RB-07-16

RB-07-16 is a C6-substituted pyrazolo[3,4-d]pyrimidine-based bisphosphonate (C6-PyraP-BP) that inhibits human geranylgeranyl pyrophosphate synthase (hGGPPS). It demonstrates antitumor efficacy by selectively engaging intracellular targets, inducing apoptosis, and downregulating prenylation of Rap-1A in multiple myeloma, pancreatic ductal adenocarcinoma, and colorectal cancer cells. In xenograft mouse models of multiple myeloma and pancreatic ductal adenocarcinoma, RB-07-16 significantly reduced tumor growth without substantial hepatotoxicity or elevation of liver enzymes. The compound is metabolically stable across species, does not inhibit major CYP 450 enzymes, and maintains good systemic circulation in rat models. Its mode of action centers on GGPPS inhibition, affecting protein prenylation pathways crucial for tumor proliferation[1].