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CPSI-1306 is an orally available small-molecule inhibitor of macrophage migration inhibitory factor (MIF), a proinflammatory cytokine implicated in chronic inflammatory diseases and various malignancies, including cutaneous squamous cell carcinoma, head and neck squamous cell carcinoma, triple negative breast cancer, and bladder cancer. CPSI-1306 acts by disrupting the homotrimerization of MIF, thereby antagonizing its biological activity. In preclinical models, systemic administration of CPSI-1306 has been shown to reduce UVB-induced skin inflammation and tumorigenesis by increasing keratinocyte apoptosis, enhancing DNA repair mechanisms (such as cyclobutane pyrimidine dimer repair), decreasing proliferation markers (Ki67), reducing tumor burden, and slowing progression to invasive squamous cell carcinoma. Additionally, it has demonstrated efficacy in reducing tumor growth in murine models of head and neck squamous cell carcinoma as well as lowering blood glucose levels associated with reduced inflammatory cytokines in diabetes models[1][2][3][4][5][6].
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