On June 8, 2021 Antengene’s Partner, Karyopharm Therapeutics Inc. (Nasdaq: KPTI), reported that updated data of eltanexor for the treatment of patients with hypomethylating agent (HMA) refractory myelodysplastic syndrome (MDS) at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Karyopharm, JUN 8, 2021, View Source [SID1234583727]).

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This Phase I/II study evaluated single-agent eltanexor in patients with higher-risk MDS, ie, high-risk or intermediate-2 MDS by the International Prognostic Scoring System (IPSS) and 5%-19% myeloblasts. The patients enrolled in the trial were evaluated with eltanexor at the two daily doses of 10 mg (n=5) or 20 mg (n=10) for 5 days per week of a 28-day cycle.

Out of the 20 patients enrolled, 15 patients were evaluable for efficacy and constitute the population studied in this analysis. Of the 15 patients evaluable for efficacy, 7 (47%) had marrow complete response (mCR) and 5 (33%) had stable disease (SD) for a total disease control rate (DCR, mCR+SD) of 80% (60% for all patients’ analysis). In the 10 mg cohort (n=5), all patients derived clinical benefit with 3 patients (60%) reaching mCR and 2 patients (40%) reaching SD. In the 20 mg cohort (n=10), 4 patients (40%) had mCR and 3 (30%) had SD. Four patients had hematologic improvement (HI) and became transfusion-independent for at least 8 weeks including 2 patients with tri-lineage HI. The overall survival (OS) for patients who reached mCR (n=7) was significantly longer than that for patients who did not reach mCR (n=8): median 11.86 vs 8.67 months (hazard ratio [HR]=0.27, p=0.05), and significantly longer than the OS for patients with progression disease (PD, n=3, mOS=3.15 mo, HR=0.23, p=0.04). Patients with disease control (n=12) had numerically longer median overall survival (mOS) than patients with PD (9.86 vs 3.15 mo, HR=0.38, p=0.09). Patients with HI had a mOS of 10.58 months.

Patients with MDS refractory to HMAs have limited therapeutic options and a dismal prognosis with a median overall survival (mOS) of 4-6 months. Eltanexor is a next-generation, oral XPO1 inhibitor that showed anti-tumor activity and lower central nervous system penetration compared to selinexor, the first-in-class XPO1 inhibitor, in nonclinical models. It was hypothesized that eltanexor could be dosed more frequently than selinexor with a lower incidence of centrally mediated nausea.

Antengene has entered into a strategic collaboration with Karyopharm, through which it obtained the rights to develop and commercialize four drug candidates including eltanexor in 17 Asia Pacific markets. Antengene is currently conducting clinical trials of eltanexor in patients with MDS or advanced solid tumors in China. Of these, the Phase I/II trial of eltanexor for the treatment of MDS (the HATCH trial) has already dosed its first patient.

About Eltanexor (ATG-016)

Eltanexor is a next-generation selective inhibitor of nuclear export (SINE) compound. In preclinical models, compared to the first-generation SINE compound, eltanexor demonstrated lower blood-brain barrier penetration and broader therapeutic window which allows more frequent dosing and a longer period of exposure at higher levels with better tolerability. Therefore, eltanexor may be used to target a broader range of indications.

On June 8, 2021 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") reported the presentation of new data in acute myeloid leukemia (AML) at the European Hematology Association (EHA) (Free EHA Whitepaper) virtual congress, taking place June 9-17 (Press release, Astellas, JUN 8, 2021, View Source [SID1234583695]).

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Eight Astellas-sponsored abstracts focused on patients with AML are being presented, comprising two oral presentations, four posters and two online-only abstract publications.

"We’re pleased to present new investigational research at EHA (Free EHA Whitepaper) 2021 that examines how patients and healthcare providers value different treatment approaches in AML," said Erhan Berrak, M.D., Vice President of Medical Affairs, Oncology, Astellas. "For example, one oral presentation sheds light on patients’ preferences for treatment, which may help to inform decisions of healthcare professionals when considering a plan for treatment after hematopoietic stem cell transplantation."

"Clinical trial results to be presented reflect our deep commitment to AML research, where we are investigating gilteritinib as monotherapy or in combination with other treatments, and across the range of patients with FLT3 mutation-positive AML, including patients whose AML is newly diagnosed or relapsed or refractory," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Oncology Therapeutic Area Head, Astellas.

Oral Presentations
Oral presentations are available online from Friday, June 11 at 9 a.m. CEST, when all prerecorded presentations will be published on the virtual congress platform.

Title: Patient and Physician Preferences for Post–Hematopoietic Stem Cell Transplantation Maintenance Treatment of Acute Myeloid Leukemia (Abstract S313)

Presenting author: Manasee V. Shah, Astellas Pharma Inc., Northbrook, Ill., USA
Title: Efficacy and Safety of Venetoclax in Combination with Gilteritinib for Relapsed/Refractory FLT3-Mutated Acute Myeloid Leukemia: Updated Analyses of a Phase 1b Study (Supported by AbbVie, Astellas and Genentech) (Abstract S135)

Presenting author: Jessica K. Altman, Robert H. Lurie Comprehensive Cancer Center, Northwestern University, Chicago, Ill., USA
E-Poster Presentations
E-poster presentations are available from Friday, June 11 at 9 a.m. CEST, when the e-posters are published on the virtual congress platform.

Title: Follow-up of Patients with FLT3-Mutated Relapsed or Refractory Acute Myeloid Leukemia in the Phase 3 ADMIRAL Trial (Abstract EP438)

Presenting author: Mark J. Levis, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, Md., USA
Title: Clinical Outcomes in Patients with Relapsed/Refractory Acute Myeloid Leukemia Treated with Gilteritinib Who Received Prior Midostaurin or Sorafenib (Abstract EP448)

Presenting author: Alexander E. Perl, Abramson Comprehensive Cancer Center, University of Pennsylvania, Philadelphia, Pa., USA
Title: Outcomes in Gilteritinib-Treated FLT3-Mutated R/R AML Patients Who Underwent Transplantation (Abstract EP441)

Presenting author: Alexander E. Perl, Abramson Comprehensive Cancer Center, University of Pennsylvania, Philadelphia, Pa., USA
Title: A Phase 1 Study of Gilteritinib in Combination with Induction and Consolidation Chemotherapy in Patients with Newly Diagnosed AML: Final Results Update (Abstract EP437)

Presenting author: Keith W. Pratz, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, Md., USA
Online-only abstract publications
Online-only abstracts are available via the virtual congress platform.

Title: Retrospective Assessment of Treatment Patterns and Resource Utilization for Patients Newly Diagnosed with Acute Myeloid Leukemia in Canada, UK, France, Germany, Italy, and Spain (Abstract PB1390)

Title: Frequency of FLT3-ITD and FLT3-TKD Mutations in Patients with Acute Myeloid Leukemia: A Systematic Literature Review and Meta-Analysis (Abstract PB1405)

The EHA (Free EHA Whitepaper) 2021 virtual congress abstracts are available in the EHA (Free EHA Whitepaper) Library.

About Acute Myeloid Leukemia (AML)
Acute myeloid leukemia (AML) is a type of cancer that affects the bone marrow and blood. It is deemed "acute," meaning that this type of leukemia can progress quickly.1 In the European Union, the incidence rate of AML is 3.7 per 100,000 per year, resulting in an estimated 16,800 individuals diagnosed.2

About Gilteritinib
Gilteritinib was discovered through a research collaboration with Kotobuki Pharmaceutical Co., Ltd., and Astellas has exclusive global rights to develop, manufacture and commercialize gilteritinib. Gilteritinib is available as XOSPATA in the U.S., Japan, China and selected European countries, among others, for the treatment of adult patients who have relapsed or refractory FLT3mut+ AML.3,4,5 Gilteritinib is an FMS-like tyrosine kinase 3 (FLT3) inhibitor with demonstrated activity against FLT3-ITD, a common driver mutation that presents with a high burden and poor prognosis, and FLT3-TKD mutations.6

European Union Important Safety Information
For important Safety Information for gilteritinib please see the full Summary of Product Characteristics at: View Source

United States Important Safety Information
For important Safety Information for gilteritinib please see Important Safety Information at: View Source

On June 8, 2021 Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology Company pioneering a differentiated approach to treating neurodegenerative disease and cancer through the inhibition of SEMA4D, reported that the first clinical sites have been activated to screen and enroll patients in its Phase 1/2 study evaluating pepinemab as a single agent in Alzheimer’s disease (AD) and in its phase 2 study in of pepinemab combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) as front-line treatment for advanced, recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) (Press release, Vaccinex, JUN 8, 2021, View Source [SID1234583711]). The Company plans to activate at least 13 U.S. sites for the Alzheimer study and 18 U.S. sites for HNSCC.

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The Alzheimer’s proof-of-concept study is expected to enroll at least 40 patients with key efficacy endpoints that include measures of cognition and brain imaging. This study has received funding support from the Alzheimer’s Drug Discovery Foundation and the Alzheimer’s Association. The HNSCC study will enroll up to 65 patients allocated to different levels of combined positive score (The HNSCC study is expected to enroll up to 65 patients allocated to different levels of combined positive score (CPS) of PD-L1 expression. CPS is a biomarker associated with benefit in response to immunotherapy. Efficacy endpoints will focus on objective response rate (ORR) per RECIST 1.1 criteria, as well as progression-free survival (PFS), overall survival (OS) and duration of response (DOR).

Dr. Maurice Zauderer, chief executive officer of Vaccinex, stated, "With this Alzheimer’s study, we are building on prior data from our SIGNAL phase 2 study in Huntington’s disease that we believe indicated cognitive benefit to patients at an early stage of this devastating neurodegenerative disease. It was particularly encouraging that this was accompanied by evidence of increased brain metabolic activity which has been shown in several independent studies to correlate with cognitive change in AD as well. AD patients are in urgent need of new therapies to effectively slow or halt disease progression, and we are looking forward to results from this important study.

"In addition, we recently published results of a prior phase 2 study of pepinemab in combination with a checkpoint inhibitor, EMD Serono’s Bavencio, that we believe indicated treatment benefit to patients with non-small cell lung cancer. We are pleased to have now initiated this new trial in HNSCC in collaboration with Merck, a global immunotherapy leader. We and others have shown that SEMA4D is highly expressed in head and neck cancer and triggers increased levels of myeloid-derived suppressor cells that inhibit immune responses to tumor, providing a compelling scientific rationale for this study," Dr. Zauderer concluded.

Multiple prior preclinical studies suggested that inhibition of SEMA4D has unique mechanisms of action that reduce activation of inflammatory glial cells in brain but increases immune infiltration and alters the balance of cytotoxic and immunosuppressive cells in a tumor microenvironment. The Company is pleased and excited to have the opportunity to develop this potentially promising therapy in multiple important indications.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

Bavencio is a registered trademark of EMD Serono, Inc., the U.S. biopharmaceutical business of Merck KGaA, Darmstadt, Germany.

On June 8, 2021 Thermo Fisher Scientific Inc. (NYSE: TMO), the world leader in serving science, reported that Marc N. Casper, chairman, president and chief executive officer, will present virtually at the Goldman Sachs 42nd Annual Global Healthcare Conference on Thursday, June 10, 2021 at 9:40 a.m. (EDT) (Press release, Thermo Fisher Scientific, JUN 8, 2021, View Source [SID1234583728]).

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You can access the webcast of the presentation via the Investors section of our website, www.thermofisher.com.

On June 8, 2021 Bioneer reported that The presentation if part of the Focus Sessions of the ISSCR Annual Meeting, where Dr. Benjamin Schmid and Dr. Mikkel Rasmussen from Bioneer will be presenting (Press release, Bioneer, JUN 8, 2021, View Source [SID1234583696]).

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Focus Sessions provide in-depth coverage of specific topics of interest and are presented by interested academic and industry groups. These educational opportunities in science, society, and education are organized by members and open to all meeting attendees. Sessions are held live, with Q&A and chat, and will be available as on-demand programming for 30 days after the meeting.

The Focus Session on developments to simplify and accelerate iPSC research is organized by The European Bank for induced Pluripotent Stem Cells (EBiSC).

The European Bank for iPSCs (EBiSC) is a centralized repository, currently in a second project phase including both non-profit and commercial iPSC researchers (EBiSC2), working to make iPSC tools available and developing protocols which improve and simplify their use. This focus session will share how EBiSC2 partners are adapting and consolidating iPSC expansion, differentiation and cryopreservation approaches to help ease transition into high volume applications whilst also ensuring accessibility for non-expert users. We will discuss how the inclusion of iPSC tool lines in these protocol developments enables rapid generation of functionally mature derived cell types and how the associated iPSC datasets can be broadly shared in an ethically compliant manner. Lastly, common stumbling blocks will be discussed to raise awareness across the community.

See below the full program of the session:

The European Bank for iPSCs Program

Julia Neubauer, PhD, Fraunhofer-IBMT, Germany
Alfredo Cabrera-Socorro, PhD, Janssen Pharmaceutica NV, Belgium
Welcome and Overview

Julia Neubauer, PhD, Fraunhofer-IBMT, Germany
Approaches Towards Expansion, Differentiation and Banking Of iPSCs At High Volume

Mikkel Rasmussen, PhD, Bioneer, Denmark
Emilie Lemesre, PhD, Servier, France
iPSC-Derived Hepatocytes in Drug Screening and Toxicology

Alfredo Cabrera-Socorro, PhD, Janssen Pharmaceutica NV, Belgium
Development of A Fully Human Neuronal and Astrocyte Co-Culture Assay Amenable For Electrophysiological Studies In Functionally Mature Neurons

Benjamin Schmid, PhD, Bioneer, Denmark
Gene-Editing in iPSCs – Unexpected Pitfalls: On-Target Effects

Andreas Kurtz, PhD, Fraunhofer-IBMT, Germany
Collection, Standardisation and Sharing Of iPSC Associated Datasets Using Open Tools

Eugenia Jones, PhD, Fujifilm Cellular Dynamics, USA
Common Non-Scientific Challenges in The Generation, Use and Sharing Of iPSC Lines.

Panel Discussion: Upcoming Challenges In iPSC Research from An Academic and Industry Perspective.