Blastic plasmocytoid dendritic cell neoplasm (BPDCN) is a rare hematological malignancy typically associated with poor patient outcomes. Naveen Pemmaraju, MD, of the University of Texas MD Anderson Cancer Center, Houston, TX, presented results from an ongoing clinical trial (NCT02268253) exploring the use of SL-401, an agent that targets CD123, for the treatment of BPDCN at the 23rd Congress of the European Haematology Association (EHA) 2018, held in Stockholm, Sweden. Dr Pemmaraju gives an overview of the extremely positive overall response rates in both the frontline and relapsed/refractory settings, as well as listing the side effects of the therapy. Finally, he discusses the potential for combining SL-401 with other agents in the future; for example, hypomethylating drugs, other targeted agents, or cytotoxic therapy.