CPX-351: From preclinical studies to future directions.

For decades, the standard induction treatment for patients with acute myeloid leukemia (AML) has been the 7 + 3 regimen (cytarabine plus daunorubicin). The US-Food and Drug Administration (FDA) approved, in August 2017, the use CPX-351 for adults with newly diagnosed secondary AML. CPX-351 (also known as VYXEOS) is a dual-drug liposomal encapsulation of cytarabine and daunorubicin in a synergistic 5:1 M ratio, and CombiPlex was utilized, a combination drug technology platform. In this review, we analyze the path from preclinical studies to drug registration. Recent research highlights intestinal and heart toxicity of the drug, as well as current and potential future uses for CPX-351.