CP-4200 represents a new family of LVT drugs for the treatment of cancer and haematological malignancies and is currently in preclinical development.
CP-4200 is a novel LVT derivative of azacytidine, which pre-dominantly does not act as a traditional cytotoxic drug, but rather a drug that modifies gene expression (epigenetic modulator). Epigenetic modulators are intended to restore normal function of genes that control how cells develop and grow in the human body at doses with low cytotoxic activity. VidazaTM (azacitidine) is currently approved in the US for all forms of myelodysplastic syndromes (MDS), while in the EU it is approved for the more severe forms of MDS as well as for certain forms of acute myeloid leukemia (AML).
Sales of VidazaTM (Celgene Corporation) are growing rapidly and in 2010 worldwide sales were more than 500 million USD. The use of epigenetic modulators is expected to expand into many cancer types in the future and thus represents a huge market potential.