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.