Cancer is one of the leading causes of death worldwide and there is significant unmet medical need for new, highly effective therapies.
Pyrazine-2-carboxylic acid N'-(7-fluoro-pyrrolo[1,2-alpha]quinoxalin-4-yl)-hydrazide-oxalic acid co-crystal, designated NVX-412, was identified as a promising drug candidate for the treatment of various cancer types due to its strong anti-neoplastic activity in preliminary assays. The findings of my PhD thesis for the first time provide an important basic understanding of the mechanisms of action of NVX-412. I could show that NVX-412 exerts its anti-neoplastic effects in a p53-independent manner in a broad range of tumor cell lines of various histology. My results suggest a bi-modal mode of action of NVX-412, which is predominantly cytostatic at lower and primarily cytotoxic at higher concentrations. Taken together, my observations indicate that NVX-412 possesses a clinically useful dose-response relationship and that it holds promise as an effective drug candidate for the treatment of various cancer types.