On November 7, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported financial results and business highlights for the third quarter ended September 30, 2019 (Press release, Stemline Therapeutics, NOV 7, 2019, View Source [SID1234550713]).
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Robert Francomano, SVP and Global Head of Commercial, stated, "We are very pleased with the sustained revenue generation and significant progress we continue to make on the ELZONRIS launch. Notably, we observed a greater than 20% increase in estimated new patient starts quarter over quarter, which is a testament to our execution and bodes very well for the future of the brand. Additionally, we implemented a number of strategies designed to increase the speed and accuracy of BPDCN diagnoses, not only in hematology and hematopathology but also within the dermatology and dermatopathology segments – where our data indicate the preponderance of misdiagnoses occurs. Our entire organization is focused on ensuring that patients with BPDCN gain access to ELZONRIS, and we look to capitalize on the positive trends we are seeing to ensure continued performance through 2020 and beyond."
Ivan Bergstein, M.D., CEO of Stemline Therapeutics, commented, "We are very pleased with the continued commercial performance and our efforts to realize additional value from ELZONRIS in other indications. Based on encouraging clinical data observed in the first stages of our clinical trial in chronic myelomonocytic leukemia (CMML), combined with a strong rationale for targeting CD123 in this disease setting, we view CMML as our next key indication. We anticipate opening enrollment of the registration-directed stage of the trial in the next few months. We look forward to data and regulatory updates around this program, including in both relapsed/refractory and first-line patients, by the second half of 2020."
Third Quarter 2019 Financial Results Review
Net revenue for ELZONRIS increased to $13.3 million for the quarter ended September 30, 2019.
Stemline ended the third quarter with $174.5 million in cash, cash equivalents and short-term investments. For the third quarter, Stemline reported a net loss of $14.9 million, with net cash expenditures of $11.8 million.
Research and development expenses were $12.3 million for the third quarter of 2019, which reflects an increase of $0.5 million compared with $11.8 million for the third quarter of 2018. The higher costs were primarily due to increased investment as we continue to explore new indications for ELZONRIS.
Selling, general and administrative expenses were $15.4 million for the third quarter of 2019, which reflects an increase of $5.8 million compared with $9.6 million for the third quarter of 2018. The increase in costs were primarily attributable to ongoing commercial launch expenses for ELZONRIS.
Recent Business Highlights
Commercial
We continue to create a positive reimbursement environment for ELZONRIS that we expect will further enhance the value proposition of the brand and generate more patient starts for the foreseeable future. In particular,
Awarding by the CMS (the Centers for Medicare and Medicaid Services) of NTAP (New Technology Add-On Payment), granted to therapies that are deemed to deliver a substantial clinical improvement over existing therapies, for ELZONRIS went into effect on October 1, 2019.
Assignment of an ELZONRIS specific J-Code which makes billing for treatment easier and speeds up claims processing time, also in effect October 1st.
With regard to private payers, ELZONRIS now has favorable coverage for over 170 million lives, with policy decisions to the label for key large commercial payers.
We continue to execute on our brand and disease awareness efforts which are designed to raise the profile of BPDCN and underscore the importance of testing patients for CD123, particularly within the dermatology and dermatopathology segment.
ELZONRIS Product Line Extension Efforts
In an ongoing effort to pursue market expansion opportunities, ELZONRIS is being investigated in a variety of clinical trials and indications, with a number of others planned. By the end of 2020, we expect substantial data and regulatory updates, including in the following programs:
Chronic myelomonocytic leukemia (CMML)
We plan to open an additional single-arm cohort, Stage 3, of patients with relapsed/refractory CMML to the currently enrolling trial in the next few months. In the first part of Stage 3 (Stage 3a), enrichment strategies (e.g. high CD123 expression levels) and certain potential efficacy endpoints, including spleen size reduction, symptom score improvement, and bone marrow complete response with partial hematologic recovery will be evaluated. First-line CMML patients not expected to benefit from available therapies will also be enrolled.
Stage 3a endpoints will be assessed for potential inclusion in the confirmatory cohort (Stage 3b), that will aim to provide the primary evidence of efficacy to support potential registration.
We expect to provide data from Stage 3a, in both relapsed/refractory and first-line patients, as well as further regulatory updates by the end of 2020.
Myelofibrosis (MF)
ELZONRIS clinical data from the ongoing Phase 1/2 trial in patients with relapsed/refractory MF were selected for oral presentation at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) conference.
We are encouraged by the clinical data thus far and are expanding the Stage 2 cohort of the currently enrolling Phase 2 trial of ELZONRIS in patients with relapsed/refractory MF.
We will continue to assess ELZONRIS in relapsed/refractory MF patients as a whole as well as in certain patient subsets of interest including patients with baseline thrombocytopenia (a setting where other agents in this area may require dose interruption or reduction), monocytosis (a potential poor prognostic feature), and high CD123 expression levels.
We expect to provide additional data, including in various subsets of interest, by the end of 2020, and possibly before.
Maintenance therapy post-stem cell transplant (SCT) in BPDCN
The Phase 2 investigator-sponsored clinical trial of ELZONRIS in patients with BPDCN as maintenance therapy post- SCT has been granted regulatory authorization to proceed. The trial will evaluate the safety and feasibility of ELZONRIS in the maintenance setting for patients with BPDCN after SCT.
We expect to provide updates by the end of 2020, and possibly before.
Acute myeloid leukemia (AML)
A Phase 1/2 investigator-sponsored trial is currently ongoing with ELZONRIS in combination with azacitidine and venetoclax in patients with relapsed/refractory AML, elderly AML unfit for chemotherapy, and high-risk myelodysplastic syndrome (MDS).
Phase 1/2 investigator-sponsored trials are currently planned with ELZONRIS in combination with other agents in patients with relapsed/refractory AML and elderly AML unfit for chemotherapy, with high CD123 expression levels and/or BPDCN-like features.
We expect to provide updates by the end of 2020, and possibly before.
Ex-U.S.
In January 2019, Stemline submitted a Marketing Authorization Application to the European Medicines Agency (EMA), seeking approval of ELZONRIS for the treatment of adult patients with BPDCN. We continue to interact with the EMA regarding the application and a scientific advisory group meeting is expected to occur in the first quarter of 2020. Based on this timeline, we expect an opinion or further questions from the Committee for Medicinal Products for Human Use (CHMP) in the first quarter 2020.
ELZONRIS clinical trial data in BPDCN was presented via oral presentation on October 13th at the Japanese Society of Hematology (JSH) meeting in Tokyo, Japan.
Additional Pipeline Candidates
Felezonexor (SL-801) is a reversible inhibitor of XPO1. Updated Phase 1 data were presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) meeting. The trial is ongoing, and we intend to provide further updates as the Phase 1 trial continues.
Stemline is also developing its preclinical assets, SL-1001 (RET kinase inhibitor) and SL-901 (kinase inhibitor), which are both in IND-enabling studies and are expected to enter the clinic next year.
Conference Call Information
The conference call can be accessed by dialing 1-800-367-2403 (domestic) or 1-334-777-6978 (international) and referring to conference ID 9176352. The webcast can be accessed via the company’s website (www.stemline.com), at the bottom of the "Investors & Media" section in the "News & Events" page, and will be available live and for replay shortly after the event.
About ELZONRIS
ELZONRIS (tagraxofusp-erzs), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).
About BPDCN
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.
About CD123
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 (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.