On December 13, 2017 The Medicines Company (NASDAQ:MDCO) reported that it will host an Investor Day on Tuesday, January 23, 2018, from 10:00 a.m. to 12:30 p.m., Eastern Time, in New York City (Press release, Medicines Company, DEC 13, 2017, View Source [SID1234522626]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The event will be led by Fred Eshelman, Pharm.D., Executive Chairman of The Medicines Company, and Clive Meanwell, M.D., Ph.D., Chief Executive Officer of the Company, who will be joined by members of the Company’s management team and outside experts and key members of the ORION coalition, including:

Eugene Braunwald, M.D., Distinguished Hersey Professor of Medicine at Harvard Medical School and founding Chairman of the Thrombolysis in Myocardial Infarction (TIMI) Study Group;
John J.P. Kastelein, M.D., Ph.D., Professor of Medicine and Chairman of the Department of Vascular Medicine at the Academic Medical Center of the University of Amsterdam; and
Marc S. Sabatine M.D., M.P.H., Lewis Dexter, M.D., Distinguished Chair in Cardiovascular Medicine at Brigham and Women’s Hospital, Professor of Medicine at Harvard Medical School and Chairman of the TIMI Study Group.
Presentations and panel discussions will focus on inclisiran, its highly-differentiated value proposition and clinical and non-clinical development, manufacturing, regulatory and pre-commercial projects, as well as on the continuing, unmet medical and market needs in atherosclerotic cardiovascular disease.

The event will be broadcast live via webcast. To access the live webcast and view the accompanying slide presentation, visit the "Investors – Events/Presentations" section of The Medicines Company website at least 15 minutes before the event is scheduled to begin to register and download or install any necessary software. In addition to the webcast, the event can be accessed, in listen-only mode, as follows:

U.S./Canada: (877) 359-9508
International: (224) 357-2393
Conference ID: 60380330
A replay of the webcast will be archived and available after the event for a limited period of time.

The in-person event is reserved for financial analysts and institutional investors and is by invitation only.

On December 13, 2017 Bayer reported that the Chinese Food and Drug Administration (CFDA) approved Stivarga (regorafenib) tablets for the second-line treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with Nexavar (sorafenib) (Press release, Bayer, DEC 13, 2017, View Source [SID1234523072]). The data from the pivotal Phase III RESORCE study showed that Stivarga (regorafenib) provided a statistically significant and clinically meaningful improvement in overall survival (OS) versus placebo; the median OS was 10.6 vs 7.8 months, (HR 0.62, 95% CI 0.50-0.78; p<0.0001). Exploratory analyses of the RESORCE trial showed that the median time from the start of prior sorafenib treatment to death was 26 months in patients receiving regorafenib versus 19.2 months in those receiving placebo. Regorafenib is the first drug approved for the second-line treatment of patients with HCC in China. The CFDA approval expands Bayer’s leadership in liver cancer with a treatment plan in HCC involving use of Stivarga directly after progression on Nexavar.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Following the approval of Stivarga for the treatment of metastatic colorectal cancer and gastrointestinal stromal tumors earlier this year in China, the approval in HCC brings new hope to Chinese patients with HCC who previously had no effective treatment options after being treated with Nexavar", said Robert LaCaze, Member of the Executive Committee of Bayer AG’s Pharmaceuticals Division and Head of the Oncology Strategic Business Unit. "The product is already approved for the treatment of HCC in many countries around the world, including US, Japan and in the EU, and this milestone expands Bayer’s global leadership in liver cancer."

Liver cancer is often more difficult to treat than other cancers with 466,000 new cases diagnosed and 422,000 deaths in China per year. Globally, it is the second leading cause of cancer-related deaths.

About Regorafenib (Stivarga)
Regorafenib is an oral multi-kinase inhibitor that potently blocks multiple protein kinases involved in tumor angiogenesis (VEGFR1, -2, -3, TIE2), oncogenesis (KIT, RET, RAF-1, BRAF), metastasis (VEGFR3, PDGFR, FGFR) and tumor immunity (CSF1R).

Regorafenib is approved under the brand name Stivarga in more than 90 countries worldwide, including the U.S., countries of the EU, China and Japan for the treatment of metastatic colorectal cancer (mCRC). The product is also approved in over 80 countries, including the U.S., countries of the EU, China and Japan, for the treatment of metastatic gastrointestinal stromal tumors (GIST). This year, it was also approved in the U.S., Japan and countries of the EU for second-line treatment of HCC.

In the EU, Stivarga is indicated as monotherapy for the treatment of adult patients with mCRC who have been previously treated with, or are not considered candidates for, available therapies including fluoropyrimidine-based chemotherapy, an anti-VEGF therapy and an anti-EGFR therapy, as well as for the treatment of adult patients with unresectable or metastatic GIST who progressed on or are intolerant to prior treatment with imatinib and sunitinib, and for the treatment of adult patients with HCC who have been previously treated with sorafenib.

Regorafenib is a compound developed by Bayer. In 2011, Bayer entered into an agreement with Onyx, now an Amgen subsidiary, under which Onyx receives a royalty on all global net sales of regorafenib in oncology.

About Oncology at Bayer
Bayer is committed to delivering science for a better life by advancing a portfolio of innovative cancer treatments. The oncology franchise at Bayer currently includes three oncology products and several other compounds in various stages of clinical development. Together, these products reflect the company’s approach to research, which prioritizes targets and pathways, with the potential to impact the way that cancer is treated.

On December 13, 2017 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel therapeutics for difficult to treat cancers, reported detailed data from its SL-401 pivotal trial in BPDCN, as well as results from other ongoing trials in additional indications, at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, held in Atlanta, GA (Press release, Stemline Therapeutics, DEC 13, 2017, View Source [SID1234522625]). Presentations are available on the Stemline website, www.stemline.com, under the Scientific Presentations tab.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

SL-401: Pivotal Trial in BPDCN – Primary endpoint met; Median overall survival (OS) not reached in first-line patients; BLA submission preparation underway

We believe the SL-401 pivotal trial in BPDCN is the largest multicenter prospective study ever conducted in this indication. The trial enrolled 45 BPDCN patients (32 first-line, 13 relapsed/refractory) and consisted of 3 Stages: Stage 1 (lead-in, dose escalation), Stage 2 (expansion), and Stage 3 (pivotal, confirmatory)

Key outcomes in Stages 1, 2, and 3 (ASH ’17 data)
– Across all 3 stages, 42 patients received SL-401 at 12 ug/kg/day
– In first-line BPDCN, SL-401 (12 ug/kg/day)
– 90% (26/29) overall response rate (ORR)
– 72% (21/29) rate of CR + CRc + CRi (complete response + clinical CR [CR with residual skin abnormality] + CR with incomplete bone marrow recovery)
– 45% (13/29) of patients were bridged to stem cell transplant (SCT)
– In relapsed/refractory BPDCN, SL-401 (12 ug/kg/day)
– 69% (9/13) ORR
– 38% (5/13) CR + CRc + CRi rate; 1 patient bridged to SCT
– Median overall survival (OS) not reached in first-line BPDCN (Stages 1-2, and 3), SL-401 (12 ug/kg/day)
– 71% 12-month OS in Stages 1 and 2; follow-up ongoing for 12-month OS in Stage 3
– Most common treatment-related adverse events (TRAEs) were: alanine aminotransferase increase (52%), aspartate aminotransferase increase (50%), hypoalbuminemia (50%), and thrombocytopenia (38%). TRAEs included capillary leak syndrome (19%), which was grade 5 in 2.4% (1/42) of BPDCN patients at 12 ug/kg/day, 2.6% (4/153) of all patients across all trials at all doses, and 1.7% (2/119) of patients across all trials at 12 ug/kg/day

Stage 3 pivotal cohort (first-line, 12 ug/kg/day)
– Met its primary endpoint with a 54% (7/13) CR + CRc rate (95% CI: 25.1, 80.8)
– The lower bound of the 95% confidence interval (CI) exceeded the pre-specified rate of 10%
– 77% (10/13) ORR
– 46% (6/13) of patients bridged to SCT

Next steps for BPDCN
– A BLA submission is in preparation
SL-401: Phase 1/2 Trial in myeloproliferative neoplasms (MPN): chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF)

Key outcomes (ASH ’17 data)
– SL-401 Phase 1/2 trial consists of a Stage 1 (lead-in, dose escalation) and Stage 2 (expansion); has enrolled 24 patients
– No dose limiting toxicities (DLT) were identified and a maximum tolerated dose (MTD) was not reached. Most common TRAEs include hypoalbuminemia (33%), thrombocytopenia (33%), and fatigue (29%). Most common TRAEs (grade 3 or higher), include thrombocytopenia (24%) and anemia (19%)
– Durable CR (14+ months) in CMML patient
– 65% (11/17 evaluable) of CMML and MF patients had spleen reductions >25% (range 29% to 100%)
– Durable SD in 4 patients (2 CMML, 2 MF) for 5+ to 8+ months. Three ongoing SD patients enrolled with baseline platelet counts <100,000, including 1 patient platelet count <50,000 are on treatment

Next Steps in MPN
– Continue enrollment and patient follow-up
– Favorable tolerability and preliminary signs of activity support both single agent and combination development strategies, including JAK-inhibitors and hypomethylating agents
SL-401: Phase 1/2 Trial in Acute Myeloid Leukemia (AML) in CR with Minimal Residual Disease (MRD)

Key outcomes (ASH ’17 data)
– SL-401 Phase 1/2 trial consists of Stage 1 (lead-in, dose escalation) and Stage 2 (expansion); has enrolled 16 patients
– No DLTs or MTD identified in Stage 1; Most common TRAEs (Stage 1 and 2) include hypoalbuminemia (44%), ALT increase (38%), AST increase (38%), and thrombocytopenia (38%). Most common TRAEs, grade 3+, include ALT increase (31%), AST increase (25%), and thrombocytopenia (19%). 2 cases (13%) of grade 3 CLS were noted
– Five patients, including one who went to SCT at 3+ months, were relapse-free for at least 5+ months (range 5+ to 14+), including 2 ongoing (8+ months [on SL-401] and 14+ months [SCT])

Next Steps for AML/MRD
– Patients enrolling and being followed for MRD alterations and response duration
– Given preclinical data indicating potential synergies between SL-401 and azacitidine in AML and high-risk MDS, and that a clinical trial is underway assessing that combination in AML, a transition to combination therapy in this indication is under active consideration
Ivan Bergstein, M.D., CEO of Stemline, commented "The remarkable data presented at ASH (Free ASH Whitepaper) from our pivotal trial with SL-401 in BPDCN sets the stage for our upcoming BLA submission and a successful 2018. In conjunction with these efforts, we kicked off our BPDCN disease awareness campaign at ASH (Free ASH Whitepaper), which includes outreach to hematologist-oncologists, dermatologists, and pathologists, including highlighting the BPDCN diagnostic signature triad of CD123, CD4 and CD56 (an easy to remember: 1, 2, 3, 4, 5, 6). We believe this endeavor is critical for the proper and timely diagnosis of BPDCN, a historically underappreciated and underdiagnosed malignancy. Additionally, we and our investigators believe SL-401 is beginning to demonstrate encouraging signs of clinical activity in indications beyond BPDCN, including CMML and MPN, as presented at ASH (Free ASH Whitepaper)."

On December 13, 2017 Eleven Biotherapeutics, Inc. (NASDAQ:EBIO), a late-stage clinical oncology company advancing novel product candidates based on its Targeted Protein Therapeutics (TPTs) platform, reported that Dr. Gregory Adams, Chief Scientific Officer, will chair a session focused on antibody drug conjugates and fusion proteins at the Antibody Engineering and Therapeutics Meeting in San Diego, CA (Press release, Eleven Biotherapeutics, DEC 13, 2017, View Source [SID1234522620]). As part of the session, Dr. Adams will give a talk on the potential for tumor-targeted payloads to prime the immune system to facilitate more effective therapy in combination with immuno-oncology agents, including checkpoint inhibitors.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Checkpoint inhibitors are a significant step forward in the quest to engage the patient’s immune system in the fight against cancer. Checkpoint inhibitors function by blocking the signals that cancers use to inhibit the immune system. However, for checkpoint inhibitors to be effective, they require the presence of an active immune response against the cancer. There is broad recognition of the potential for tumor-targeted payloads, such as Eleven’s Targeted Protein Therapeutics (TPTs), to provide this spark to the immune system and act synergistically with checkpoint inhibitors," said Dr. Gregory Adams, Chief Scientific Officer of Eleven Biotherapeutics. "Earlier this year at the American Association of Cancer Research Conference, we presented data which supports this potential, demonstrating that TPTs induce immunogenic cell death as evidenced by the presence of damage-associated molecular patterns. Our collaboration with the National Cancer Institute and AstraZeneca aims to build on this evidence."

Session Details

Title: Antibody-Drug Conjugates & Fusion Proteins
Date: Wednesday, December 13, 2017
Time: 8:10 am – 12:00 pm PT
Location: Manchester Grand Hyatt; San Diego, CA

About Vicinium

Vicinium is manufactured as a single protein anti-epithelial cell adhesion molecule (anti-EpCAM) fusion protein fused with Pseudomonas Exotoxin A (ETA) designed to specifically target and deliver a potent anti-cancer payload directly into tumor cells. It is constructed with a stable, genetically-engineered linker to ensure its potent protein payload remains attached until it is internalized by the cancer cell, which is believed to decrease the risk of toxicity to healthy tissues thereby improving the compound’s safety. Vicinium’s one-step manufacturing process offers significant cost advantages and results in the production of a homogenous product, with less batch-to-batch variability than most antibody drug conjugates. Vicinium is currently in a Phase 3 registration clinical trial for the treatment of high-grade non-muscle invasive bladder cancer (NMIBC) in patients who have previously received two courses of Bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive. Eleven Biotherapeutics intends to enroll 134 subjects in the trial, including 77 subjects with carcinoma in situ (CIS), at over 70 centers in the United States and Canada. Primary and secondary endpoints include complete response (CR) in CIS subjects, time to disease recurrence and event free survival. The Company expects to complete patient enrollment in the first quarter of 2018 and to report topline three-month data in mid-2018.

On December 12, 2017 Palleon Pharmaceuticals, a company focused on developing Glycoimmune Checkpoint Inhibitors to treat cancer, reported an agreement with King’s College London to license intellectual property developed in the laboratory of Joy Burchell, Ph.D., Professor of Glyco-Oncology at the university. This agreement gives Palleon the exclusive rights to a patent portfolio that will facilitate the development of drugs that target Glycoimmune Checkpoints, a novel approach to overcoming resistance to first-generation immuno-oncology drugs.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Professor Burchell has been a leader in the field of aberrant glycosylation in breast cancer for over 25 years. Earlier in her career she developed tools that demonstrated that more than 90% of breast cancers, and many other carcinomas, carry glycans that are different from those carried by proteins on normal cells. She was the first to show that a mucin known as MUC1 is present in the sera of breast cancer patients. This discovery enabled the development of the CA15.3 test, a serum assay used to measure the response to breast cancer treatment and to monitor recurrence of breast cancer. More recently, Professor Burchell has been investigating how aberrant glycosylation of MUC1 plays a significant role in immunosuppression and allows the cancer to thrive.

Jim Broderick, M.D., Chief Executive Officer and Founder of Palleon, commented, "Professor Burchell’s research is at the forefront of understanding how tumors use glycans to evade the immune system. We now know that tumor cells down-regulate a wide spectrum of immune cell types by cloaking themselves in certain glycan patterns, and that this mechanism of immunosuppression can be targeted by a new class of drugs. This licensing agreement strengthens Palleon’s position as the leader in this new approach to defeating cancer’s suppression of the human immune system."

Dr. Burchell added, "We have known about the alteration of glycans on the surface of malignant cells for decades. However, recent discoveries in the field of glycoscience have demonstrated the role of glycans in immunosuppression. Glycobiology is now emerging as a major axis of immunosuppression in cancer. We expect these findings to provide the foundation for developing immuno-oncology drugs that will have a significant impact on the lives of patients."