On December 6, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported that highlighted three upcoming ELZONRIS (tagraxofusp), presentations, including an oral presentation of ELZONRIS clinical data in myelofibrosis, at this year’s American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, that is being held from December 7-10 in Orlando, FL (Press release, Stemline Therapeutics, DEC 6, 2019, View Source [SID1234551990]).

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Details on the ASH (Free ASH Whitepaper) presentations are as follows:

Myelofibrosis – ELZONRIS monotherapy, clinical; oral presentation
Title: Results from a Phase 1/2 Clinical Trial of Tagraxofusp (SL-401) in Patients with Intermediate, or High Risk, Relapsed/Refractory Myelofibrosis
Presenter: Naveen Pemmaraju, MD; MD Anderson Cancer Center
Abstract: 558
Session: 634. Myeloproliferative Syndromes: Clinical: Emerging and Novel Targeted Therapies
Date/Time: Monday, December 9, 2019 8:15 AM ET
Location: Orange County Convention Center, W304EFGH

Multiple Myeloma – ELZONRIS in combination with Pom+Dex, clinical; poster presentation
Title: Results from Phase 1/2 Trial of Tagraxofusp in Combination with Pomalidomide and Dexamethasone in Relapsed or Refractory Multiple Myeloma
Presenter: Paul G. Richardson, M.D.; Dana-Farber Cancer Institute
Abstract: 3145
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster II
Date/Time: Sunday, December 8, 2019 6:00 PM–8:00 PM ET
Location: Orange County Convention Center, Hall B

Myeloproliferative Neoplasms – ELZONRIS in combination with ruxolitinib, preclinical; poster presentation
Title: Evaluation of Tagraxofusp (SL-401) Alone and in Combination with Ruxolitinib for the Treatment of Myeloproliferative Neoplasms
Presenter: Aishwarya Krishnan, MD; Memorial Sloan Kettering Cancer Center
Abstract: 2967
Session: 635. Myeloproliferative Syndromes: Basic Science: Poster II
Date/Time: Sunday, December 8, 2019 6:00 PM–8:00 PM ET
Location: Orange County Convention Center, Hall B

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.

On December 6, 2019 ViewRay, Inc. (Nasdaq: VRAY), maker of the MRIdian, which combines MRI and external-beam radiation therapy to simultaneously image and treat cancer patients, reported the completion of its previously announced underwritten public offering of 47,782,500 shares of common stock at a price to the public of $3.13 per share, for gross proceeds of approximately $149.6 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by ViewRay (Press release, ViewRay, DEC 6, 2019, View Source [SID1234552009]). All of the shares sold in the offering were sold by ViewRay. The shares sold included the full exercise of the underwriters’ option to purchase additional shares.

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ViewRay intends to use the net proceeds from the offering for working capital and general corporate purposes, including capital expenditures, research and development expenses, investments, commercial expenses, clinical data generation costs and infrastructure expenses.

Elekta AB (Nasdaq: EKTA-B.ST), an affiliate of Medtronic (NYSE: MDT) and an affiliate of ViewRay’s largest shareholder, Fosun International Limited, each consummated their previously-announced investments in ViewRay by participating in the offering.

Piper Jaffray & Co. acted as the lead bookrunner. Guggenheim Securities, LLC also acted as a bookrunner. Oppenheimer & Co. Inc. acted as co-manager for the offering.

On December 6, 2019 Luminex Corporation (Nasdaq:LMNX) (the "Company") reported that its board of directors declared a cash dividend for the fourth quarter of 2019 of $0.09 per share of common stock payable on January 15, 2020 to stockholders of record as of the close of business December 19, 2019 (Press release, Luminex, DEC 6, 2019, View Source [SID1234552010]).

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On December 6, 2019 Oncopeptides AB (Nasdaq Stockholm: ONCO), reported that the company will host a webcast on Tuesday, December 10th, 2019, at 13:00 (CET) to provide an update regarding the data presented December 7-8 at the American Society Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2019 (Press release, Oncopeptides, DEC 6, 2019, View Source [SID1234552011]). The webcast will be hosted by CEO Jakob Lindberg.

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The webcast can be followed via the link:

View Source

Participants who would like to ask questions can use the telephone numbers below:

Sweden: +46-8-56642706
Europe: +44-3333009270
USA: +1-8335268384

The presentation can be found at:

View Source

This information was submitted for publication at 14:00 CET, December 6, 2019

On December 6, 2019 Bristol-Myers Squibb Company (NYSE: BMY) and bluebird bio, Inc. (Nasdaq: BLUE) reported positive top-line results from KarMMa, a pivotal, open-label, single arm, multicenter, Phase 2 study of idecabtagene vicleucel (ide-cel; bb2121) (Press release, bluebird bio, DEC 6, 2019, View Source [SID1234551995]). KarMMa, which evaluated the efficacy and safety of the companies’ lead investigational BCMA-targeted chimeric antigen receptor (CAR) T cell therapy candidate for patients with relapsed and refractory multiple myeloma, met its primary endpoint and key secondary endpoint.

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KarMMa enrolled 140 patients, of whom 128 patients were treated with ide-cel across the target dose levels of 150-450 x 106 CAR+ T cells. All treated patients were exposed to at least three prior therapies, including an immunomodulatory (IMiD) agent, a proteasome inhibitor (PI) and an anti-CD38 antibody, and all were refractory to their last regimen. Ninety-four percent of patients were refractory to an anti-CD38 antibody and 84% percent were triple refractory (refractory to an IMiD agent, PI and anti-CD38 antibody).

Results for the primary endpoint (overall response rate [ORR]) and key secondary endpoint (complete response rate [CR]), as well as duration of response (DoR) and progression-free survival (PFS) across the target dose levels and at each of the three target doses explored in the study are presented in the table below. The median follow-up duration for all subjects was 11.3 months.

Median DOR and median PFS are not reported for the 150 x 106 CAR+ T cells

dose group due to the small number of evaluable patients

Overall, the safety results were consistent with those observed in the phase 1 CRB-401 study, which evaluated the preliminary safety and efficacy of ide-cel. Instances of grade 3 or higher cytokine release syndrome (CRS) occurred in 5.5% (7/128) of patients, including one fatal CRS event. Investigator identified grade 3 or higher neurotoxicity events (iiNT) occurred in 3.1% (4/128) of patients and there were no Grade 4 iiNT events reported. Grade 3 or higher CRS and iiNT events were reported in <6% of subjects at each target dose. CRS of any grade occurred in 83.6% (107/128) of patients and iiNT of any grade occurred in 18% (23/128) of patients.

"For multiple myeloma patients who have relapsed and become refractory to current treatment options, there remains a high unmet need, as these patients typically experience low response rates, short response durations and poor survival," said Kristen Hege, M.D., Senior Vice President, Hematology/Oncology and Cell Therapy, Early Clinical Development for Bristol-Myers Squibb. "The KarMMa study provides further support for ide-cel as a potential therapeutic option in this heavily pre-treated patient population, and we are encouraged by these data, especially the outcomes observed at the highest target dose of 450 x 106 CAR+ T cells. We are actively preparing for submission of these data to Health Authorities for proposed initial registration of ide-cel as a first-in-class BCMA-targeted CAR T cell therapy."

"Multiple myeloma is a relentless disease and there is significant need to find new treatment options for patients who advance through the current therapies available to them," said Joanne Smith-Farrell, Ph.D., oncology franchise lead and chief business officer, bluebird bio. "With these data in hand, bluebird bio and Bristol-Myers Squibb remain fully focused on advancing ide-cel as quickly as possible for patients in late-line myeloma, while continuing to execute our broad development program to understand the potential benefits of ide-cel across earlier lines of therapy."

More comprehensive data from KarMMa will be submitted for presentation at a future medical meeting.

About KarMMa

KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multi-center phase 2 study evaluating the efficacy and safety of ide-cel in adult patients with relapsed and refractory multiple myeloma, in North America and Europe. The primary endpoint of the study is overall response rate as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria. Complete response rate is a key secondary endpoint. Other efficacy endpoints include time to response, duration of response, progression-free survival, overall survival and minimal residual disease evaluated by Next-Generation Sequencing (NGS) assay. The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 106 CAR+ T cells after receiving lymphodepleting chemotherapy. All enrolled patients had received at least three prior treatment regimens, including an IMiD agent, a PI and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.

About Ide-cel

Ide-cel is a CAR T cell therapy targeting B-cell maturation antigen (BCMA), which is expressed on the surface of normal and malignant plasma cells. The ide-cel CAR construct includes an anti-BCMA scFv-targeting domain for antigen specificity, a transmembrane domain, a CD3-zeta activation domain, and a 4-1BB co-stimulatory domain hypothesized to increase T-cell activation, proliferation and persistence. Ide-cel CAR T cells are proposed to recognize and bind to BCMA on the surface of multiple myeloma cells leading to apoptosis.

In November 2017, ide-cel was granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration and PRIority Medicines (PRIME) eligibility by the European Medicines Agency based on preliminary clinical data from the phase 1 CRB-401 study.

Bristol-Myers Squibb and bluebird bio’s broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3) in earlier lines of treatment for patients with multiple myeloma. For more information visit: clinicaltrials.gov.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between BMS and bluebird bio.

Ide-cel is not approved for any indication in any geography.

Bristol-Myers Squibb: Advancing Cancer Research

At Bristol-Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase quality, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.