On August 9, 2018 Stemline Therapeutics, Inc. (Nasdaq: STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, reported its financial results for the quarter ended June 30, 2018 (Press release, Stemline Therapeutics, AUG 9, 2018, View Source [SID1234528708]). The Company also reviewed recent clinical and regulatory events, and outlined key upcoming milestones:

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ELZONRIS (SL-401, tagraxofusp) – BLA submission completed

We completed submission of a rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for ELZONRIS, which has been granted breakthrough therapy designation (BTD), for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). If successful, we anticipate acceptance of our BLA within 60 days of submission (i.e. in the coming weeks) and potential U.S. marketing approval by 1Q19, or possibly sooner.

Data from our pivotal trial in patients with BPDCN was the subject of an oral presentation at the 23rdCongress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Stockholm, Sweden.

We anticipate feedback from the European Medicines Agency (EMA) later this year regarding a potential ELZONRIS regulatory filing in Europe.

In preparation for potential marketing approval, we continue to build out our pre-launch and commercial activities, including our disease awareness campaign targeting key stakeholders including hematologist-oncologists, dermatologists, and pathologists.
ELZONRIS – Other potential indications

ELZONRIS is also being evaluated in clinical trials in additional indications including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF).

Clinical data from ongoing trials in patients with CMML and MF were selected for presentation at EHA (Free EHA Whitepaper) in June.

In relapsed/refractory CMML (n=16 patients), ELZONRIS demonstrated 100% (8/8) spleen responses in evaluable patients with baseline splenomegaly by physical exam and 2 bone marrow complete responses (BMCRs), coupled with a tolerable safety profile. Given the results observed to date, we are currently formulating registrational trial designs.

In relapsed/refractory MF (n=15 patients), ELZONRIS demonstrated 50% (6/12) spleen responses in evaluable patients with baseline splenomegaly (>5 cm palpable below the costal margin by physical exam), coupled with a tolerable safety profile. Given the results observed to date, we are currently evaluating next steps including possible registrational trial designs.
SL-801

Data from the ongoing Phase 1 trial of SL-801 in patients with advanced solid tumors were presented at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June. Patient enrollment and dose escalation continues.
SL-701

Data from the Phase 2 trial of SL-701 in patients with second-line glioblastoma (GBM) were presented at the 2018 ASCO (Free ASCO Whitepaper) meeting in June. Notably, there were long-term (>12 month) overall survivors in the SL-701+bevacizumab cohort which consisted primarily of patients who demonstrated an elevated immune response (i.e. potentially representing an "immunocompetent" population). Further analyses, including registration-directed designs are ongoing.
Ivan Bergstein, MD, CEO of Stemline, commented, "We have completed our rolling BLA submission and are quickly approaching the very important milestone of a potential BLA acceptance for filing by the FDA. We continue to advance closer to our ultimate goal of potentially making ELZONRIS widely available to patients with BPDCN. In parallel, ELZONRIS continues to generate very promising clinical data in additional indications including CMML and MF, two settings of unmet medical need for which we are actively evaluating registration pathways. Additionally, we continue to expand our commercial infrastructure including the build out of our sales and marketing teams ahead of our potential approval."

Second Quarter 2018 Financial Results Review
Stemline ended the second quarter of 2018 with $97.1 million in cash, cash equivalents and investments, with a cash burn of $17.6 million in the second quarter. The Company ended the second quarter of 2018 with 30.9 million shares outstanding. For the second quarter of 2018, Stemline had a net loss of $18.9 million, or $0.66 per share, compared with a net loss of $15.5 million, or $0.66 per share, for the same period in 2017.

Research and development expense was $11.2 million for the quarter ended June 30, 2018, compared with $11.5 million for the quarter ended June 30, 2017, representing a decrease of $0.3 million.

General and administrative expense was $8.6 million for the quarter ended June 30, 2018, compared with $4.5 million for the quarter ended June 30, 2017, representing an increase of $4.1 million. The increase in expense was primarily attributed to a $3.0 million increase in pre-launch expenses in support of our potential commercialization of ELZONRIS in BPDCN, if marketing approval from the FDA is received. Additionally, the higher expense was also due to an increase of non-cash stock-based compensation expense and increased headcount.