Stemline Therapeutics Announces SL-701 Granted Orphan Drug Designation for the Treatment of Glioma

January 30, 2015 at 12:00 AM EST

NEW YORK, Jan. 30, 2015 (GLOBE NEWSWIRE) -- Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing oncology therapeutics targeting cancer stem cells (CSCs) and tumor bulk, announced today that SL-701 has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of glioma.

SL-701 is an enhanced immunotherapy designed to activate the immune system to kill malignant gliomas, which are aggressive malignancies of the brain that arise in both adults and children. A multicenter Phase 2 clinical trial is currently evaluating the antitumor activity of SL-701 in adult patients with second-line glioblastoma multiforme (GBM), a particularly aggressive type of glioma. An earlier version of the therapy demonstrated clinical activity, including durable complete responses (CRs) and partial responses (PRs) as well as prolonged disease stabilizations and an overall survival signal, in both adults and children with malignant glioma.

"We are pleased with the FDA's decision to grant Orphan Drug designation to SL-701 as it provides Stemline with a number of benefits through development and commercialization of this novel therapy," noted Eric K. Rowinsky, M.D., Stemline's Chief Medical Officer and Head of Research and Development. He continued, "GBM is a highly aggressive disease with few effective treatment options and remains a major unmet need. Instead of targeting a single component of the cancer, SL-701 is designed to activate and direct the immune system against multiple targets overexpressed on glioma cells. We are very pleased about the high level of interest in the study from patients and clinicians alike."

About Orphan Drug Designation

Orphan Drug designation is granted by the FDA's Office of Orphan Products Development for drugs that are expected to provide significant therapeutic advantage over existing treatments and that target conditions affecting 200,000 or fewer U.S. patients annually. Orphan Drug designation qualifies a company for several benefits under the Orphan Drug Act of 1983. The benefits apply across all stages of drug development and include accelerated approval process; seven years of market exclusivity following marketing approval; tax credits on U.S. clinical trials; eligibility for Orphan Drug grants; and waiver of Prescription Drug User Fee Act (PDUFA) and certain other administrative fees.

About Stemline Therapeutics

Stemline Therapeutics, Inc. is a clinical stage biopharmaceutical company developing novel therapeutics that target both cancer stem cells (CSCs) and tumor bulk. Stemline is currently developing two clinical stage product candidates, SL-401 and SL-701, and a pipeline of preclinical candidates including SL-801 and SL-501. SL-401 is a targeted therapy directed to the interleukin-3 receptor (IL-3R) present on CSCs and tumor bulk of a wide range of hematologic cancers. Three multicenter clinical trials with SL-401 are currently open in the following indications: 1) blastic plasmacytoid dendritic cell neoplasm (BPDCN) and relapsed/refractory acute myeloid leukemia (AML), the expansion stage of which may serve as a registration trial for BPDCN; 2) AML patients in first complete remission (CR) with minimal residual disease (MRD); and 3) four types of advanced high-risk myeloproliferative neoplasms (MPN), including systemic mastocytosis, advanced symptomatic hypereosinophilic disorder, myelofibrosis, and chronic myelomonocytic leukemia. Additional SL-401 studies are currently planned in myeloma and certain other lymphomas and leukemias. SL-701 is an enhanced immunotherapy designed to activate the immune system to attack tumors. A multicenter clinical trial with SL-701 is currently open and accruing adult patients with glioblastoma multiforme (GBM) in first recurrence. Additional SL-701 studies are currently planned in pediatric high-grade glioma. Preclinical candidates being advanced toward investigational new drug (IND) filings include SL-801, a novel, oral small molecule reversible inhibitor of XPO1, being developed for solid and hematologic cancers, and SL-501, a next generation IL-3R targeted therapy, being developed for oncologic and certain autoimmune indications. For more information about Stemline Therapeutics, visit

Forward-Looking Statements

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: statements regarding the proposed public offering and the intended use of proceeds from the proposed offering; the success and timing of our preclinical studies and clinical trials, including site initiation, internal review board approval, scientific review committee approval, and patient accrual; our plans to develop and commercialize our product candidates; our available cash; our ability to obtain and maintain intellectual property protection for our product candidates; the ability of our product candidates to successfully perform in clinical trials; our ability to manufacture; the performance of third-party manufacturers, clinical research organizations, clinical trial sponsors and clinical trial investigators; and other risk factors identified from time to time in our reports filed with the Securities and Exchange Commission. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not intend to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof.

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