On November 1, 2017 Incyte Corporation (Nasdaq:INCY) reported that more than 30 abstracts including data from its clinical development programs for Jakafi (ruxolitinib), JAK1, PI3Kδ, PIM and BRD will be presented at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2017 in Atlanta, Georgia from December 9-12, 2017 (Press release, Incyte, NOV 1, 2017, View Source [SID1234521410]).

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“Data accepted for presentation include long-term, 4-year follow-up from our RESPONSE trial, updated data from our PI3Kδ inhibitor program in relapsed or refractory B-cell malignancies as well as first-in-man data from our BRD and PIM kinase inhibitor programs.”
“We look forward to presenting new data from across our clinical development portfolio,” said Steven Stein, M.D., Chief Medical Officer at Incyte. “Data accepted for presentation include long-term, 4-year follow-up from our RESPONSE trial, updated data from our PI3Kδ inhibitor program in relapsed or refractory B-cell malignancies as well as first-in-man data from our BRD and PIM kinase inhibitor programs.”

Select key abstract presentations include:

Jakafi (ruxolitinib)

Promising Results of a Phase 1/2 Clinical Trial of Ruxolitinib in Patients with Chronic Myelomonocytic Leukemia (Abstract #162)

Saturday, December 9, 2017, 12:00-1:30 PM, Building B, Level 3, B312-B314, Oral Session 637, Myelodysplastic Syndromes—Clinical Studies: Predicting Drug Response Using Novel Genomic Algorithms
Examining The Treatment Patterns And Blood Counts Among Patients With Polycythemia Vera Treated With Hydroxyurea In The United States: An Analysis From The REVEAL Study (Abstract #1633)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2,
Poster Session I
Role Of Symptom Burden In Disability Leave Among Patients With Myeloproliferative Neoplasms (MPNs): Findings From The Living With MPN Patient Survey (Abstract #1637)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2,
Poster Session I
Ruxolitinib Or Dasatinib In Combination With Chemotherapy For Patients With Relapsed/Refractory Philadelphia (Ph)-Like Acute Lymphoblastic Leukemia: A Phase I-II Trial (Abstract #1322)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2,
Poster Session I
The Combination Of Ruxolitinib (RUX) With Decitabine (DAC) In Patients (Pts) With Post-Myeloproliferative Neoplasm Acute Myeloid Leukemia (Post-MPN AML): Interim Report Of A Phase I/II Trial (Abstract #1379)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2,
Poster Session I
A Phase Ib Study To Assess The Safety And Tolerability Of Oral Ruxolitinib In Combination With Azacitidine In Patients With Advanced Phase Myeloproliferative Neoplasms (MPN), Including Myelodysplastic Syndromes (MDS) Or Acute Myeloid Leukaemia (AML) Arising From MPN (The Bloodwise / TAP PHAZAR Study On Behalf Of The UK MPN CSG) (Abstract #1649)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2,
Poster Session I
Results From The 208-Week (4-year) Follow-Up Of RESPONSE Trial, A Phase 3 Study Comparing Ruxolitinib (Rux) With Best Available Therapy (BAT) For The Treatment Of Polycythemia Vera (PV) (Abstract #322)

Sunday, December 10, 2017, 8:15 AM, Building C, Level 2, C208-C210,
Oral Session 634, Myeloproliferative Syndromes: Clinical: Phase III and Long-Term Outcome Studies in MPNs
Patient-Reported Symptom Burden And Peripheral Blood Counts Among Patients With Polycythemia Vera: And Analysis From The REVEAL Study (Abstract #2924)

Sunday, December 10, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session II
Safety And Efficacy Of Ruxolitinib (Rux) In An Open-Label, Multicenter, Expanded Treatment Protocol In Patients (Pts) With Polycythemia Vera (PV) Who Are Hydroxyurea (HU) Resistant Or Intolerant And For Whom No Alternative Treatments Are Available (Abstract #2918)

Sunday, December 10, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session II
Primary Analysis Of JUMP, A Phase 3b, Expanded-Access Study Evaluating The Safety And Efficacy Of Ruxolitinib In Patients With Myelofibrosis (N=2233) (Abstract #4204)

Monday, December 11, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session III
Characteristics Of 809 Patients With Essential Thrombocythemia In Real-World Clinical Practice: A Chart Review Study In The United States (Abstract #1636)

Monday, December 11, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session III
Real-World Patterns Of First-Line Hydroxyurea Treatment Among Patients With Essential Thrombocythemia In US Community Oncology Practices (Abstract #4203)

Monday, December 11, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session III
Ponatinib

First Report of the Gimema LAL1811 Phase II Prospective Study of the Combination of Steroids with Ponatinib As Frontline Therapy of Elderly or Unfit Patients with Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Abstract #99)

Saturday, December 9, 2017, 9:30-11:00 AM, Building C, Level 2, C211-C213,
Oral Session 612, Acute Lymphoblastic Leukemia: Clinical Studies: Advances in the Treatment of ALL
Efficacy And Safety Of Ponatinib In Chronic Phase-Chronic Myeloid Leukemia (CP-CML) According To The Extent Of Treatment With Prior Tyrosine Kinase Inhibitors (TKIs): Final (5-Year) Results Of The PACE Study (Abstract #1617)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2
Arterial Occlusive Events (AOEs) In The Phase 2 Ponatinib PACE Trial: 5-Year Update In Heavily Treated Patients (Pts) With Chronic-Phase Chronic Myeloid Leukemia (CP-CML) (Abstract #2896)

Sunday, December 10, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session II
Pipeline

Results From A Phase 1/2 Study Of INCB050465, A Highly Selective And Highly Potent PI3Kδ Inhibitor, In Patients With Relapsed Or Refractory B-Cell Malignancies (CITADEL-101) (Abstract #410)

Sunday, December 10, 2017, 12:15 PM, Building C, Level 1, Hall C1, Oral Session 623, Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma—Clinical Studies: Indolent Lymphomas, Novel Therapies and Diagnostics
Preliminary Results From An Ongoing Phase 1/2 Study Of INCB053914, A Pan-Proviral Integration Sites For Moloney Virus (PIM) Kinase Inhibitor, In Patients With Advanced Hematologic Malignancies (Abstract #2585)

Sunday, December 10, 2017, 6:00-8:00 PM, Building A, Level 1, Hall A2,
Poster Session II
A Phase 1/2 Study Of The Oral Novel JAK1 Inhibitor INCB052793 As Monotherapy And In Combination With Standard Therapies In Patients With Advanced Hematologic Malignancies (Abstract #640)

Monday, December 11, 2017, 10:30 AM-12:00 PM, Building B, Level 5, Murphy BR1-2,
Oral Session 613, Acute Myeloid Leukemia: Clinical Studies: Novel Therapies for AML and APL
Preliminary Results From An Ongoing Phase 1/2 Study Of INCB057643, A Bromodomain And Extraterminal (BET) Protein Inhibitor, In Patients (pts) With Advanced Malignancies (Abstract #4048)

Monday, December 11, 2017, 6:00–8:00 PM, Building A, Level 1, Hall A2,
Poster Session III
Preclinical

The Pan-PIM Inhibitor INCB053914 Displays Potent Synergy At Low Doses In Combination With Ruxolitinib In Pre-Clinical Models Of MPNs (Abstract #1661)

Saturday, December 9, 2017, 5:30-7:30 PM, Building A, Level 1, Hall A2,
Poster Session I
Targeting Cell Non-Autonomous MAPK Activation As A Novel Therapeutic Strategy In Myeloproliferative Neoplasms (Abstract #381)

Sunday, December 10, 2017, 10:00 AM, Building C, Level 2, C208-C210, Oral Session 635, Myeloproliferative Syndromes: Basic Science: Identification of novel targets for the treatment of myeloproliferative neoplasms
Redundant JAK, SRC And PI3 Kinase Signaling Pathways Regulate Cell Survival In Human Ph-Like ALL Cell Lines And Primary Cells (Abstract #717)

Monday, December 11, 2017, 2:45-4:15 PM, Building B, Level 2, B216-B217
Full session details and data presentation listings for ASH (Free ASH Whitepaper) 2017 can be found at: View Source

About Jakafi (ruxolitinib)

Jakafi is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. Food and Drug Administration, for treatment of people with intermediate or high-risk myelofibrosis (MF), including primary MF, post–polycythemia vera MF, and post–essential thrombocythemia MF.

Jakafi is also indicated for treatment of people with polycythemia vera (PV) who have had an inadequate response to or are intolerant of hydroxyurea.

Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States. Jakafi is a registered trademark of Incyte Corporation. Jakavi is a registered trademark of Novartis AG in countries outside the United States.

Important Safety Information

Jakafi can cause serious side effects, including:

Low blood counts: Jakafi (ruxolitinib) may cause your platelet, red blood cell, or white blood cell counts to be lowered. If you develop bleeding, stop taking Jakafi and call your healthcare provider. Your healthcare provider will perform blood tests to check your blood counts before you start Jakafi and regularly during your treatment. Your healthcare provider may change your dose of Jakafi or stop your treatment based on the results of your blood tests. Tell your healthcare provider right away if you develop or have worsening symptoms such as unusual bleeding, bruising, tiredness, shortness of breath, or a fever.

Infection: You may be at risk for developing a serious infection during treatment with Jakafi. Tell your healthcare provider if you develop any of the following symptoms of infection: chills, nausea, vomiting, aches, weakness, fever, painful skin rash or blisters.

Skin cancers: Some people who take Jakafi have developed certain types of non-melanoma skin cancers. Tell your healthcare provider if you develop any new or changing skin lesions.

Increases in Cholesterol: You may have changes in your blood cholesterol levels. Your healthcare provider will do blood tests to check your cholesterol levels during your treatment with Jakafi.

The most common side effects of Jakafi include: low platelet count, low red blood cell counts, bruising, dizziness, headache.

These are not all the possible side effects of Jakafi. Ask your pharmacist or healthcare provider for more information. Tell your healthcare provider about any side effect that bothers you or that does not go away.

Before taking Jakafi, tell your healthcare provider about: all the medications, vitamins, and herbal supplements you are taking and all your medical conditions, including if you have an infection, have or had tuberculosis (TB), or have been in close contact with someone who has TB, have or had hepatitis B, have or had liver or kidney problems, are on dialysis, had skin cancer or have any other medical condition. Take Jakafi exactly as your healthcare provider tells you. Do not change or stop taking Jakafi without first talking to your healthcare provider. Do not drink grapefruit juice while on Jakafi.

Women should not take Jakafi while pregnant or planning to become pregnant, or if breast-feeding.

Full Prescribing Information, which includes a more complete discussion of the risks associated with Jakafi, is available at www.jakafi.com.

On November 1, 2017 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on developing gene and cell-based immunotherapies for cancer, reported that five abstracts highlighting data from the Company’s adoptive cell-based therapeutic programs have been accepted for presentation at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, December 9-12, in Atlanta (Press release, Ziopharm, NOV 1, 2017, View Source [SID1234521408]).

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Data from ongoing clinical and preclinical studies will be highlighted at ASH (Free ASH Whitepaper) including research, conducted with collaborators at The University of Texas MD Anderson Cancer Center and Intrexon Corporation (NYSE:XON), that further validates the Company’s Sleeping Beauty (SB) platform in chimeric antigen receptor (CAR) modified T cell therapy and demonstrates the potential for very rapid T-cell production (<2 days) with point-of-care (P-O-C) technology.

Presentations include long-term follow-up data from the initial clinical trial infusing first-generation SB-modified CD19-specific CAR+ T cells and interim data from the Company’s ongoing clinical trial of second-generation SB-modified CD19-specific CAR+ T cells that serves as a gateway to the trial infusing T cells manufactured under the P-O-C platform.

Additional abstracts include an update on the Company’s lentiviral approach to express CD33-specific CAR with a kill switch in patients with relapsed of refractory acute myeloid leukemia, as well as preclinical data supporting the use of genetically engineered regulatory T cells to treat graft versus host disease.

Details for ASH (Free ASH Whitepaper) presentations are as follows:

Title: Long Term Follow up after Adoptive Transfer of CD19-Specific CAR+T Cells Genetically Modified Via Non-Viral Sleeping Beauty System Following Hematopoietic Stem Cell Transplantation (HSCT)
Presenter: Partow Kebriaei, M.D.
Session Title: 801. Gene Therapy and Transfer: Poster I
Date and Time: Saturday, December 9, 2017, 5:30 — 7:30 p.m. ET
Publication ID: 2059
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2

Title: Shortening the Time to Manufacture CAR+ T Cells with Sleeping Beauty System Supports T-Cell Engraftment and Anti-Tumor Effects in Patients with Refractory CD19+ Tumors
Presenter: Partow Kebriaei, M.D.
Session Title: 801. Gene Therapy and Transfer: Poster I
Date and Time: Saturday, December 9, 2017, 5:30 — 7:30 p.m. ET
Publication ID: 2060
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2

Title: Genetically Engineered Regulatory T Cells for Treatment of Graft-Versus-Host-Disease
Presenter: Hanspeter Waldner, Ph.D.
Session Title: 701. Experimental Transplantation: Basic Biology, Pre-Clinical Models: Poster II
Date and Time: Sunday, December 10, 2017, 6:00 — 8:00 p.m. ET
Publication ID: 3176
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2

Title: CD19-Specific Chimeric Antigen Receptor-Modified T Cells with Safety Switch Produced Under “Point-Of-Care” Using the Sleeping Beauty System for the Very Rapid Manufacture and Treatment of B-Cell Malignancies
Presenter: Tim Chan, Ph.D.
Session Title: 614. Acute Lymphoblastic Leukemia: Therapy, excluding Transplantation: Poster
Date and Time: Saturday, December 9, 2017, 5:30 — 7:30 p.m. ET
Publication ID: 1324
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2

Title: Autologous T Cells Modified to Co-express CD33-Specific Chimeric Antigen Receptor and a Kill Switch for Treatment of CD33+ Acute Myeloid Leukemia
Presenter: Tim Chan, Ph.D.
Session Title: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster
Date and Time: Saturday, December 9, 2017, 5:30 — 7:30 p.m. ET
Publication ID: 1376
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2

On November 1, 2017 Verastem, Inc. (NASDAQ: VSTM), focused on discovering and developing drugs to improve the survival and quality of life of cancer patients, reported that four abstracts have been selected for presentation, including two oral presentations, at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) 2017 Annual Meeting being held December 9-12, 2017 in Atlanta (Press release, Verastem, NOV 1, 2017, View Source [SID1234521407]). Included among the oral presentations are the detailed results from the Phase 3 DUO study evaluating the efficacy and safety of duvelisib, a first-in-class oral dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, in patients with relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL). As previously announced, the DUO study achieved its primary endpoint with oral duvelisib monotherapy demonstrating superiority over ofatumumab for progression free survival (PFS) in patients with CLL/SLL.

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“Previously reported top-line data from the pivotal Phase 3 DUO study significantly favored duvelisib monotherapy over ofatumumab, an approved standard of care treatment for patients with relapsed or refractory CLL/SLL, achieving a statistically significant improvement in median PFS and a hazard ratio (HR) of 0.52 (p<0.0001), with a consistent and manageable safety profile," said Robert Forrester, President and Chief Executive Officer of Verastem. "We look forward to sharing the detailed results from the DUO study with the medical community at ASH (Free ASH Whitepaper) this year."

Verastem recently announced that a meeting was held with the U.S. Food and Drug Administration (FDA) regarding the regulatory path for duvelisib. Based on the meeting with, and written feedback from the FDA, Verastem intends to submit a New Drug Application (NDA) requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory CLL/SLL and accelerated approval for the treatment of patients with relapsed or refractory follicular lymphoma (FL). Along with the clinical data from the DUO study, the duvelisib NDA submission will also contain the favorable results from the Phase 2 DYNAMO study in double-refractory indolent non-Hodgkin’s lymphoma (iNHL), which also achieved its primary endpoint with an ORR of 46% (p<0.0001). In the subset of patients enrolled in the DYNAMO study with double-refractory FL (n=83), duvelisib demonstrated an ORR of 41%. The Company expects to submit the duvelisib NDA during the first quarter of 2018.

Details for the ASH (Free ASH Whitepaper) 2017 presentations are as follows:

Oral Presentations

Title: Results from the Phase 3 DUOTM Trial: A Randomized Comparison of Duvelisib Vs Ofatumumab in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma
Presenter: Ian Flinn, M.D., Ph.D., Sarah Cannon Research Institute
Abstract Number/Publication ID: 493
Session: 642. CLL: Therapy, excluding Transplantation: Targeting MRD Negative CLL with Combinations of Novel Agents and Chemoimmunotherapy Regimens, New Treatments; Sunday, December 10, 2017 from 4:30-6:00 PM ET
Date and Time: Sunday, December 10, 2017 at 4:30 PM ET
Location: Georgia World Congress Center, Building B, Level 5, Murphy BR 3-4

Title: In Vitro, In Vivo, and Parallel Phase I Evidence Support the Safety and Activity of Duvelisib, a PI3K-δ,γ Inhibitor, in Combination with Romidepsin or Bortezomib in Relapsed/Refractory T-Cell Lymphoma
Presenter: Steven Horwitz, M.D., Memorial Sloan Kettering Cancer Center
Abstract Number/Publication ID: 819
Session: 624. Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: T-Cell Lymphoma Clinical Studies; Monday, December 11, 2017 from 4:30-6:00 PM ET
Date and Time: Monday, December 11, 2017 at 5:00 PM ET
Location: Georgia World Congress Center, Building A, Level 4, Marcus Auditorium

Poster Presentations

Title: The Dual PI3K-δ,γ Inhibitor Duvelisib Stimulates Anti-Tumor Immunity and Enhances Efficacy of Immune Checkpoint and Co-Stimulatory Antibodies in a B Cell Lymphoma Model
Presenter: Jonathan Pachter, Ph.D., Verastem
Abstract Number/Publication ID: 1541
Session: 625. Lymphoma: Pre-Clinical—Chemotherapy and Biologic Agents: Poster I
Date and Time: Saturday, December 9, 2017 from 5:30-7:30 PM ET
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

Title: Combinatorial Inhibition of Focal Adhesion Kinase and BCL-2 in AML
Presenter: Xiangmeng Wang, Ph.D., MD Anderson Cancer Center
Abstract Number/Publication ID: 2653
Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster II
Date and Time: Sunday, December 10, 2017 from 6:00-8:00 PM ET
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

About Duvelisib
Duvelisib is a first-in-class investigational, dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells and T-cells. PI3K signaling may lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 Duvelisib is currently being evaluated in late- and mid-stage extension trials, including DUO, a randomized, Phase 3 monotherapy study in patients with relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL),4 and DYNAMO, a single-arm, Phase 2 monotherapy study in patients with refractory indolent non-Hodgkin lymphoma (iNHL).5 Both DUO and DYNAMO achieved their primary endpoints and Verastem intends to submit a New Drug Application (NDA) requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory CLL/SLL, and accelerated approval for the treatment of patients with relapsed or refractory follicular lymphoma (FL). Duvelisib is also being developed by Verastem for the treatment of peripheral T-cell lymphoma (PTCL), and is being investigated in combination with other agents through investigator-sponsored studies.6 Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

On November 1, 2017 TG Therapeutics, Inc. (NASDAQ:TGTX), reported that updated data for TGR-1202 (umbralisib), the Company’s once-daily PI3K delta inhibitor, and TG-1101 (ublituximab), the Company’s novel glycoengineered anti-CD20 monoclonal antibody, have been selected for presentation at the upcoming 59th American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, to be held December 9-12, 2017, at the Georgia World Congress Center in Atlanta, Georgia (Press release, TG Therapeutics, NOV 1, 2017, View Source [SID1234521406]). Abstracts are now available online and can be accessed on the ASH (Free ASH Whitepaper) meeting website at www.hematology.org . Clinical abstract highlights as well as the details of clinical and preclinical posters to be presented at ASH (Free ASH Whitepaper) are outlined below.

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Clinical Abstract Highlights:

TGR-1202 Integrated Analysis: 336 patients with relapsed/refractory Lymphoid Malignancies exposed to a TGR-1202 based regimen for upwards of 4+ years, demonstrated a favorable safety profile with infrequent Grade 3/4 adverse events prevalent amongst prior generation PI3K delta inhibitors: transaminitis ( < 3%), colitis ( < 1%), and pneumonitis ( < 0.5%)
Kinase Inhibitor Intolerance Study: In patients with Chronic Lymphocytic Leukemia (CLL) who are intolerant to prior BTK or PI3K delta inhibitor therapy, no patients (n=22) discontinued due to TGR-1202 intolerance with a median follow-up of 6 months
TG-1101 + TGR-1202 + PD-1 Triplet: 60% ORR (3 of 5) observed in BTK refractory CLL patients treated with the triple combination of TGR-1202 plus TG-1101 plus pembrolizumab
Poster presentations at the ASH (Free ASH Whitepaper) 2017 meeting include the following:

Sunday December 10, 2017:

Title: Phase I/II Study of Pembrolizumab in Combination with Ublituximab (TG-1101) and Umbralisib (TGR-1202) in Patients with Relapsed/Refractory CLL
Abstract Number: 3010
Session: 642. CLL: Therapy, excluding Transplantation: Poster II
Date and Time: Sunday, December 10, 2017; 6:00 PM – 8:00 PM ET
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
Presenter: Anthony R. Mato, MD, University of Pennsylvania, Philadelphia, PA

Title: Umbralisib/TGR-1202 as a Novel Dual PI3K/CK1 Inhibitor Has a Unique Therapeutic Role in Silencing Oncogenes in Aggressive Lymphomas
Abstract Number: 2809
Session: 625. Lymphoma: Pre-Clinical—Chemotherapy and Biologic Agents: Poster II
Date and Time: Sunday, December 10, 2017; 6:00 PM – 8:00 PM ET
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
Presenter: Ipsita Pal, PhD, Columbia University Medical Center, New York, NY

Title: Differential Regulation of T Cells By PI3K Delta Inhibitors in a CLL Murine Model
Abstract Number: 3009
Session: 642. CLL: Therapy, excluding Transplantation: Poster II
Date and Time: Sunday, December 10, 2017; 6:00 PM – 8:00 PM ET
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
Presenter: Kamira K. Maharaj, BS, Moffit Cancer Center, Tampa, FL
Monday, December 11, 2017:

Title: An Integrated Safety Analysis of the Next Generation PI3Kδ Inhibitor Umbralisib (TGR-1202) in Patients with Relapsed/Refractory Lymphoid Malignancies
Abstract Number: 4037
Session: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma—Clinical Studies: Poster III
Date and Time: Monday, December 11, 2017; 6:00 PM – 8:00 PM ET
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
Presenter: Matthew S. Davids, MD, Dana Farber Cancer Institute, Boston, MA

Title: KI Intolerance Study: A Phase 2 Study to Assess the Safety and Efficacy of Umbralisib (TGR-1202) in Patients with Chronic Lymphocytic Leukemia (CLL) Who Are Intolerant to Prior BTK or PI3K-δ Inhibitor Therapy
Abstract Number: 4314
Session: 642. CLL: Therapy, excluding Transplantation: Poster III
Date and Time: Monday, December 11, 2017; 6:00 PM – 8:00 PM ET
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
Presenter: Anthony R. Mato, MD, University of Pennsylvania, Philadelphia, PA

Title: PI3K-Delta Inhibitors Induce Primary Monocyte Cytotoxicity but Do Not Alter Monocyte Differentiation
Abstract Number: 4284
Session: 641. CLL: Biology and Pathophysiology, excluding Therapy: Poster III
Date and Time: Monday, December 11, 2017; 6:00 PM – 8:00 PM ET
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
Presenter: Daphne Friedman, MD, Durham VA-Duke University Medical Center, Durham, NC
The above referenced abstracts can be viewed online through the ASH (Free ASH Whitepaper) meeting website at www.hematology.org. Following each presentation, the data presented will be available on the Publications page of the Company’s website at www.tgtherapeutics.com.

TG THERAPEUTICS INVESTOR & ANALYST EVENT

TG Therapeutics will also host a reception on Sunday, December 10, 2017 beginning at 8:00pm ET with featured presentations beginning promptly at 8:15pm ET. The event will take place at the Ritz Carlton Atlanta (Downtown) in the Congress Room. This event will be webcast live and will be available on the Events page, located within the Investors & Media section of the Company’s website at www.tgtherapeutics.com, as well as archived for future review. This event will also be broadcast via conference call. To access the conference line, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), and reference Conference Title: TG Therapeutics December 2017 Investor & Analyst Event.

On November 1, 2017 Stemline Therapeutics, Inc. (Nasdaq: STML), a clinical stage biopharmaceutical company developing novel therapeutics for difficult to treat cancers, reported that the pivotal Phase 2 trial of SL-401 in blastic plasmacytoid dendritic cell neoplasm (BPDCN) has met its primary endpoint (Press release, Stemline Therapeutics, NOV 1, 2017, View Source [SID1234521405]). Based on feedback from the U.S. Food and Drug Administration (FDA), Stemline remains on track to begin submission of its Biologics License Application (BLA) in the 4Q17-1Q18 timeframe.

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SL-401 has been granted Breakthrough Therapy Designation (BTD) by the FDA for the treatment of BPDCN, and Orphan Drug Designation (ODD) by the FDA and EU for the treatment of patients with BPDCN and acute myeloid leukemia (AML).

Pivotal Trial

The pivotal Phase 2 trial of SL-401 is the largest multicenter prospective study ever conducted in BPDCN. The trial enrolled 45 BPDCN (32 first-line, 13 relapsed/refractory) patients at 7 sites in the U.S. The trial consisted of 3 Stages: Stage 1 (lead-in, dose escalation), Stage 2 (expansion), and Stage 3 (pivotal, confirmatory).

Stage 3: Design

Stage 3 of the Phase 2 trial was designed with input from the FDA to provide the pivotal, confirmatory evidence of efficacy of SL-401 in BPDCN. First-line-only BPDCN patients received SL-401 intravenously at 12 mcg/kg/day on days 1-5 of a 21-day cycle. The primary endpoint of Stage 3 was the rate of CR, defined per protocol as CR + CRc + CRi (CR = complete response; CRc = clinical complete response: absence of gross disease with minimal residual skin abnormality; CRi = CR with incomplete hematologic recovery) by investigator assessment. Statistical significance is achieved if the lower bound of the 95% confidence interval (CI) of the primary endpoint exceeds 10%.

Stage 3: Top-Line Results

In Stage 3 of the trial, 13 first-line BPDCN patients were enrolled. Stage 3 met its primary endpoint, with a CR rate of 54% (7/13) (95% CI: 25.1, 80.8). Overall response rate (ORR) was 77% (10/13). 46% (6/13) of patients were bridged to stem cell transplant (SCT) following remission on SL-401. 86% (6/7) of complete responders remain relapse-free at 5+ to 8+ months, ongoing.

All Stages (1, 2, and 3): Top-Line Results

Across all stages, lines, and doses in the trial (n=45 BPDCN patients; 32 first-line, 13 relapsed/refractory), the CR rate was 60% (27/45) and the ORR was 82% (37/45). In first-line patients who received SL-401 at all tested doses (n=32 patients), the CR rate was 69% (22/32) and the ORR was 88% (28/32). 41% (13/32) of first-line patients who received SL-401 at all tested doses have been bridged to SCT following remission on SL-401.



All SL-401 Trials: Top-Line Safety Results

The most common treatment-related adverse events (TRAEs) with SL-401 across BPDCN and other clinical trials (acute myeloid leukemia (AML), myeloproliferative neoplasms (MPN), and multiple myeloma) (n=148 patients) were hypoalbuminemia (47%), aspartate aminotransferase increase (46%), alanine aminotransferase increase (45%), nausea (28%), and thrombocytopenia (28%). Capillary leak syndrome (CLS), a well-documented side effect, occurred in 19% of patients, of which 2% (3/148) were grade 5, as previously reported.

Andrew A. Lane, M.D., Ph.D., Director of the BPDCN Center at the Dana-Farber Cancer Institute and Assistant Professor of Medicine at Harvard Medical School in Boston, and a co-investigator on the study, commented, “BPDCN is a devastating malignancy for which there are no approved therapies. Due to the increasing awareness around BPDCN and the promise of novel targeted agents such as SL-401, we have built a new BPDCN center at Dana-Farber to focus on this historically poorly understood, yet increasingly appreciated, patient population with unmet need. SL-401 has demonstrated efficacy with meaningful clinical benefit in BPDCN while maintaining a manageable safety profile, particularly notable in this predominantly older population, representing a major advance in the management of BPDCN. Also, given the presence of CD123 on additional aggressive hematologic cancers, we are also exploring SL-401 in combination with other agents in clinical trials of high risk MDS and AML.”

Ivan Bergstein, MD, CEO of Stemline, commented, “The successful completion of the largest prospective clinical trial ever conducted in BPDCN, is a major milestone for Stemline, our investigators, and most importantly, patients and their families. We would like to thank and congratulate all those involved in this groundbreaking trial. We look forward to working closely with regulatory authorities in an effort to provide SL-401 to patients as quickly as possible. In parallel, our commercial team continues to ramp-up activities setting the stage, if SL-401 is approved, for a successful launch.”

Conference Call and Webcast

Stemline Therapeutics will host a conference call and audio webcast this morning, Tuesday, October 31, 2017 at 8:30 AM ET. Interested participants and investors may access the conference call by dialing 844-389-8660 (U.S./Canada) or 478-219-0408 (International) and referencing conference ID: 1686609. An audio webcast can also be accessed via the Investor Relations tab of the Stemline Therapeutics website at View Source

About BPDCN

Please visit www.bpdcninfo.com and the BPDCN awareness booth (#3143) at ASH (Free ASH Whitepaper) 2017