Stemline's SL-401 receives FDA Orphan Drug designation for treatment of acute myeloid leukemia

Stemline Therapeutics, Inc. today announced that SL-401, the Company's lead compound, has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML).

SL-401 has completed a multi-center Phase I/II clinical trial in AML where it has demonstrated single agent efficacy, including two durable complete responses (CRs), multiple blast reductions and disease stabilizations, and an overall survival (OS) benefit in heavily pre-treated patients. SL-401 was well-tolerated and bone marrow-sparing. The trial results were presented at the 52nd Annual Meeting of the American Society of Hematology (ASH) in December 2010. SL-401 is now poised for Phase III clinical trials in patients with advanced AML.

Stemline's CEO, Ivan Bergstein, MD, noted, "Receipt of Orphan Drug designation represents another key milestone in the advancement of SL-401 through the regulatory process. We are also very encouraged by the potential of SL-401 to benefit patients with advanced stage AML, an unmet medical need, as well as additional hematologic malignancies including MDS, CML and potentially certain lymphomas."