Stemline Therapeutics' SL-401 has won orphan drug designation from the Food and Drug Administration (FDA) for the treatment of aggressive hematologic malignancy blastic plasmacytoid dendritic cell neoplasm (BPDCN).
With an orphan drug status for the treatment of acute myeloid leukemia (AML), the novel targeted therapy is directed to the interleukin-3 receptor (IL-3R) present on tumor bulk and cancer stem cells (CSCs) of multiple hematologic cancer indications.
Stemline research and development head and chief medical officer Eric Rowinsky said SL-401 has the potential to treat IL-3R-expressing hematologic malignancies.
"SL-401 is demonstrating robust clinical activity in heavily-pretreated patients with BPDCN who are refractory to available therapies, including high-dose chemotherapy and allogeneic stem cell transplantation," Rowinsky added.
"This Orphan Drug designation provides us with a number of benefits that further strengthen our SL-401 program."
In patients with advanced hematologic cancers, such as BPDCN, AML, and myelodysplastic syndrome (MDS), SL-401 has established single agent clinical activity.
The clinical-stage biopharmaceutical company plans to advance the product into pivotal trials in BPDCN and AML to continue evaluating SL-401 for additional indications.