On November 1, 2018 Incyte (Nasdaq:INCY) reported that multiple abstracts, including data from its clinical development programs for ruxolitinib (Jakafi), pemigatinib and INCB50465 will be presented at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2018 in San Diego, California, from December 1-4, 2018 (Press release, Incyte, NOV 1, 2018, View Source [SID1234530536]).

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Key data presented at ASH (Free ASH Whitepaper) 2018 will include results from the pivotal REACH1 trial evaluating ruxolitinib in combination with corticosteroids for the treatment of patients with acute graft-versus-host disease (GVHD) who have had an inadequate response to corticosteroids, which have been accepted for oral presentation. Additionally, new data from the Phase 2 study evaluating INCB50465 in combination with ruxolitinib in patients with myelofibrosis (MF) and initial data from the Phase 2 fight-203 study evaluating pemigatinib in patients with myeloproliferative neoplasms (MPNs) with activating FGFR1 translocations have been accepted for oral presentation.

"We are looking forward to highlighting our later-stage, targeted therapy portfolio at this year’s ASH (Free ASH Whitepaper) Annual Meeting," said Steven Stein, M.D., Chief Medical Officer, Incyte. "Specifically, we are pleased to present results from our REACH1 study, which formed the basis of our supplemental new drug application for ruxolitinib as a treatment for acute GVHD that is currently under Priority Review by the FDA, and new data from our INCB50465 and pemigatinib clinical development programs, which further underscore our leadership in MPNs."

Key abstract presentations include:

Ruxolitinib (Jakafi)

Oral Presentations

Safety and Efficacy of Combined Ruxolitinib and Thalidomide in Patients with Myelofibrosis: Initial Results of a Phase 2 Study (Abstract #354)

Sunday, December 2, 2018, 9:30-11:00 a.m., Manchester Grand Hyatt, Seaport Ballroom F, Oral Session 634, Myeloproliferative Syndromes: Clinical: Addressing Areas of Unmet Need in Prognostic Assessments and Therapy for MPNs
Results from REACH1, a Single-arm Phase 2 Study of Ruxolitinib in Combination with Corticosteroids for the Treatment of Steroid-Refractory Acute Graft-vs-Host Disease (Abstract #601)

Monday, December 3, 2018, 7:00-8:30 a.m., Manchester Grand Hyatt, Grand Hall A, Oral Session 722, Clinical Allogeneic Transplantation: Acute and Chronic GVHD
A Phase 2 Study of Ruxolitinib with Chemotherapy in Children with Philadelphia Chromosome-like Acute Lymphoblastic Leukemia (INCB18424-269/AALL1521): Dose-Finding Results from the Part 1 Safety Phase (Abstract #555)

Monday, December 3, 2018, 7:00-8:30 a.m., San Diego Convention Center, Ballroom 20A, Oral Session 614, Acute Lymphoblastic Leukemia: Therapy, excluding Transplantation: Targeted Therapy in ALL: Immunotherapy and Beyond
RUXOPEG, a Multi-Center Bayesian Phase ½ Adaptive Randomized Trial of the Combination of Ruxolitinib and Pegylated Interferon Alpha 2a in Patients with Myeloproliferative Neoplasms-Associated Myelofibrosis*(Abstract #581)

Monday, December 3, 2018, 7:00-8:30 a.m., Manchester Grand Hyatt, Grand Hall D, Oral Session 634, Myeloproliferative Syndromes: Clinical: Interferon Therapy and Mutational Analysis in the MPNs
Poster Sessions

Updated Results from An Open-Label, Multicenter, Expanded Treatment Protocol (ETP) Phase (Ph) 3b Study Of Ruxolitinib (Rux) In Patients (Pts) With Polycythemia Vera (PV) Who Are Hydroxyurea (HU) Resistant Or Intolerant And For Whom No Alternative Treatments Are Available (Abstract #1774)

Saturday, December 1, 2018, 6:15-8:15 p.m., San Diego Convention Center, Hall GH, Poster Session 634, Myeloproliferative Syndromes: Clinical: Poster I
Characteristics Associated with Hydroxyurea Treatment Change in Patients with Polycythemia Vera: An Analysis from the REVEAL Study (Abstract #1770)

Saturday, December 1, 2018, 6:15-8:15 p.m., San Diego Convention Center, Hall GH, Poster Session 634, Myeloproliferative Syndromes: Clinical: Poster I
Real-World Risk Assessment and Treatment of Patients with Myelofibrosis at Community Oncology Practices in the United States(Abstract #1765)

Saturday, December 1, 2018, 6:15-8:15 p.m., San Diego Convention Center, Hall GH, Poster Session 634, Myeloproliferative Syndromes: Clinical: Poster I
Impact of Myeloproliferative Neoplasms on Patients’ Employment and Income: Findings from the Living with MPN Survey(Abstract #2250)

Saturday, December 1, 2018, 6:15-8:15 p.m., San Diego Convention Center, Hall GH, Poster Session 902, Health Services Research – Malignant Diseases: Poster I
Elevated White Blood Cell levels and Thrombotic Events in Patients with Polycythemia Vera: A Real-World Analysis of Veterans Health Administration Data (Abstract #1758)

Saturday, December 1, 2018, 6:15-8:15 p.m., San Diego Convention Center, Hall GH, Poster Session 634, Myeloproliferative Syndromes: Clinical: Poster I
Correlation between MPN-SAF TSS and EORTC QLQ-C30 Scores in Patients with PV: Data from the REVEAL Study (Abstract #2259)

Saturday, December 1, 2018, 6:15-8:15 p.m., San Diego Convention Center, Hall GH, Poster Session 902, Health Services Research – Malignant Diseases: Poster I
Long-term Efficacy and Safety (5 Years) in RESPONSE, a Phase 3 Study Comparing Ruxolitinib (rux) With Best Available Therapy (BAT) in Hydroxyurea (HU)-resistant/intolerant Patients (pts) With Polycythemia Vera (PV)

Saturday, December 1, 2018, 6:15-8:15 p.m., San Diego Convention Center, Hall GH, Poster Session 634, Myeloproliferative Syndromes: Clinical: Poster I
Ruxolitinib for the Treatment of Inadequately Controlled Polycythemia Vera Without Splenomegaly: 156-Week Follow-Up From the Phase 3 RESPONSE-2 Study (Abstract #1754)

Saturday, December 1, 2018, 6:15-8:15 p.m., San Diego Convention Center, Hall GH, Poster Session 634, Myeloproliferative Syndromes: Clinical: Poster I
Disease and Clinical Characteristics of Patients with Essential Thrombocythemia Enrolled in the MOST Study (Abstract #4306)

Monday, December 3, 2018, 6:00-8:00 p.m., San Diego Convention Center, Hall GH, Poster Session 634, Myeloproliferative Syndromes: Clinical: Poster III
Disease Characteristics of Minority Patient Populations with Polycythemia Vera: An Analysis from the REVEAL Study (Abstract #4735)

Monday, December 3, 2018, 6:00-8:00 p.m., San Diego Convention Center, Hall GH, Poster Session 902, Health Services Research – Malignant Diseases: Poster III
Real-World Management of Myelofibrosis with Ruxolitinib: Initial Analysis of an Italian Observational Study (ROMEI) (Abstract #4312)

Monday, December 3, 2018, 6:00-8:00 p.m., San Diego Convention Center, Hall GH, Poster Session 634, Myeloproliferative Syndromes: Clinical: Poster III
Impact of Myeloproliferative Neoplasms (MPNs) on Health-Related Quality of Life (HRQOL) and Medical Resource Utilization: Results from the MERGE Registry (Abstract #4311)

Monday, December 3, 2018, 6:00-8:00 p.m., San Diego Convention Center, Hall GH, Poster Session 634: Myeloproliferative Syndromes: Clinical: Poster III
INCB50465 (PI3Kδ)

Oral Presentation

A Phase 2 Study of the Safety and Efficacy of INCB050465, a Selective PI3Kδ Inhibitor, in Combination with Ruxolitinib in Patients with Myelofibrosis (Abstract #353)

Sunday, December 2, 2018, 9:30-11:00 a.m., Manchester Grand Hyatt, Seaport Ballroom F, Oral Session 634, Myeloproliferative Syndromes: Clinical: Addressing Areas of Unmet Need in Prognostic Assessments and Therapy for MPNs
Poster Session

Cell-of-Origin Subtype Prediction of Diffuse Large B-cell Lymphoma Using Gene Expression and Proteomic Data (Abstract #1712)

Saturday, December 1, 2018, 6:15-8:15 p.m., San Diego Convention Center, Hall GH, Poster Session 627, Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Retrospective/Observational Studies: Poster I
Pemigatinib (INCB054828)

Oral Presentation

Interim Results from fight-203, a Phase 2, Open-Label, Multicenter Study Evaluating the Efficacy and Safety of Pemigatinib (INCB054828) in Patients with Myeloid/Lymphoid Neoplasms with Rearrangement of Fibroblast Growth Factor Receptor 1 (FGFR1) (Abstract #690)

Monday, December 3, 2018, 10:30-12:00 p.m., Manchester Grand Hyatt, Grand Hall A, Oral Session 634, Myeloproliferative Syndromes: Clinical: Emerging Therapies and Prognostic Scoring in Myelofibrosis and Other MPNs
Itacitinib

Plasma Biomarker Association with Response in Acute GVHD Subjects Treated With the Combination of Itacitinib and Corticosteroids in a Phase 1 Clinical Trial (Abstract #4559)

Monday, December 3, 2018, 6:00-8:00 p.m., San Diego Convention Center, Hall GH, Poster Session 722, Clinical Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Poster III
Ponatinib

Efficacy and Safety of Ponatinib in CML and Ph+ ALL Patients in Real World Clinical Practice – Data from a Belgian Registry (Abstract #1744)

Saturday, December 1, 2018, 6:15-8:15 p.m., San Diego Convention Center, Hall GH, Poster Session 632, Chronic Myeloid Leukemia: Therapy: Poster I
Preclinical

The Persistent Survival of MPN Cells to JAK2 Inhibition is Dependent on SHP2 Activity, Which May Provide a Therapeutic Target to Enhance Current Anti-MPN Therapies (Abstract #3064)

Sunday, December 2, 2018, 6:00-8:00 p.m., San Diego Convention Center, Hall GH, Poster Session 635, Myeloproliferative Syndromes: Basic Science: Poster II
Itacitinib, a JAK1 Selective Inhibitor Preserves Graft-versus-Leukemia (GVL), Enhances Survival and is Highly Efficacious in a MHC-mismatched Mouse Model of Acute GvHD (Abstract #4522)

Monday, December 3, 2018, 6:00-8:00 p.m., San Diego Convention Center, Hall GH, Poster Session 701, Experimental Transplantation: Basic Biology, Pre-Clinical Models Poster III
Ruxolitinib, a JAK1/JAK2 Selective Inhibitor is Highly Efficacious in Corticosteroid Untreated and Refractory MHC-mismatched Mouse Model of Acute GvHD (Abstract 4523)

Monday, December 3, 2018, 6:00-8:00 p.m., San Diego Convention Center, Hall GH, Poster Session 701, Experimental Transplantation: Basic Biology, Pre-Clinical Models: Poster III
Full session details and data presentation listings for ASH (Free ASH Whitepaper) 2018 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 polycythemia vera (PV) who have had an inadequate response to or are intolerant of hydroxyurea.

Jakafi is also indicated 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 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. Ruxolitinib is also being evaluated in patients with acute and chronic GVHD who have an inadequate response to corticosteroids in the REACH2 and REACH3 clinical studies, respectively. It is expected that these two pivotal studies will complete in 2019, and could support additional regulatory submissions, in the U.S. by Incyte and ex-U.S. by Novartis, in 2020.

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, 2018 GT Biopharma, Inc. (OTCQB: GTBP and Euronext Paris GTBP.PA) ("GT Biopharma" or the "Company"), an immuno-oncology biotechnology company focused on innovative treatments based on the company’s proprietary NK-engager and Bispecific Antibody Drug Conjugate platforms, reported that its Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) is now open and it is authorized to initiate a first-in-human Phase 1 study with GTB-3550 (OXS-3550), its first-in-class (TriKE), for the treatment of acute myelogenous leukemia (AML), myelodysplatic syndrome (MDS) and mastocytosis (Press release, GT Biopharma , NOV 1, 2018, View Source [SID1234539521]). The study will be led by Principal Investigator, Sarah A. Cooley, MD, MS, Associate Professor, Division of Hematology, Oncology and Transplantation at Masonic Cancer Center, University of Minnesota.

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"GTB-3550 is a protein immune engager that binds to natural killer (NK) cells and targets them specifically to leukemia cells," said renowned NK cell expert, Jeffrey Miller, MD, Deputy Director, Masonic Cancer Center, University of Minnesota. "Our team has been working on the optimal construct for years and we are excited to see it is ready for clinical testing. In addition, the same TriKE protein will deliver an interleukin-15 stimulus, a growth factor that makes NK cells proliferate and be more active."

"The clinical trials team at the University of Minnesota is excited to commence the Phase 1 trial testing this novel immunotherapeutic agent, GTB-3550," said Dr. Cooley. "Building on over a decade of successful trials using NK cell infusions from related donors to kill tumors, Masonic Cancer Center researchers designed this protein to activate a patient’s own NK cells and, importantly, to direct them to specifically kill CD33+ tumor cells. The pre-clinical data are extraordinarily compelling, and success with GTB-3550 in this study will allow us to develop a broad pipeline of TriKE agents against different tumor targets."

This single center, first-in-human Phase 1 clinical trial of GTB-3550 will enroll up to 60 subjects with CD33-expressing high risk for refractory/relapsed AML, MDS, or advanced systemic mastocytosis. Subjects will receive a single course of GTB-3550 TriKE given as 3 weekly treatment blocks. Each block consists of four consecutive 24-hour continuous infusions of GTB-3550 TriKE followed by a 72-hour break after Block #1 and #2. Disease response will be assessed by bone marrow biopsy performed between Day 21 and Day 42 after the start of the 1st infusion. Follow-up for response and survival continues through 6 months from treatment start. The primary objective from the Phase 1 dose finding portion of the study will be to identify the maximum tolerated dose (MTD) of GTB-3550 TriKE defined as the dose level that most closely corresponds to a dose limiting toxicity rate (DLT) of 20%. The primary objective from the Phase 2 extended portion of the study will be the potential efficacy of GTB-3550 TriKE, measured using rates of complete and partial remission. Subjects experiencing clinical benefit and no unacceptable side effects may be considered for a 2nd course of GTB-3550 TriKE on a compassionate basis.

"The opening of this IND allows us to proceed with our first-in-class TriKE, Phase 1 study and importantly, marks a significant step forward in our clinical development strategy of our potentially revolutionary product candidate," commented Raymond Urbanski, M.D., Ph.D., Chief Executive Officer of GT Biopharma. "We are privileged to be advancing this program with the world’s leading experts in NK cell-based therapy."

GT Biopharma has an exclusive worldwide license agreement with the University of Minnesota to further develop and commercialize cancer therapies using proprietary TriKE technology developed by researchers at the university to target NK cells to cancer.

About Acute Myelogenous Leukemia (AML)

AML is the most common form of adult leukemia with 21,000 new cases expected in 2018 alone, according to the American Cancer Society. AML patients typically receive frontline therapy, most commonly chemotherapy, which includes cytarabine and an anthracycline, a therapy that has not changed in over 40 years. However, there remains a significant unmet need in these therapies with about half of AML patients experiencing relapses or requiring alternative therapies. The Company is developing GTB-3550 to serve as a relatively safe, cost-effective, and easy-to-use therapy for resistant/relapsing AML and could also be combined with chemotherapy as frontline therapy thus targeting the larger patient population.

About Myelodysplastic Syndrome (MDS)

Myelodysplastic syndromes (MDS) are conditions that can occur when the blood-forming cells in the bone marrow become abnormal, leading to low numbers of one or more types of blood cells. There are several different types of MDS, based on how many types of blood cells are affected and other factors, although the most common finding in MDS is a shortage of red blood cells (anemia). The number of people with MDS diagnosed in the U.S. each year is estimated to be ~10,000. MDS is uncommon before age 50 and is most commonly diagnosed in people in their 70s. In about 1 in 3 patients, MDS can progress to AML, a rapidly growing cancer of bone marrow cells.

About Mastocytosis

Mastocytosis is a rare disorder characterized by abnormal accumulations of mast cells in the skin, bone marrow, and internal organs (liver, spleen, gastrointestinal tract and lymph nodes). Cases beginning during adulthood tend to be chronic and involve the bone marrow in addition to the skin, whereas, during childhood, the condition is often marked by skin manifestations with no internal organ involvement and can often resolve during puberty. In most adult patients, mastocytosis tends to be persistent, and may progress into a more advanced category in a minority of patients. Mastocytosis affects both males and females and can begin during childhood or adulthood. In children, 80% of cases appear during the first year of life, and the majority is limited to the skin. Adults who develop mastocytosis more often have systemic forms of the disease. Cutaneous forms of the disease account for less than 5% of adult cases. An estimate of prevalence from a recent population-based study is approximately 1 case per 10,000 people.

About GTB-3550

GTB-3550 (OXS-3550) is the Company’s first Tri-specific Killer Engager (TriKE) product candidate being initially developed for the treatment AML. GTB-3550 is a single-chain, tri-specific scFv recombinant fusion protein conjugate composed of the variable regions of the heavy and light chains of anti-CD16 and anti-CD33 antibodies and a modified form of IL-15. When the NK stimulating cytokine human IL-15 is used as a crosslinker between the two scFvs, it provides a self-sustaining signal that activates NK cells and enhances their ability to kill. We intend to study this anti-CD16-IL-15-anti-CD33 tri-specific killer engager, or TriKE, in CD33 positive leukemias, a marker expressed on tumor cells in AML, myelodysplastic syndrome, or MDS, and other hematopoietic malignancies. CD33 is primarily a myeloid differentiation antigen with endocytic properties broadly expressed on AML blasts and, possibly, some leukemic stem cells. CD33 or Siglec-3 (sialic acid binding Ig-like lectin 3, SIGLEC3, SIGLEC3, gp67, p67) is a transmembrane receptor expressed on cells of myeloid lineage. It is usually considered myeloid-specific, but it can also be found on some lymphoid cells. The anti-CD33 antibody fragment that will be used for these studies was derived from the M195 humanized anti-CD33 scFV and has been used in multiple human clinical studies. It has been exploited as target for therapeutic antibodies for many years. Improved survival seen in many patients when the antibody-drug conjugate gemtuzumab was added to conventional chemotherapy validates this approach. GT Biopharma believes that GTB-3550 could serve as a relatively safe, cost-effective, and easy-to-use therapy for resistant/relapsing AML and could also be combined with chemotherapy as frontline therapy thus targeting the larger patient population.

On November 1, 2018 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that updated data for umbralisib (TGR-1202), the Company’s once-daily PI3K delta inhibitor, and ublituximab (TG-1101), the Company’s novel glycoengineered anti-CD20 monoclonal antibody, have been selected for presentation at the upcoming 60thAmerican Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting and exposition, to be held December 1-4, 2018, at the San Diego Convention Center in California (Press release, TG Therapeutics, NOV 1, 2018, View Source [SID1234532243]). Abstracts are now available online and can be accessed on the ASH (Free ASH Whitepaper) meeting website at www.hematology.org. Abstract highlights and presentation details are outlined below.

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Abstract Highlights:

Umbralisib + Ublituximab + Pembrolizumab Triple Therapy Oral Presentation:
89% ORR (8 of 9) observed in relapsed or refractory Chronic Lymphocytic Leukemia (CLL) patients with 75% ORR (3 of 4) in BTK refractory CLL patients
Notably 2 of 4 BTK-refractory CLL patients achieved a response to umbralisib plus ublituximab ("U2") alone, prior to the addition of pembrolizumab
50% ORR (2 of 4) observed in patients with Richter’s Transformation (RT)
Both responders were ibrutinib refractory and achieved durable Complete Responses (CRs) (time on therapy 15+ months and 7+ months)

Umbralisib + Ublituximab + Bendamustine Triple Therapy Poster Presentation:
85% ORR (11 of 13) observed in relapsed or refractory Follicular Lymphoma (FL) patients, including 54% CRs
48% ORR (12 of 25) observed in relapsed or refractory Diffuse Large B-cell Lymphoma (DLBCL) patients, including 32% CRs
Oral Presentation Details:

Title: Phase I/II Study of Umbralisib (TGR-1202) in Combination with Ublituximab (TG-1101) and Pembrolizumab in Patients with Relapsed/Refractory CLL and Richter’s Transformation
Publication Number: 297
Oral Session: 642. CLL: Therapy, excluding Transplantation: Cellular Therapy and Immunomodulation in CLL
Session Date and Time: Sunday, December 2, 2018; 7:30 AM – 9:00 AM PT
Presentation Time: 8:00 AM PT
Location: Marriott Marquis San Diego Marina, Pacific Ballroom 20
Presenter: Anthony R. Mato, MD, Memorial Sloan-Kettering Cancer Center, New York, NY
Poster Presentation Details:

Title: Combination of Umbralisib, Ublituximab, and Bendamustine Is Safe and Highly Active in Patients with Advanced Diffuse Large B-Cell Lymphoma and Follicular Lymphoma
Abstract Number: 4197
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Poster III
Date and Time: Monday, December 3, 2018; 6:00 PM – 8:00 PM PT
Location: San Diego Convention Center, Hall GH
Presenter: Matthew A. Lunning, DO, University of Nebraska Medical Center, Omaha, NE
Following each presentation, the data presented will be available on the Publications page of the Company’s website at View Source

TG THERAPEUTICS INVESTOR & ANALYST EVENT

TG Therapeutics will also host a reception on Sunday, December 2, 2018 beginning at 7:30 PM PT with featured presentations beginning promptly at 8:00 PM PT. The event will take place at the Marriott Gaslamp in San Diego California. The 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 View Source, 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 TherapeuticsDecember 2018 Investor & Analyst Event.

On November 1, 2018 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported business highlights and financial results for the third quarter ended September 30, 2018 (Press release, Agios Pharmaceuticals, NOV 1, 2018, View Source [SID1234530489]).

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"On the heels of the third quarter U.S. approval of TIBSOVO, our first wholly owned precision medicine, we remain focused on executing on our remaining 2018 milestones and continuing to create value from our portfolio," said David Schenkein, M.D., chief executive officer at Agios. "Based on regulatory discussions, we are accelerating our frontline strategy in IDH1m AML with the planned submission of an sNDA expanding TIBSOVO’s label to newly diagnosed AML patients not eligible for standard treatment and a shorter enrollment timeline for the Phase 3 AGILE trial. In addition, we continue to activate new sites globally for our pivotal PK deficiency program and accrue patients in the dose-escalation portion of the Phase 1 study in MTAP-deleted tumors. We believe these programs coupled with our robust preclinical pipeline, support our next phase of growth toward becoming a fully integrated, sustainable biopharmaceutical company."

THIRD QUARTER & RECENT 2018 HIGHLIGHTS

Launched TIBSOVO (ivosidenib) for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by a test approved by the U.S. Food and Drug Administration (FDA).
Awarded the U.S. Prix Galien Award for Best Pharmaceutical Product of 2018 for IDHIFA (enasidenib), an isocitrate dehydrogenase-2 (IDH2) inhibitor indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an IDH2 mutation as detected by a test approved by the FDA. Each year, the Prix Galien USA Committee recognizes outstanding achievements in improving the human condition through the development of innovative therapies.
Reached agreement with the FDA to submit a supplemental new drug application (sNDA) for single agent TIBSOVO in newly diagnosed AML patients with an IDH1 mutation who are not eligible for standard treatment.
Reached agreement with the FDA that event free survival (EFS) is an acceptable primary endpoint for the Phase 3 AGILE trial of ivosidenib combination with azacitidine in newly diagnosed AML patients with an IDH1 mutation who are ineligible for intensive chemotherapy. Full enrollment for AGILE is now expected to complete in 2020 vs. previous guidance of 2021.
Received global rights to AG-881, a brain-penetrant, pan-IDH inhibitor that was previously part of a joint worldwide collaboration with Celgene.
Submitted an investigational new drug (IND) application for AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies.
Announced that effective February 1, 2019, CEO David Schenkein, M.D., will transition to the role of executive chairman of the board of directors and Jacqualyn ("Jackie") Fouse, Ph.D., will succeed Dr. Schenkein as Agios’ next chief executive officer.
KEY UPCOMING MILESTONES

The company expects to achieve the following near-term milestones:

Cancer:

Submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for TIBSOVO for the treatment of adult patients with R/R AML with an IDH1 mutation by year end 2018.
Support, in conjunction with Celgene, the initiation of HOVON 150, an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib with standard induction and consolidation chemotherapy followed by a maintenance therapy period in frontline AML patients with an IDH1 or IDH2 mutation, respectively, by year end 2018.
Submit an sNDA to the FDA for TIBSOVO for the treatment of patients with newly diagnosed AML with an IDH1 mutation who are not eligible for standard therapy by the end of January 2019.
Rare Genetic Diseases:

Initiate a Phase 2 proof-of-concept trial of mitapivat in thalassemia by year end 2018.
FOURTH QUARTER CLINICAL DATA PRESENTATIONS

Updated data from Phase 1 trial of AG-881 in solid tumors, including glioma, has been accepted as an oral presentation at the 2018 Society for Neuro-Oncology (SNO) Annual Meeting on November 15-18 in New Orleans.
Updated data from the ongoing Phase 1 trial of single agent ivosidenib in IDH1m hematologic malignancies in a subset of patients with newly diagnosed AML not eligible for standard therapy has been accepted as an oral presentation at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition on December 1-4 in San Diego.
Updated data from the ongoing Phase 1 combination trial of ivosidenib or enasidenib with standard-of-care intensive chemotherapy in patients with newly diagnosed AML with an IDH2 or IDH1 mutation also been accepted as an oral presentation at ASH (Free ASH Whitepaper).
Updated data in myelodysplastic syndrome (MDS) from the ongoing Phase 1 study of single agent ivosidenib in IDH1m hematologic malignancies has been accepted as a poster presentation at ASH (Free ASH Whitepaper).
THIRD QUARTER 2018 FINANCIAL RESULTS

Revenue for the quarter ended September 30, 2018 was $15.2 million, which includes $4.5 million of net product revenue from U.S. sales of TIBSOVO, $8.7 million of collaboration revenue and $2.0 million of royalty revenue from net global sales of IDHIFA under our collaboration agreements with Celgene. Revenue for the quarter ended September 30, 2017 was $11.4 million and consisted of $10.6 million of collaboration revenue and $0.7 million of royalty revenue under our agreements with Celgene. The year-over-year increase in total revenue for the third quarter was primarily driven by U.S. sales of TIBSOVO and royalty revenue from U.S. sales of IDHIFA, offset by a decrease in collaboration revenue recognized during the quarter.

Cost of sales for the quarter ended September 30, 2018 were $0.7 million and relate to manufacturing costs associated with TIBSOVO sales.

Research and development (R&D) expenses were $82.6 million, including $13.4 million of stock-based compensation expense, for the quarter ended September 30, 2018, compared to $72.9 million, including $7.6 million in stock-based compensation expense, for the comparable period in 2017. The increase in R&D expense was primarily attributable to start-up costs for the mitapivat (AG-348) pivotal program in PK deficiency and IND enabling activities for AG-636, our DHODH inhibitor. R&D expense also increased as a result of ongoing research efforts across our discovery platform programs.

Sales, general and administrative (SG&A) expenses were $31.1 million, including $10.8 million of stock-based compensation expense, for the quarter ended September 30, 2018, compared to $17.5 million, including $4.6 million of stock-based compensation expense, for the quarter ended September 30, 2017. The increase in SG&A expense was primarily attributable to the growth in our U.S. commercial organization to support the launch of TIBSOVO and personnel costs related to increased headcount.

Net loss for the quarter ended September 30, 2018 was $94.7 million, compared to a net loss of $77.1 million for the quarter ended September 30, 2017.

Cash, cash equivalents and marketable securities as of September 30, 2018 were $878.4 million, compared to $567.8 million as of December 31, 2017. The increase in cash was driven by the net proceeds of $516.2 million from the January follow-on offering, $14.8 million of cost reimbursements and royalty payments under our collaboration agreements with Celgene, $12.0 million under our collaboration agreement with CStone and $29.2 million received from employee stock transactions. This was offset by expenditures to fund operations of $263.0 million during the nine months ended September 30, 2018.

The company expects that its cash, cash equivalents and marketable securities as of September 30, 2018, together with anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements under our collaboration and license agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2020.

CONFERENCE CALL INFORMATION

Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss third quarter 2018 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 5285068. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On November 1, 2018 Cerus Corporation (Nasdaq: CERS) reported financial results for the third quarter ended September 30, 2018 (Press release, Cerus, NOV 1, 2018, View Source [SID1234530505]).

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Third Quarter Highlights and Recent Events

Third quarter product revenue of $15.4 million, a 43% increase compared to the third quarter of 2017
Year-over-year worldwide disposable kit volumes increased 85% in the third quarter of 2018
2018 product revenue guidance revised upwards to a range of $58 million to $60 million, representing an increase of 33% to 38% compared to 2017 product revenue
Notified TÜV SÜD in October of the Company’s request to file its planned CE Mark submission in 60 days for INTERCEPT red blood cells
Received FDA Breakthrough Device Designation for pathogen-reduced cryoprecipitate
"The market adoption of the INTERCEPT Blood System continues to be strong with third quarter product revenue totaling $15.4 million," said William ‘Obi’ Greenman, Cerus’ president and chief executive officer. "Year-over-year product revenue growth was driven by sales of INTERCEPT platelet kits and was broad based with all major geographic regions delivering gains. Given our strong year-to-date results and increasing visibility into our commercial pipeline, we recently revised our 2018 product revenue guidance to a range of $58 million to $60 million."

"In addition to our strong third quarter results, we recently notified TÜV SÜD, our Notified Body, of our request to file our CE Mark submission in 60 days for INTERCEPT red blood cells. Our red cell team is working diligently in preparation for the planned submission and is on track to deliver on this important milestone," continued Greenman.

A Notified Body is an organization accredited by a member country of the European Union to determine if a product conforms to predetermined standards.

Revenue

Product revenue during the third quarter of 2018 was $15.4 million, compared to $10.8 million during the same period in 2017. The increase in third quarter product revenue was led by gains in platelet kit sales, which were partially offset by a year-over-year decline in illuminator sales. Third quarter 2017 illuminator sales benefited from the initial instrument shipments pursuant to the Company’s expanded supply agreement with EFS, the French National Blood Service. Year-to-date product revenue totaled $44.4 million, an increase of 62% compared to the same period of the prior year.

Government contract revenue from the Company’s Biomedical Advanced Research and Development Authority (BARDA) agreement was $3.9 million during the third quarter of 2018, compared to $2.3 million during the same period in 2017, as a result of increasing INTERCEPT red cell clinical and development activities. Year-to-date government contract revenue totaled $11.4 million compared to $5.4 million in the first nine months of 2017.

BARDA is part of the Office of the Assistant Secretary for Preparedness and Response within the U.S. Department of Health and Human Services. The development of the INTERCEPT red blood cell program has been funded in whole or in part with Federal funds from the Department of Health and Human Services; Office of the Assistant Secretary for Preparedness and Response; Biomedical Advanced Research and Development Authority, under Contract No. HHSO100201600009C.

Gross Margins

Gross margins on product revenue during the third quarter of 2018 were 47%, compared to 50% for the third quarter of 2017. The change in gross margin was primarily attributable to lower selling prices associated with high volume customers, and to a lesser extent, the unfavorable impact of foreign exchange rates. Gross margins through the first nine months of 2018 were 48% compared to 51% in the same period of the prior year.

Operating Expenses

Total operating expenses were $24.8 million for the quarter ended September 30, 2018, compared to $20.1 million for the quarter ended September 30, 2017. Year-to-date, operating expenses totaled $72.2 million compared to $66.0 million in the same period of the prior year.

Selling, general, and administrative (SG&A) expenses for the third quarter of 2018 totaled $14.0 million, compared to $12.2 million for the third quarter of 2017. The year-over-year increase was primarily tied to higher commercial activity in the U.S. Year-to-date SG&A expenses totaled $42.0 million, compared to $40.1 million during the first nine months of 2017.

Research and development (R&D) expenses for the third quarter of 2018 were $10.8 million, compared to $7.9 million for the third quarter of 2017. The increase in year-over-year R&D expenses was primarily due to additional activities and costs tied to the development of INTERCEPT red blood cells, including preparation for the planned CE Mark submission, trials and activities in pursuit of FDA approval of INTERCEPT red blood cells and activities aimed at expanded label claims for INTERCEPT platelets and plasma. Year-to-date R&D expenses through the third quarter of 2018 totaled $30.1 million, compared to $25.9 million during the first nine months of 2017.

Net Loss

Net loss for the third quarter of 2018 was $14.2 million, or $0.11 per diluted share, compared to a net loss of $13.4 million, or $0.12 per diluted share, for the third quarter of 2017. Year-to-date net loss was $41.4 million, or $0.32 per diluted share, compared to a net loss of $49.1 million, or $0.46 per diluted share, in the first nine months of 2017.

Cash, Cash Equivalents and Investments

At September 30, 2018, the Company had cash, cash equivalents and short-term investments of $119.0 million, compared to $60.7 million at December 31, 2017.

At September 30, 2018, the Company had approximately $29.9 million in outstanding debt under its loan agreement with Oxford Finance compared to $29.8 million at December 31, 2017.

QUARTERLY CONFERENCE CALL

The Company will host a conference call and webcast at 4:15 P.M. EDT this afternoon, during which management will discuss the Company’s financial results and provide a general business overview and outlook. To access the live webcast, please visit the Investor Relations page of the Cerus website at View Source Alternatively, you may access the live conference call by dialing (866) 235-9006 (U.S.) or (631) 291-4549 (international).

A replay will be available on the Company’s website, or by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and entering conference ID number 7095077. The replay will be available approximately three hours after the call through November 15, 2018.