KSI-3716

KSI-3716 is a **small molecule** inhibitor of **c-MYC**, a transcription factor frequently overexpressed in cancers. It functions by blocking the binding of c-MYC to its partner MAX at target gene promoters, thereby inhibiting c-MYC-mediated transcriptional activity[1][3][5][7]. The inhibition leads to decreased expression of key oncogenic c-MYC target genes, including cyclin D2, CDK4, and hTERT, which are involved in cell cycle progression and proliferation[1][3][5][7]. KSI-3716 induces cell cycle arrest and apoptosis, exhibiting potent cytotoxic effects against bladder cancer cells in preclinical models[1][7]. Intravesical administration in murine models of orthotopic bladder cancer resulted in significant suppression of tumor growth with minimal systemic toxicity[3][5][7]. It is particularly effective against gemcitabine-resistant bladder cancer cells, and sequential treatment with gemcitabine followed by KSI-3716 showed synergistic inhibition of tumor cell proliferation[2][4][6].