On March 23, 2017 Therapeutics, Inc. (Nasdaq: STML), a clinical-stage biopharmaceutical company developing novel therapeuticsfor oncology indications of unmet medical need, reported that enrollment of Stage 3 in the SL-401 pivotal trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN) has been completed (Press release, Stemline Therapeutics, MAR 23, 2017, View Source [SID1234518259]). The company also reviewed key milestones for the SL-401 program over the coming year. Schedule your 30 min Free 1stOncology Demo! Completes Stage 3 Enrollment
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Stemline has completed enrollment of the Stage 3 cohort of the SL-401 pivotal trial in BPDCN. Stage 3 enrolled 13 first-line BPDCN patients; statistical analysis will be based on evaluable first-line patients. The registration pathway was previously agreed upon with the U.S. Food and Drug Adminstration (FDA). Depending on the data from the trial, Stemline plans to use the results generated to support the potential filing of a Biologics License Application (BLA) for full approval in first-line BPDCN, and is targeting possible BLA filing in the second half of this year.
This multicenter, pivotal trial has enrolled 47 BPDCN patients at seven centers in the U.S. The 47 BPDCN patients were comprised of 32 first-line patients (29 dosed at 12 ug/kg/day) and 15 relapsed/refractory patients dosed at 12 ug/kg/day. Stemline plans to provide a clinical update on patients enrolled in Stages 1 and 2 at a medical conference around mid-year, with top-line data from Stage 3 expected in the second half of 2017. To ensure ongoing patient access to SL-401, Stemline plans to continue to enroll both first-line and relapsed/refractory
BPDCN patients under the current protocol.
SL-401 received Breakthrough Therapy Designation (BTD) by the FDA in August 2016.
BLA Preparation and Pre-Commercial Activities Underway
Stemline’s clinical, preclinical, manufacturing, and regulatory teams are working toward a timely and comprehensive potential BLA filing. Ongoing efforts include compiling the necessary supportive data and assembling the BLA modules, including clinical, clinical pharmacology, non-clinical, and CMC (chemistry, manufacturing, and controls). In parallel, the commercial team is working to define the BPDCN market landscape, including factors related to patient flow, market access and pricing considerations, all with an eye toward setting the stage for a successful launch of SL-401, if approved.
"I am very proud of the entire Stemline team and our clinical investigators, who helped to make this key milestone possible," said Ivan Bergstein, M.D., Chief Executive Officer of Stemline. Dr. Bergstein, continued "Completing enrollment in our pivotal trial is an important step toward our goal of bringing to market a novel treatment for patients suffering from BPDCN, a devastating disease. We continue to focus on following patients, data collection, and advancing our BLA-targeted efforts on all fronts."