The FDA has granted a priority review designation to a biologics license application (BLA) for SL-401 (tagraxofusp, Elzonris) for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), according to Stemline Therapeutics, the developer of the novel stemness inhibitor.
BPDCN affects mostly elderly patients, who may not be able to tolerate chemotherapies. SL-401 may have a better safety and toxicity profile for this patient population due to its targeted mechanism of action. The drug is an IL-3 conjugated truncated diphtheria toxin, which essentially delivers diphtheria toxin to cells that have the IL-3 receptor. Many hematologic malignancies have CD123 expression, but BPDCN in particular has characteristically high expression. When the diphtheria toxin penetrates a cell, it binds to and ADP-ribosylates elongation factor 2, a factor necessary for protein translation. When the protein synthesis process is inhibited, the cell dies by apoptosis.
- Pemmaraju N, Sweet KL, Lane AA, et al. Ongoing phase 2 clinical trial of SL-401 in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN): stage 1 and stage 2 results. Poster presented at: the 59th American Society of Hematology Annual Meeting; December 9-12, 2017; Atlanta, GA. http://www.stemline.com/ScientifcPresDownloads/SL-401/BPDCN%20Pivotal%20Results%20ASH%202017.pdf. Accessed April 9, 2018.
- Stemline Therapeutics presents detailed SL-401 pivotal data in BPDCN at ASH and kicks off its BPDCN awareness campaign; updated results from ongoing trials in additional malignancies also presented [press release]. New York, NY: Stemline Therapeutics, Inc; December 13, 2017. http://ir.stemline.com/news-releases/news-release-details/stemline-therapeutics-presents-detailed-sl-401-pivotal-data. Accessed April 4, 2018.
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