- Stemline Therapeutics (NASDAQ:STML) hits its enrollment target in the third and last stage in its Phase 2 clinical trial assessing Breakthrough Therapy-tagged SL-401 in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN).
- The study has enrolled a total of 47 BPDCN patients, 32 first-line and 15 relapsed/refractory. The company plans to provide a clinical update on patients participating in Stages 1 and 2 at a medical conference around mid-year and will release top-line data from Stage 3 in H2. Enrollment will continue in both first-line and relapsed/refractory under the current protocol.
- If all goes well, Stemline plans to file a Biologics License Application (BLA) with the FDA in H2.
- Shares cratered in February on the announcement that three patients in the study died from a side effect called capillary lead syndrome, a condition where large volumes of plasma and other blood components leak into the body cavity.
- SL-401 is a recombinant fusion protein composed of certain domains of the diptheria toxin fused to interleukin 3 (IL3). It is called a targeted therapy because the IL3 seeks out IL3 receptors on cancer stem cells (CSCs), the malignant "seeds" of tumors, and tumor bulk of blood cancers. Binding to the receptors delivers the cytotoxin directly to the cancer cells.
- Previously: Stemline's SL-401 shows treatment effect in mid-stage study in type of leukemia, but three patients die; shares down 35% (Feb. 2)