OTX-2002 + checkpoint inhibitor

**OTX-2002 + checkpoint inhibitor** refers to the investigational combination therapy of OTX-2002 (a first-in-class MYC-targeted Epigenomic Controller) with an immune checkpoint inhibitor for the treatment of cancers, particularly hepatocellular carcinoma (HCC). OTX-2002 is a programmable mRNA drug encapsulated in a lipid nanoparticle for intravenous administration, designed to downregulate MYC gene expression pre-transcriptionally through targeted epigenomic modulation. Checkpoint inhibitors are immunotherapies that block inhibitory pathways in T cells, thereby enhancing antitumor immune responses, most commonly through targets such as PD-1, PD-L1, or CTLA-4. Preclinical and early clinical studies demonstrate that OTX-2002 can induce rapid and durable downregulation of MYC and increase tumor cell apoptosis, and ongoing trials include cohorts evaluating its combination with checkpoint inhibitors for enhanced efficacy[1][2][3][4][5][7]. The combination is being studied for patients with relapsed/refractory HCC and other MYC-driven solid tumors.

Other names

OTX-2002 + checkpoint inhibitor