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RGX-104 + nivolumab is a combination investigational therapy being evaluated for the treatment of advanced solid tumors and lymphomas, including lung, neuroendocrine, ovarian, renal, bladder, and triple-negative breast cancer. RGX-104 is an orally administered small molecule agonist of the Liver X Receptor (LXR) that induces transcriptional activation of the ApoE gene, depletes immunosuppressive myeloid-derived suppressor cells (MDSCs), activates dendritic cells, and stimulates cytotoxic T-lymphocytes, thereby promoting anti-tumor immunity. Nivolumab is a monoclonal antibody immunotherapy targeting PD-1, an immune checkpoint, thereby enhancing T-cell mediated anti-tumor response. The rationale for combining RGX-104 with nivolumab is based on preclinical and early clinical findings that RGX-104 can overcome immunosuppressive mechanisms and resistance to checkpoint inhibitor therapy by depleting MDSCs and activating immune cells, which may enhance the efficacy of nivolumab's checkpoint blockade. The combination is currently in phase 1 clinical trials for various advanced malignancies[1][2][4][5][7]. RGX-104 is developed by Inspirna (formerly RGENIX). Nivolumab is developed by Bristol Myers Squibb.
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