Stemline Therapeutics has in-licensed the rights to SL-401, a compound directed at a key cancer stem cell target and used in the treatment of acute myeloid leukemia.
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SL-401 has demonstrated single agent anti-tumor activity in a phase I dose escalation study. Thirty adult patients with relapsed, refractory, or poor risk acute myeloid leukemia (AML) were treated with escalating doses of SL-401 without dose limiting toxicity.
Anti-tumor activity has been observed, including one durable complete response in a patient refractory to standard chemotherapy, two partial responses, and three minor responses.
SL-401 targets the interleukin-3 receptor (IL-3R) and has been shown to impair the ability of cancer stem cells to form tumors. IL-3R is over-expressed on multiple hematological cancers. Importantly, in AML, IL-3R is over-expressed on both leukemia blasts as well as leukemia cancer stem cells. Cancer stem cells are believed to be a major contributor to treatment failure, relapse, and death from malignancy.
“SL-401 constitutes the first concerted clinical effort to target leukemia stem cells,” stated Dr David Rizzieri of Duke University Medical Center.
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