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Etoposide toniribate is a small molecule investigational anticancer agent and a derivative of etoposide designed to improve pharmacological properties and efficacy. It acts primarily as a DNA topoisomerase II inhibitor—disrupting the enzyme’s ability to manage DNA topology during replication and transcription—which leads to DNA strand breaks and apoptosis in rapidly dividing cancer cells[3][4]. It may also have activity as a carbonic anhydrase stimulant[4]. Etoposide toniribate has been developed for intravenous administration in oncology settings. Its main clinical focus is on relapsed or refractory biliary tract cancer (BTC), where it has received orphan drug designation from the FDA based on phase II trial data showing improved disease control rates compared with best supportive care[3][4]. The drug was originally developed by the Institute for Medical Immunology Charite - Universitatsmedizin Berlin and further advanced by CellAct Pharma/Mundipharma/Imbrium Therapeutics[3][4].
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