On November 1, 2018 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported that new interim results from the Company’s ongoing Phase 2a study of cerdulatinib in patients with relapsed/refractory peripheral T-cell lymphoma (PTCL) and cutaneous T-cell lymphoma (CTCL), will be presented during an oral session at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, December 1-4, 2018 in San Diego, California (Press release, Portola Pharmaceuticals, NOV 1, 2018, View Source;p=RssLanding&cat=news&id=2374918 [SID1234530526]).

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The Company also will present new outcomes-based research on the burden of hospital readmissions for venous thromboembolism among patients with cancer during an oral session on Sunday, December 2, and two investigator-initiated animal studies highlighting the anticoagulant reversal agent Andexxa [coagulation Factor Xa (recombinant), inactivated-zhzo] will be presented in poster sessions.

"As shown at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and European Hematology Association (EHA) (Free EHA Whitepaper) meetings earlier this year, cerdulatinib has shown encouraging results across a range of B- and T-cell malignancies and we look forward to presenting new data specifically focused on patients with PTCL and CTCL," said John Curnutte, M.D., Ph.D., head of research and development for Portola. "These data also will serve as a basis for further discussions with regulatory agencies regarding the potential regulatory path forward for cerdulatinib in certain tumor subtypes."

Cerdulatinib is an investigational oral, dual Syk/JAK kinase inhibitor that uniquely inhibits two key cell signaling pathways that promote cancer cell growth in certain hematologic malignancies. It is being developed for the treatment of resistant or relapsed hematologic cancer.

Oral Presentations

1001. The Novel Syk/JAK Inhibitor Cerdulatinib Demonstrates Good Tolerability and Clinical Response in a Phase 2a Study in Relapsed/Refractory Peripheral T-Cell Lymphoma and Cutaneous T-Cell Lymphoma
Abstract: View Source
Session: 624 (Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: T Cell Lymphoma: Chemotherapy and Targeted Approaches)
Presenter: Steven M. Horwitz, M.D., Memorial Sloan-Kettering Cancer Center
Date: Monday, December 3, 2018 at 7:15 p.m. PST; Room 6F

365. Burden of Hospital Readmissions for Venous Thromboembolism Among Patients with Cancer
Abstract: View Source
Session: 901 (Health Services Research—Non-Malignant Conditions: Thrombosis and Anticoagulation
Hematology Disease Topics & Pathways)
Presenter: Alpesh Amin, M.D., M.B.A., UC Irvine
Date: Sunday, December 2, 2018 at 10:30 a.m. PST; Room 8
Poster Presentations

2456. Reversal of Apixaban Anticoagulation with Reduced Doses of Andexanet Alfa in a Porcine Polytrauma Model
Abstract: View Source
Session: 321 (Blood Coagulation and Fibrinolytic Factors: Poster II Hematology Disease Topics & Pathways)
Presenter: Oliver Grottke, M.D., Ph.D., M.P.H., University Hospital RWTH Aachen, Germany
Date: Sunday, December 2, 2018 from 6:00 – 8:00 p.m. PST; Hall GH

3778. Comparison of Second and First Generation of Andexanet Alfa in a Porcine Polytrauma Model with Apixaban Anticoagulation
Abstract: View Source
Session: 321 (Blood Coagulation and Fibrinolytic Factors: Poster II Hematology Disease Topics & Pathways)
Presenter: Oliver Grottke, M.D., Ph.D., M.P.H., University Hospital RWTH Aachen, Germany
Date: Monday, December 3, 2018 from 6:00 – 8:00 p.m. PST; Hall GH

On November 1, 2018 Celyad (Euronext Brussels and Paris, and Nasdaq: CYAD), a clinical-stage biopharmaceutical company focused on the development of specialized CAR-T cell-based therapies, reported that two abstracts detailing updated clinical results from the Phase 1 THINK dose-escalation trial and anticipated clinical trials for the CYAD-01 program will be presented at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego, December 1-4, 2018 (Press release, Celyad, NOV 1, 2018, View Source [SID1234530542]). Company management will also review the results of the THINK trial and provide an update on Celyad’s clinical development program for CYAD-01 at an Analyst/Investor event, which will also be available via webcast on December 3, 2018.

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"We are encouraged by the preliminary THINK study data evaluating CYAD-01, without preconditioning chemotherapy in patients with relapsed or refractory acute myeloid leukemia," said Dr. Christian Homsy, CEO of Celyad. "The data supplement a growing body of evidence that CYAD-01 shows encouraging clinical activity and is well-tolerated and suggests its potential for the treatment of acute myeloid leukemia, a challenging disease with limited therapeutic options. In addition to this important milestone, we continue to investigate CYAD-01 in alternative protocols to further optimize its clinical benefit."

Updated data from the THINK trial of CYAD-01 in patients with r/r AML will be presented by Principal Investigator David A. Sallman, M.D., of the Moffitt Cancer Center, on December 3, 2018. The presentation will include new information on safety, activity and correlative science data of the complete dose-escalation segment of the trial.

Top-line data from the abstract as of a data cut-off date of July 31, 2018, included:

Of the seven response-evaluable r/r AML patients enrolled in the trial who received the per-protocol dose of CYAD-01, the best overall response rate was 42% (three patients). Two additional patients experienced important clinical benefit with hematologic improvement and bone marrow blasts decrease, leading to clinical activity of 71% (five patients).
One patient experienced a complete remission with partial hematologic recovery (CRh) and two patients experienced a complete remission with incomplete marrow recovery (CRi). One CRh and one CRi occurred at dose level 1 (DL1) with an additional CRi at dose level 3. All three responders achieved a response by day 29 (i.e., prior to the third administration of CYAD-01).
The patient with CRh from DL1 was bridged to allogeneic hematopoietic stem cell transplantation (allo-HSCT) on day +97 post treatment with CYAD-01. This patient remains in durable complete molecular remission (CRMRD-) for more than one year (ongoing). A detailed case report of this patient was published in Haematologica in April 2018.
Of the two additional r/r AML patients who experienced a clinical benefit, one patient had a decrease in blast counts from 24% to 10%, while a second patient had a decrease from 9.8% to 5.5%. Disease stabilization in these patients were observed for three months and over four months (ongoing), respectively. Both patients were treated in dose level 2 of the study.
Overall, 12 patients with hematological malignancies (AML, myelodysplastic syndrome and multiple myeloma) treated with CYAD-01 in the cohort had reached the safety follow-up. The most common treatment-related adverse events (AEs) included pyrexia, cytokine release syndrome (CRS), hypoxia, lymphopenia, fatigue and nausea. CRS occurred in five patients (three grade 1/2 AEs and two grade 3 AEs), with rapid resolution following the appropriate treatment, including tocilizumab. Overall, five patients experienced grade 3/4 treatment-related AEs. No neurotoxicity AEs were observed in patients treated with CYAD-01.

CYAD-01 and THINK Trial Design

CYAD-01 is an investigational CAR-T therapy in which a patient’s T cells are engineered to express the chimeric antigen receptor NKG2D, a receptor expressed on natural killer (NK) cells that binds to eight stress-induced ligands expressed on tumor cells.

The THINK trial (NCT03018405) is an open-label, dose-escalation Phase 1 trial assessing the safety and clinical activity of multiple CYAD-01 administrations without prior preconditioning in two parallel cohorts: i) patients with hematological malignancies, including r/r AML, and ii) patients with metastatic solid tumors. The dose escalation segment of the study evaluates three dose levels (300 million, 1 billion and 3 billion cells per injection) of one cycle of three CYAD-01 administrations with two-week intervals.

ASH Analyst/Investor Event and Webcast Information

Celyad will host an Analyst/Investor event on Monday, December 3, 2018, beginning at 8:30 p.m. PT to review data presented at ASH (Free ASH Whitepaper). The event will be webcast live and can be accessed under Events & Webcasts in the Investors section of the Company’s website.

A complete list of Celyad and collaborator presentations to be made at ASH (Free ASH Whitepaper) appears below:

Oral Presentation

Remissions in Relapse/Refractory Acute Myeloid Leukemia Patients Following Treatment with NKG2D CAR-T Therapy Without a Prior Preconditioning Chemotherapy (Abstract #111326 – Publication Number 902)

Presenter: David A. Sallman, M.D., Moffitt Cancer Center

Date: Monday, December 3, 2018, 4:45 p.m. Pacific Time

Location: Manchester Grand Hyatt San Diego, Seaport Ballroom F

Poster Presentation

Phase 1 Studies Assessing the Safety and Clinical Activity of Multiple Doses of a NKG2D-based CAR-T Therapy, CYAD-01, in Acute Myeloid Leukemia (Abstract #114747 – Publication Number 1398)

Presenter: Jason B Brayer, MD, Moffitt Cancer Center

Date: Saturday, December 1, 2018, 6:15 PM – 8:15 PM

Location: San Diego Convention Center, Hall GH

On November 1, 2018 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, reported it will report its third quarter 2018 financial results and business progress on Thursday, November 8, 2018, after the close of the U.S. financial markets (Press release, Tocagen, NOV 1, 2018, View Source;p=RssLanding&cat=news&id=2374897 [SID1234530570]).

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On November 1, 2018 4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 antagonist drugs to improve immune cell trafficking to treat rare disease and cancer, reported that an abstract highlighting X4P-001-RD, the company’s CXCR4 antagonist, has been selected for poster presentation at the 60th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) meeting, taking place Dec. 1-4 in San Diego (Press release, X4 Pharmaceuticals, NOV 1, 2018, View Source [SID1234530651]). The presentation will describe Phase 2 results used to determine the Phase 3 dose in the ongoing Phase 2/3 study of X4P-001-RD in patients with WHIM syndrome, a rare genetic, primary immunodeficiency disease.

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At ASH (Free ASH Whitepaper): X4 Pharmaceuticals to present clinical data used to determine Phase 3 Dose from Phase 2/3 Study of X4P-001-RD in WHIM Syndrome.

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Details of the presentation on X4P-001-RD in WHIM syndrome are as follows:

Title: Determination of Phase 3 Dose for X4P-001 in Patients with WHIM Syndrome

Date & Time: Saturday, December 1, 6:15 PM – 8:15 PM

Location: San Diego Convention Center, Hall GH

Publication #: 1102

Poster Session: 201. Granulocytes, Monocytes, and Macrophages: Poster I

About WHIM Syndrome
WHIM syndrome is a primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene resulting in susceptibility to certain types of infections. WHIM is an abbreviation for the characteristic clinical symptoms of the syndrome: Warts, Hypogammaglobulinemia, Infections, and Myelokathexis. Within the overall category of primary immunodeficiencies, there are between 15,000 and 100,000 patients in the U.S. that are classified with primary immunodeficiency disease of unknown origin – of which WHIM is one.1,2,3 WHIM syndrome is a rare disorder and the precise prevalence or incidence of patients that have the genetic mutation responsible for WHIM syndrome is unknown. Individuals with WHIM syndrome are more susceptible to potentially life-threatening bacterial infections4. Additionally, WHIM syndrome is associated with significant morbidity beginning in early childhood and continuing throughout life. Current therapy is limited to treatment of acute infections with antibiotics or prevention through the use of intravenous immunoglobulin or G-CSF. There is no approved therapy for the treatment of WHIM syndrome.

On November 1, 2018 ImmunSYS, Inc. reported that Chairman & CEO Eamonn Hobbs presented on October 31st at the 2018 NYC Oncology Investor Conference, held at Rockefeller Center in Manhattan (Press release, ImmunSYS, NOV 1,2018, View Source [SID1234577208]). ImmunSYS was one of 31 companies accepted to present to investors attending the conference.

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The NYC Oncology Investor Conference 2018 hosted by OneMed Forum, and sponsored by the National Foundation for Cancer Research, the International Cancer Impact Fund, Klosters Innovation Partners, Venable LLP, Torreya Partners, and Marcum is the leading conference for early-stage private and public cancer investing.

Meeting attendees included leading life science and oncology venture capitalists, family offices, lawyers, pharma executives, startup public and private cancer companies, and cancer foundations. The conference provides a forum for discussion of trends, opportunities, and risks in oncology investing, corporate presentations by a select group of public and private oncology companies, and updates on cutting edge science.