SL-701

February 2, 2017 By Mark Terry , BioSpace.com Breaking News Staff New York-based Stemline Therapeutics seems to be dancing a fine line with how it is releasing negative details about its clinical trials. Adam Feuerstein , writing for TheStreet, notes that one day before a $45 million stock offering, a patient died from a severe side effect related to its drug SL-401—and that was the third patient death with the same cause. Stemline Therapeutics is a clinical stage drug company with three clinical stage product candidates, SL-401, SL-801, and SL-701. SL-401 is directed to the interleukin-3 receptor (CD123) observed on numerous malignancies. On December 5, 2016, the company gave an oral presentation of positive clinical data of its Phase II trial of SL-401 in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). It was given at the 2016 American Society of Hematology (ASH) Annual Meeting in San Diego. The results showed a 100 percent (16/16) overall response rate, as well as an 81 percent (13/16) complete response rate in first-line BPDCN patients treated at the recommended dose of 12 ug/kg/day, and a 95 percent (18/19) ORR in first-line patients treated at the recommended dose. Naveen Pemmaraju , assistant professor, Department of Leukemia at the University of Texas MD Anderson Cancer Center in Houston, TX, an investigator in the study, said in a statement, “The encouraging results generated to date with SL-401 continue to indicate the agent could very well emerge as the standard of care for both first-line and relapsed/refractory BPDCN, a devastating disease for which there had previously been no effective therapy.” On January 20, 2017, the company announced the pricing of an underwritten public offering of 4.5 million shares of its common stock at $10 per share, with expected proceeds to hit $45 million. But, per Feuerstein, the day before the financing, a patient in the trial died from capillary leak syndrome. It was the third death due to capillary leak syndrome, and hasn’t been reported by the company, but was confirmed by a member of the patient’s family. He writes, it “is potentially troubling because it occurred after Stemline had already increased safety monitoring and added new dosing rules to reduce the incidence and severity of the side effect.” Capillary leak syndrome involves large amounts of blood fluid leaking into the body cavity from blood vessels. The results are swelling and dramatic decrease in blood pressure that can lead to organ failure and death. It is very rare. Feuerstein writes, “To date, SL-401 has demonstrated robust overall tumor response rates of 84%, including 56% complete or near-complete response in patients enrolled in its clinical trial. But the drug is also now tied to three patient deaths.” The company is working to finish enrollment totaling about 50 patients in the Phase II study by the end of this quarter. Stemline hopes to use the result of the trial for a marketing application to the U.S. Food and Drug Administration (FDA) by the end of the year. When asked about the death of the BPDCN patient on January 18, Ken Hoberman , Stemline’s chief operating officer, stated, “We are not in a position to comment about any specific outcomes that may or may not have occurred in any of our existing trials. As you know, in any trial of an experimental agent for patients with advanced cancer, patient deaths often occur. When deaths occur in a trial, then careful analysis must be done to understand probable causes and relation, if any, of the death to the use of the experimental product. It would be inappropriate for Stemline to comment on the death of any patient or patients in a trial, including any trial of SL-401, until such an analysis has been conducted, has concluded, and has yielded any information that should be shared publicly.” It’s the timing of the death and the public offering that causes the problem. According to filings with the U.S. Securities and Exchange Commission (SEC) , one BPDCN patient died from capillary leak syndrome. A second patient died, who was on the drug for advanced acute myeloid leukemia, apparently of the same diagnosis. The third patient, whose sister spoke with TheStreet, received the first two doses of SL-401 on January 12 and 13. Feuerstein writes, “Her third daily infusion was postponed because of deteriorating health due to side effects. On January 17, the patient was diagnosed with capillary leak syndrome. She died the next day, never having received three of the scheduled five doses of SL-401 in the initial treatment cycle of the clinical trial.” Stemline had implemented more safety precautions after a dose-ranging stage of the Phase II trial to cut the risk of capillary leak syndrome, and the data presented as ASH didn’t report severe capillary leak syndrome. “But,” Feuerstein writes, “that clean safety streak ended with the death of the BPDCN patient on January 18, raising concerns that Stemline may not have the risk of fatal capillary leak syndrome under control.” Stemline is currently trading for $9.75.

NEW YORK, Nov. 12, 2014 (GLOBE NEWSWIRE) -- Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical stage biopharmaceutical company developing novel drugs targeting cancer stem cells (CSCs) and tumor bulk, today reported financial results for the quarter ended September 30, 2014 and provided a corporate update. Ivan Bergstein, M.D., Stemline's Chief Executive Officer, commented, "This third quarter marked an extremely productive period for Stemline as we initiated and are actively enrolling patients in trials with SL-401 in BPDCN and relapsed/refractory AML, as well as with SL-701 in adult brain cancer. We've also recently opened another study with SL-401 in an earlier stage of AML." He continued, "We anticipate significant upcoming milestones, with trial updates expected during 1H15 and throughout 2015, as we continue to pursue our objective of building a leading commercial-stage biopharmaceutical company." Business Highlights Actively enrolling patients in SL-401 trial of BPDCN and relapsed/refractory AML. During the quarter, Stemline treated its first patients and is actively enrolling in a multicenter, open-label trial of SL-401 in blastic plasmacytoid dendritic cell neoplasm (BPDCN) and relapsed/refractory acute myeloid leukemia (AML). Target enrollment is approximately 60 patients, and includes a brief lead-in that transitions into a larger expansion stage for each indication. Opened additional SL-401 trial. During the quarter, Stemline opened another SL-401 trial. This open-label multicenter trial will enroll patients with AML who are in first complete remission (CR) following their first course of induction chemotherapy, but display evidence of minimal residual disease (MRD) in their bone marrow and remain at high risk for relapse. Target enrollment is approximately 33 patients, and includes a brief lead-in that transitions into a larger expansion stage in this indication. Actively enrolling patients in SL-701 trial of adult second-line GBM. Stemline continues to actively enroll and treat patients in its multicenter, open-label study of SL-701 in adult second-line glioblastoma multiforme (GBM). Target enrollment is approximately 100 patients. Additional SL-401 trials planned in other IL-3R+ cancers. Stemline is planning additional SL-401 trials in other malignancies including several uncommon stem cell-derived myeloproliferative disorders. Abstracts accepted at upcoming leading oncology conferences. Stemline scientific abstracts were accepted for presentation at the Society for Neuro-Oncology (SNO) (SL-701 clinical trial design); the San Antonio Breast Cancer Symposium (StemScreen drug discovery platform in breast cancer); and the American Society of Hematology (ASH) (preclinical data from SL-401 in IL-3R+ cancers including myeloma, SL-501 in Hodgkin's and Non-Hodgkin's lymphoma, and SL-101 in AML). Further expanded intellectual property portfolio. A recent addition to Stemline's patent estate around the therapeutic targeting of IL-3R (CD123) was granted in the United States. This U.S Patent covers the use of naked monoclonal antibodies that target CD123 in AML. This patent joins additional Stemline issued patents from its family in the U.S and abroad that cover both naked antibodies as well as antibody-drug conjugates (ADCs) directed to CD123 as a method of treating certain hematologic diseases. In addition, Stemline has other broad patents around therapeutic antibodies to cancer stem cell (CSC) cell surface targets in general, as well as patents around its specific IL-3R-targeted agents, SL-401 and SL-501. Third Quarter 2014 Financial Results Review Stemline ended the third quarter of 2014 with $65.0 million in cash, cash equivalents and investments as compared to $69.5 million as of June 30, 2014, which reflects a cash burn of $4.5 million for the quarter. Existing cash, cash equivalents and investments are expected to fund operations for at least the next two years. The company ended the third quarter of 2014 with 13.3 million shares outstanding. For the third quarter of 2014, Stemline had a net loss of $6.9 million, or $0.53 per share, compared with a net loss of $5.6 million, or $0.45 per share, for the same period in 2013. The net loss for the first nine months of 2014 was $21.9 million, or $1.69 per share, as compared with a net loss of $16.5 million, or $1.74 per share, for the nine-month period ended September 30, 2013. Research and development expense was $5.0 million for the third quarter of 2014, an increase of $1.7 million or 50% compared with $3.3 million for the same period in 2013. The higher costs were primarily attributable to the ramp up in development activities for the SL-401 and SL-701 clinical programs which included manufacturing and regulatory activities coupled with the initiation and execution of clinical trials as compared to the same period in the prior year. General and administrative expense was $2.0 million for the third quarter of 2014, which reflects a decrease of $0.3 million, or 11% compared with $2.3 million for the same period in 2013. This decrease in costs was primarily attributable to a reduction in consulting and advisory 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. SL-401 is a targeted therapy directed to the interleukin-3 receptor (IL-3R). A multicenter clinical trial with SL-401 is currently open and accruing patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) and advanced acute myeloid leukemia (AML). An additional clinical trial with SL-401 is currently open in patients with AML who are in first complete remission (CR) with minimal residual disease (MRD), which is associated with a high relapse rate. Stemline is also planning SL-401 trials in additional hematologic cancers including several uncommon, stem cell-derived myeloproliferative disorders. Previously, SL-401 demonstrated single-agent activity, including multiple durable complete responses (CRs), in a Phase 1/2 trial in several indications including BPDCN and relapsed/refractory AML. 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. Previously, an earlier version of the therapy demonstrated clinical activity, including durable CRs and partial responses (PRs), in Phase 1/2 trials in adults and children with advanced brain cancers. 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: 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. Table 1. Stemline Therapeutics, Inc. - Balance Sheets September 30, 2014 December 31, (Unaudited) 2013 Assets Current assets: Cash and cash equivalents $ 28,489,426 $ 44,200,420 Short-term investments 16,002,407 — Related party receivable — 199,615 Prepaid expenses and other current assets 735,896 292,916 Total current assets 45,227,729 44,692,951 Furniture and fixtures, net 268,333 383,333 Long-term investments 20,531,546 40,204,912 Total assets $ 66,027,608 $ 85,281,196 Liabilities and stockholders' equity Current liabilities: Accounts payable and accrued expenses $ 4,399,103 $ 5,013,808 Total current liabilities 4,399,103 5,013,808 Deferred grant revenue 429,428 643,000 Total liabilities 4,828,531 5,656,808 Stockholders' equity: Preferred stock $0.0001 par value, 5,000,000 shares authorized, none issued and outstanding at September 30, 2014 and December 31, 2013 — — Common stock $0.0001 par value, 33,750,000 shares authorized at September 30, 2014 and December 31, 2013, 13,270,232 shares issued and outstanding at September 30, 2014 and 13,095,726 shares issued and outstanding at December 31, 2013 1,327 1,310 Additional paid-in capital 114,445,189 111,032,619 Accumulated other comprehensive gain (loss) 16,108 (43,775) Accumulated deficit (53,263,547) (31,365,766) Total stockholders' equity 61,199,077 79,624,388 Total liabilities and stockholders' equity $ 66,027,608 $ 85,281,196 Table 2. Stemline Therapeutics, Inc. - Statements of Operations (Unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2014 2013 2014 2013 Revenues: Grant revenue $71,429 — $213,858 — Operating expenses: Research and development 4,979,291 $ 3,323,662 16,186,735 $ 10,569,909 General and administrative 2,008,957 2,253,652 6,024,182 5,492,545 Total operating expenses 6,988,248 5,577,314 22,210,917 16,062,454 Loss from operations (6,916,819) (5,577,314) (21,997,059) (16,062,454) Other income 2,810 — 3,443 30,649 Other expense (11,284) — (30,354) — Interest expense — — — (505,037) Interest income 40,136 3,790 126,189 7,034 Net loss from operations (6,885,157) (5,573,524) (21,897,781) (16,529,808) Net loss attributable to common stockholders $ (6,885,157) $ (5,573,524) $ (21,897,781) $ (16,529,808) Net loss attributable to common stockholders per common share: Basic and Diluted $ (0.53) $ (0.45) $ (1.69) $ (1.74) Weighted-average shares outstanding: Basic and Diluted 12,950,027 12,471,472 12,922,117 9,520,179 CONTACT: Investor Relations Stemline Therapeutics, Inc. 750 Lexington Avenue Eleventh Floor New York, NY 10022 Tel: 646-502-2307 Email: investorrelations@stemline.com Help employers find you! 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