Stemline Therapeutics Recaps ELZONRIS Clinical Data Presentations in Patients with Myelofibrosis (oral presentation) and Mult...
December 10 2019 - 8:00AM
Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage
biopharmaceutical company focused on the development and
commercialization of novel oncology therapeutics, presented data
from ELZONRIS® (tagraxofusp) Phase 1/2 clinical trials in
myelofibrosis (oral presentation) and multiple myeloma, at the 2019
ASH annual meeting. The presentations are now available on the
Stemline website, www.stemline.com, under the Scientific
Presentations tab. Highlights of results are presented below.
Myelofibrosis: Clinical Trial of
ELZONRIS in Patients with Intermediate or High-Risk,
Relapsed/Refractory Myelofibrosis (MF) – Oral
presentation
- ELZONRIS demonstrated efficacy (spleen size reductions and
total symptom score [TSS] reductions) with a predictable and
manageable safety profile, including in patients with poor
prognostic factors, such as thrombocytopenia, CMML-type
features/monocytosis, and clonal evolution
- 45% (9/20) of patients had symptom burden reduction, including
3 with symptom response per IWG-MRT 2013 MF response criteria
- 53% (8/15) of patients with baseline splenomegaly >= 5cm
experienced a spleen size reduction; 20% (3/15) had a reduction
>35% (specifically 100%, 47% and 46% reductions)° 100% (5/5) of
patients with CMML-type features/monocytosis experienced a spleen
size reduction, and 40% (2/5) of these patients had a reduction
>35%° 64% (7/11) of patients with thrombocytopenia (platelet
count < 100x109/L) experienced a spleen size reduction, and 18%
(2/11) had a reduction >35%; 60% (3/5) of patients with
thrombocytopenia (platelet count < 50x109/L) experienced a
spleen size reduction, and 20% (1/5) had a reduction
>35%
- Most common treatment related adverse events (TRAEs) include
alanine aminotransferase increase, headache, hypoalbuminemia (all
17%), anemia, and thrombocytopenia (both 14%)° There was one case
of capillary leak syndrome (CLS), which was grade 3
- 43% overall survival (OS) at 18 months; 29% OS at 2 years
Next Steps
- Expansion of Phase 2 trial to include additional sites is
underway
- Patient enrollment and follow up continues to inform design of
potential registration-directed trial of ELZONRIS in patients with
relapsed/refractory MF, including poor prognosis patients with:°
Thrombocytopenia, and/or° Monocytosis (CD123-overexpressed
subsets)
Other areas of evaluation in MF
- An additional ASH presentation demonstrated that ELZONRIS,
either alone or in combination with ruxolitinib, had preclinical
activity against primary MF patient samples, including those in
accelerated phase and with high molecular risk profiles where
current therapeutic options beyond ruxolitinib are limited and
CD123 expression is evident. These data support further evaluation
of tagraxofusp in MF, not only as single agent but potentially in
combination with Jak inhibitors.
Multiple Myeloma: Clinical Trial of ELZONRIS in Patients
with Relapsed/Refractory Multiple Myeloma (MM)
- ELZONRIS, in combination with pomalidomide (POM) and
dexamethasone (DEX), demonstrated activity and a predictable and
manageable safety profile in heavily pretreated patients with
relapsed/refractory MM
- 56% of patients (5/9) who received ELZONRIS in combination with
POM+DEX had partial responses (PRs)° Notably, these patients also
had decreases in their plasmacytoid dendritic cells (pDCs), a cell
type implicated in myeloma growth and aggressiveness, and the cell
of origin of blastic plasmacytoid dendritic cell neoplasm
(BPDCN)
- Given these clinical data, ELZONRIS may offer a novel mechanism
of action and future therapeutic option for patients with MM
Next Steps
- Given these encouraging early results, potential avenues for
further ELZONRIS development are being assessed, including
evaluating additional patients with this and/or potentially other
regimens including in combination with daratumumab or novel agents
such as XPO1 inhibitors. Moreover, the clinical knockdown of pDCs
in MM patients may also be relevant with respect to other diseases,
such as chronic myelomonocytic leukemia (CMML), MF and certain
acute myeloid leukemia (AML) patient subsets, where pDCs have been
similarly implicated in the aggressiveness of these
malignancies.
About ELZONRIS®
ELZONRIS® (tagraxofusp), 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 CD123CD123 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.
About Stemline Therapeutics Stemline
Therapeutics, Inc. is a commercial-stage biopharmaceutical company
focused on the development and commercialization of novel oncology
therapeutics. ELZONRIS® (tagraxofusp), a
targeted therapy directed to CD123, is FDA-approved and
commercially available in the U.S. for the treatment of adult and
pediatric patients, two years and older, with blastic plasmacytoid
dendritic cell neoplasm (BPDCN). In Europe, a marketing
authorization application (MAA) is under review by the European
Medicines Agency (EMA). ELZONRIS is also being evaluated in
clinical trials in additional indications including chronic
myelomonocytic leukemia (CMML), myelofibrosis (MF) and acute
myeloid leukemia (AML). Additional pipeline candidates include:
felezonexor (SL-801) (XPO1 inhibitor; Phase 1 in advanced solid
tumor patients ongoing), SL-1001 (novel RET kinase inhibitor,
IND-enabling studies ongoing), SL-701 (immunotherapeutic; Phase 2
in glioblastoma patients completed), and SL-901 (novel kinase
inhibitor; prior abbreviated European Phase 1, IND-enabling studies
ongoing). For more information, please visit the company’s website
at www.stemline.com.
Forward-Looking StatementsSome 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 Europe; the success
and timing of our clinical trials and preclinical studies for our
product and product candidates, including ELZONRIS in additional
indications and our other pipeline candidates, including site
initiation, institutional review board approval, scientific review
committee approval, patient accrual, safety, tolerability and
efficacy data observed, and input from regulatory authorities
including the risk that the FDA, EMA, or other ex-U.S. national
drug authority ultimately does not agree with our data, find our
data supportive of approval, or approve any of our product
candidates; the possibility that results of clinical trials are not
predictive of safety and efficacy results of our product candidates
in broader patient populations or of our products if approved; our
plans to develop and commercialize our product candidates,
including, but not limited to delays in arranging satisfactory
manufacturing capabilities and establishing commercial
infrastructure for ELZONRIS; product efficacy or safety concerns
resulting in product recalls or regulatory action; the risk that
estimates regarding the number of patients with the diseases that
our product and product candidates may treat are inaccurate;
inadequate market penetration of our products; our products not
gaining acceptance among patients (and providers or third party
payors) for certain indications (due to cost or otherwise); the
risk that third party payors (including governmental agencies) will
not reimburse for the use of ELZONRIS at acceptable rates or at
all; the company’s ability to produce, maintain or increase sales
of ELZONRIS; the company’s ability to develop and/or commercialize
ELZONRIS; the adequacy of our pharmacovigilance and drug safety
reporting processes; our available cash and investments; our
ability to obtain and maintain intellectual property protection for
our product and product candidates; delays, interruptions, or
failures in the manufacture and supply of our product and product
candidates; the performance of third-party businesses, including,
but not limited to, 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 SEC. 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.
Contact: Investor RelationsStemline
Therapeutics, Inc.750 Lexington AvenueEleventh FloorNew York, NY
10022Tel: 646-502-2307Email: investorrelations@stemline.com
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