Stemline have announced that their lead clinical candidate, SL-401, has been granted an orphan drug designation by the FDA for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). BPDCN is a rare and aggressive haematological malignancy for which there are currently no approved treatments.
In a Phase 1/2 trial, SL-401 demonstrated single agent activity against multiple advanced haematological cancers, including BPDCN, Acute Myeloid Leukaemia (AML) and myelodysplastic syndrome (MDS), and the biologic has also previously been granted orphan status for the treatment of AML. The novel biologic works by targeting the interleukin-3 receptor (IL-3R) that is overexpressed on leukaemia blasts and cancer stem cells (CSCs) relative to normal haematopoietic cells. It comprises human IL-3 coupled to a truncated diphtheria toxin payload. “We are focused on the rapid development of SL-401 due to its potential for the treatment of IL-3R-expressing hematologic malignancies,” commented Eric K. Rowinsky, M.D. “”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.”
Stemline say the designation will allow them to further strengthen the SL-401 program, and the company will progress SL-401 into pivotal trials in BPDCN and AML soon. SL-401 is being advanced into a pivotal Phase 2b trial in patients with BPDCN.
BPDCN has also been previously known as blastic NK cell lymphoma and agranular CD4+/CD56+ haematodermic neoplasm.
If you want to know more about innovation and strategy in the orphan drug market, you might be interested in attending the World Orphan Drug Congress Asia, 18-19 June 2013, Singapore, or the 4th World Orphan Drug Congress Europe, in Geneva on the 14th November 2013.