Stemline Therapeutics Highlights Recent Clinical and Regulatory Developments and Details Upcoming Milestones following its Annual Shareholder Meeting
- ELZONRIS® clinical data were presented at the 2019
American Society of Clinical Oncology( ASCO) annual meeting in Chicago, Illinoisand the 24th Congressof the European Hematology Association(EHA) in Amsterdam, Netherlands. Presentations highlighted updated results from ongoing Phase 2 clinical trials in chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), as well as final results of the pivotal trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN). In addition, ELZONRIS preclinical results in systemic sclerosis, an autoimmune disorder in which CD123+ plasmacytoid dendritic cells (pDCs) play a role in disease pathogenesis, were presented at the Annual European Congress of Rheumatology(EULAR) in Madrid, Spain.
- As previously announced, an additional single-arm cohort of patients with previously-treated CMML, Stage 3, will be added to the currently enrolling trial. In the first part of Stage 3 (Stage 3a), enrichment strategies and certain efficacy endpoints (including spleen size reduction) will be assessed for inclusion in the confirmatory cohort (Stage 3b), that will aim to provide the primary evidence of efficacy to support potential registration. We expect to open enrollment of the new cohort in 4Q19.
- Stemline continues to build out a European commercial infrastructure in advance of potential approval by the
European Medicines Agency(EMA). A scientific advisory group meeting is planned for September, and the ELZONRIS marketing authorization application (MAA) review will proceed on a standard timeline. We expect an opinion by the Committee for Medicinal Products for Human Use(CHMP) later this year. If successful, Stemline is targeting a commercial launch in Europein 1Q20.
- In an ongoing market expansion effort, corporate and investigator-sponsored clinical trials with ELZONRIS in additional indications are ongoing in myelofibrosis (MF) and acute myeloid leukemia (AML) and others are planned to roll out later this year and next.
- Stemline continues to advance its clinical-stage assets, including SL-801 (a reversible inhibitor of XPO1). XPO1 is a clinically validated target in oncology, and the
FDArecently approved an XPO1 inhibitor in patients with relapsed/refractory multiple myeloma. Stemline is also developing its preclinical assets SL-1001 (RET kinase inhibitor) and SL-901 (kinase inhibitor).
- Stemline will provide further details on commercial and clinical progress during its second quarter financial results teleconference in early August. Call-in details will be provided in advance of the call.
ELZONRIS® (tagraxofusp-erzs), a CD123-directed cytotoxin, is approved by the
BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.
CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin’s lymphoma (HL), and certain Non-Hodgkin’s lymphomas (
Some of the statements included in this press release may be forward-looking statements that involve a number of risks and uncertainties. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. The factors that could cause our actual results to differ materially include: the success of our U.S. launch and commercialization; the success of our MAA submission to the EMA and potential launch in
Source: Stemline Therapeutics, Inc.