001) is a Toll-like receptor 9 (TLR9) agonist consisting of a CpG-A oligodeoxynucleotide encapsulated within a virus-like particle (VLP) derived from the bacteriophage Qβ. In a Phase 1 clinical trial (CA209-991) sponsored by Sheba Medical Center, vidutolimod was evaluated as part of a combination immunotherapy regimen alongside the PD-1 inhibitor nivolumab and the CTLA-4 inhibitor ipilimumab, supplemented by liver radiosurgery. This multi-modal approach was designed to treat microsatellite stable (MSS) or mismatch repair proficient (MMR-P) metastatic colorectal cancer with liver metastases by stimulating the innate immune system to convert "cold" tumors into "hot," immune-responsive environments. However, the study found that the combination lacked efficacy in this specific patient population and was associated with significant hepatic toxicity, particularly when liver irradiation was juxtaposed with intratumoral vidutolimod injection. Vidutolimod was developed by Checkmate Pharmaceuticals, which was later acquired by Regeneron.
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