On December 5, 2020 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, reported in an oral presentation the updated and expanded results from a Phase 1 clinical study of GDA-201, an investigational, natural killer (NK) cell-based cancer immunotherapy for the treatment of patients with non-Hodgkin lymphoma (NHL) and multiple myeloma (MM), at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, which is being held virtually December 5–8 (Press release, Gamida Cell, DEC 5, 2020, View Source [SID1234572243]).

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GDA-201 was well-tolerated and no dose-limiting toxicities were observed in 35 patients (19 with NHL and 16 with MM). The data show that therapy using GDA-201 with monoclonal antibodies demonstrated significant clinical activity in heavily pretreated patients with advanced NHL. Of the 19 patients with NHL, 13 complete responses and one partial response were observed, with an overall response rate of 74 percent and a complete response rate of 68 percent. The maximum tolerated dose was not achieved, as no dose limiting toxicities were observed in patients who received the maximum target dose (2 x 108 cells/kg).

"Data from an expanded group of patients in this Phase 1 clinical study for GDA-201 show that NK cell therapies continue to exhibit impressive therapeutic potential to treat relapsed and refractory patients with lymphomas, while maintaining a favorable safety profile," said Veronika Bachanova, M.D., Ph.D., Professor of Medicine in the Division of Hematology, Oncology and Transplantation at the University of Minnesota and principal investigator of the study. "Despite recent advancements in therapies for patients with hematologic malignancies, too many patients progress to develop refractory or resistant disease. I look forward to the continued clinical development of this novel investigational therapy."

NK cell immunotherapies are thought to offer tremendous potential for transforming the care of hematologic malignancies. With GDA-201, Gamida Cell is pioneering a novel approach that harnesses the power of its cell expansion technology to improve antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells.

"These additional results again show that GDA-201 has striking signs of efficacy and safety in patients with heavily pre-treated NHL," said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "With these results in hand, we plan to initiate a Phase 2 clinical study, with the goal of submitting an IND in 2021."

GDA-201 Phase 1 Clinical Data

The presentation, "Results of a Phase 1 Trial of GDA-201, Nicotinamide-Expanded Allogeneic Natural Killer (NK) Cells in Patients with Refractory Non-Hodgkin Lymphoma (NHL) and Multiple Myeloma," described results from the Phase 1 clinical study of GDA-201 in heavily pre-treated patients with advanced NHL and MM. Preliminary results from this study were presented at the 2019 ASH (Free ASH Whitepaper) Annual Meeting.

In the study, cell therapy using GDA-201 with monoclonal antibodies was shown to be safe; there were no dose-limiting toxicities, neurotoxic events, confirmed cytokine release syndrome, graft versus host disease or marrow aplasia. Overall survival and progression-free survival at one year in the NHL cohort suggest durable disease control, with a median follow-up of ten months (range 1–28 months). The most common adverse events were decreased neutrophil count, febrile neutropenia, anemia and low platelet counts.

In the NHL cohort, durable complete responses were observed in patients with both follicular and diffuse large B cell lymphoma, with an overall response rate of 74 percent. Future development of GDA-201 may include cryopreservation and the exploration of multiple treatment cycles in a multi-center Phase 2 trial in patients with NHL.

About GDA-201

Gamida Cell applied the capabilities of its NAM-based cell expansion technology to develop GDA-201, an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.1 For more information on the clinical study of GDA-201, please visit www.clinicaltrials.gov.

GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the U.S. Food and Drug Administration or any other health authority.

About the NAM Therapeutic Platform

Gamida Cell’s proprietary NAM-based cell expansion platform is designed to enhance the number and functionality of donor cells in culture, enabling the creation of potentially transformative therapies that move beyond what is possible with existing approaches. The NAM therapeutic platform leverages the unique properties of nicotinamide to enable the expansion of multiple cell types — including stem cells and natural killer (NK) cells — with appropriate growth factors to maintain the cells’ original phenotype and potency. This can enable the administration of a therapeutic dose of cells with the potential to improve patient outcomes.

On December 5, 2020 Rhizen Pharmaceuticals, a clinical-stage oncology-focussed biopharmaceutical company, reported presentation of the interim results of a Phase I/II combination study of Tenalisib with Romidepsin for relapsed/refractory T-Cell Lymphoma patients at the upcoming 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting and exposition, to be held virtually from December 5 – 8, 2020 (Press release, Rhizen Pharmaceuticals, DEC 5, 2020, View Source [SID1234572242]).

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The poster presentation at ASH (Free ASH Whitepaper), 2020 includes interim data from a multi-center, open label, Phase I/II study in patients with T cell lymphoma (NCT03770000) with the objective of determining the MTD/optimal dose of Tenalisib given in combination with Romidepsin and assessing the preliminary efficacy of the combination. No dose limiting toxicities were reported and Tenalisib 800 mg BID plus Romidepsin 14 mg/m2 was determined to be the maximum tolerated dose for the expansion cohorts. Overall, the Tenalisib and Romidepsin combination was well tolerated across all patients (N=33) with no unexpected adverse events or increased frequency of adverse events previously reported for the individual agents.

As of the cut-off date (5th Nov 2020) for data analysis, seven PTCL patients had completed their first efficacy assessment (C3D1), of which four patients showed a complete response, one demonstrated a partial response and two had stable disease. The duration of response ranged from 1.4+ to 8.16+ months. Of the eleven CTCL patients who had completed their first efficacy assessment (C3D1), one patient showed a complete response, three demonstrated a partial response and five had stable disease. The duration of response ranged from 1.20+ to 10.6+ months.

Swaroop Vakkalanka, the company’s President and CEO stated, "We are extremely encouraged by the interim data being presented today which reinforces Tenalisib’s stellar safety and impressive efficacy when studied in combination with Romidepsin. Both PTCL and CTCL are difficult to treat indications and patients quickly run out of options, so we are excited with the preliminary results. We expect this study to eventually support a pivotal trial of the combination and we plan to approach the FDA when the full study results are available.

The Tenalisib poster session details at ASH (Free ASH Whitepaper) 2020 are noted below:

Poster Title: A Multi-Center, Open Label, Phase I/II Study to Assess the Safety and Efficacy of Tenalisib Given in Combination with Romidepsin in Patients with Relapsed/Refractory T-Cell Lymphoma (Publication Number: 1155)
Session: 624. Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: Poster I
Date: Saturday, December 5, 2020; 7:00 AM – 3:30 PM (Pacific Time)
Presenter: Swaminathan P Iyer, MD, Professor, Department of Lymphoma/Myeloma, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX
About Tenalisib (RP 6530):

Tenalisib (RP6530) is a highly selective next generation orally active dual PI3K δ/γ inhibitor, which is in Phase 2 clinical development for hematological malignancies and solid tumors. Tenalisib has been granted US FDA Fast Track Designations for treatment of relapsed/refractory peripheral T-cell lymphoma and cutaneous T-cell lymphoma (R/R PTCL and R/R CTCL), in addition to Orphan-Drug Designations for treatment of peripheral and cutaneous T-cell lymphoma (PTCL and CTCL).

About T-Cell Lymphomas:

T-Cell Lymphomas (TCL) are a group of cancers that originate in T-cells and develop in lymphoid tissues such as the lymph nodes and spleen, or outside of lymphoid tissues (i.e., gastrointestinal tract, liver, nasal cavity, skin, and others). TCL constitute ~7-15% of all NHL cases and can be generally classified based on their presentation, as indolent or aggressive.

Peripheral T-Cell Lymphoma (PTCL) describes a heterogeneous group of lymphoproliferative disorders arising from mature T-Cells and accounts for ~10% of all NHL cases. PTCL is an aggressive disease that most commonly presents in patients over the age of 60 and usually has a worse prognosis than diffuse large B cell lymphoma.

Cutaneous T-Cell Lymphoma (CTCL) describes a group of typically indolent lymphomas that appear on, and are most often confined to, the skin and accounts for ~3% of all NHL cases and usually affects adults. CTCL subtypes include the more common and indolent Mycosis Fungoides (MF), which is largely confined to the skin, and the less common but more severe Sezary Syndrome (SS) that affects both the skin and blood and has poor prognosis.

On December 5, 2020 Imago BioSciences, Inc., ("Imago") a clinical-stage biotechnology company developing innovative treatments for myeloid diseases, reported interim data from 49 patients in its ongoing Phase 2b study evaluating bomedemstat (IMG-7289) for the treatment of myelofibrosis (Press release, Imago BioSciences, DEC 5, 2020, View Source [SID1234572241]). The data demonstrated sustained clinical activity and a favorable tolerability profile for the investigational treatment. The data were presented in an oral session by Kristen M. Pettit, M.D., assistant professor of medicine at the University of Michigan Rogel Cancer Center, during the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

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The interim results included an analysis of total symptom score (TSS) reduction at 12 weeks in 32 patients, with 78% of patients showing an improvement. In addition, 86% of patients evaluable (N=14) for spleen volume showed a reduction at 12 weeks. In this patient population in which 51% were classified as high molecular risk for evolution to AML, a serial analysis of mutant alleles revealed that 32% of patients experienced a decrease in the frequency of mutant alleles from baseline and 55% remained at stable frequencies. In patients followed for up to 570 days, no patient acquired new mutations in any of the 261 genes associated with myeloid malignancies and none to date have developed leukemia.

"As we continue to advance bomedemstat through Phase 2b clinical trials in myelofibrosis and essential thrombocythemia, we are encouraged by the interim results presented today," said Hugh Young Rienhoff, Jr. MD, CEO, Imago BioSciences. "There is a grave unmet need today among patients with myeloid diseases, and we look forward to realizing the promise of bomedemstat as a new therapeutic option with the potential to alter the natural history of these diseases."

Safety data were presented showing a total of 917 adverse events including 6 serious adverse events each occurring once deemed by Investigators as possibly related to IMG-7289. There have been no safety signals, dose-limiting toxicities, or deaths related to the drug.

About Bomedemstat (IMG-7289)

Bomedemstat is an orally available small molecule discovered and developed by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme shown to be vital in cancer stem/progenitor cells, particularly neoplastic bone marrow cells. In non-clinical studies, bomedemstat demonstrated robust in vivo anti-tumor efficacy across a range of myeloid malignancies as a single agent and in combination with other therapeutic agents. Bomedemstat is an investigational agent currently being evaluated in ongoing clinical trials (ClinicalTrials.gov Identifier: NCT03136185, NCT04262141, NCT04254978 and NCT04081220).

Bomedemstat has U.S. FDA Orphan Drug and Fast Track Designation for the treatment of myelofibrosis and essential thrombocythemia, Orphan Drug Designation for treatment of acute myeloid leukemia and PRIME designation by the European Medicines Agency for the treatment of MF.

Bomedemstat is being evaluated in three open-label Phase 2 clinical trials for the treatment of advanced myelofibrosis (MF), polycythemia vera (PV) and essential thrombocythemia (ET), bone marrow cancers that interfere with the production of blood cells. MF patients who are resistant to a Janus Kinase (JAK) inhibitor are eligible for the study of bomedemstat. ET patients who have failed one standard of care treatment are eligible for the bomedemstat ET study.

On December 5, 2020 Bristol Myers Squibb (NYSE: BMY) reported the presentation of research across its hematology portfolio at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, which will take place virtually from December 5 to 8, 2020 (Press release, Bristol-Myers Squibb, DEC 5, 2020, View Source [SID1234572240]). Data from nearly 100 company-sponsored studies will be featured, reinforcing the depth and diversity of the company’s development program and commitment to discovering potential new options to treat patients with blood cancers and other serious hematologic diseases.

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Key data being presented by Bristol Myers Squibb and its partners at the 62nd ASH (Free ASH Whitepaper) Annual Meeting and Exposition include:

Multiple analyses of CD19-targeted CAR T cell therapy liso-cel, highlighting studies of the treatment in additional hematologic malignancies, including results from the Phase 1 TRANSCEND NHL 001 study in patients with heavily pretreated, relapsed or refractory (R/R) mantle cell lymphoma and results from the Phase 1 TRANSCEND CLL 004 study in patients with R/R chronic lymphocytic leukemia. Moreover, a matching-adjusted indirect comparison of liso-cel vs. axicabtagene ciloleucel and tisagenlecleucel, and an analysis of outcomes in the outpatient setting will highlight new insights in treating R/R large B-cell lymphoma.
Reinforcing the company’s commitment to patients with multiple myeloma, presentations evaluating BCMA-targeted CAR T cell therapies in collaboration with bluebird bio, including: an analysis for ide-cel from the pivotal KarMMA study evaluating quality of life outcomes in R/R multiple myeloma. Additional safety, patient subgroup and correlative analyses from the KarMMa study highlighting the impact of prior therapies and features associated with CAR T expansion. In addition, updated results from the Phase 1 CRB-401 trial evaluating safety and responses in heavily pretreated patients with longer follow-up will be reported. Finally, updated results from the Phase 1 CRB-402 study of early-stage CAR T cell therapy bb21217 will also be presented.
The first efficacy and safety results from a triplet combination study including iberdomide, a cereblon E3 ligase modulator (CELMoD) agent, with daratumumab or bortezomib and dexamethasone in patients with heavily pretreated R/R multiple myeloma.
More than 40 abstracts highlighting Bristol Myers Squibb’s recent treatment advances for hard-to-treat myeloid diseases, with multiple quality of life analyses including data on reduced hospitalizations and associated estimated costs with Onureg (azacitidine tablets) in patients with acute myeloid leukemia in first remission from the Phase 3 QUAZAR AML-001 study. New health-related quality of life outcomes from the Phase 3 BELIEVE and MEDALIST studies of Reblozyl (luspatercept-aamt), in beta thalassemia and lower-risk myelodysplastic syndromes, will also be presented.
"Our purpose continues to be translating groundbreaking research across many hard-to-treat diseases and the nearly 100 studies being presented at this meeting illustrate our continued focus, with new modalities and different targets in our pipeline supporting the next waves of innovation in hematology," said Samit Hirawat, M.D., executive vice president, chief medical officer, global drug development, Bristol Myers Squibb. "This year’s ASH (Free ASH Whitepaper) represents an opportunity to highlight new data supporting our recently approved therapies, as well as other important advances across our pipeline. As we mark one year in growing a combined organization, we look forward to ASH (Free ASH Whitepaper) as an exchange that underscores our commitment to delivering potentially beneficial survival outcomes and quality of life improvements for patients."

Summary of Presentations:
Selected Bristol Myers Squibb studies at the 62nd ASH (Free ASH Whitepaper) Annual Meeting and Exposition include:

Acute Myeloid Leukemia

Escalated Dosing Schedules of CC-486 are Effective and Well Tolerated for Patients Experiencing First Acute Myeloid Leukemia (AML) Relapse: Results from the Phase III QUAZAR AML-001 Maintenance Trial
Presenter: H. Doehner
Presentation: #111
Date/Time: Saturday, December 5, 9:30 am EST
Health-related Quality of Life with CC-486 in Patients with Acute Myeloid Leukemia in First Remission Following Induction Chemotherapy: Results from the Phase III QUAZAR AML-001 Maintenance Trial
Presenter: G. Roboz
Presentation: #214
Date/Time: Saturday, December 5, 12:15 pm EST
CC-486 Reduces Hospitalization and Associated Estimated Costs in Patients with Acute Myeloid Leukemia in First Remission After Intensive Chemotherapy: Results of the QUAZAR AML-001 Maintenance Trial
Presenter: E. Olivia
Presentation: #621
Date/Time: Monday, December 7, 9:15 am EST
CC-486 Prolongs Survival for Patients with Acute Myeloid Leukemia in Remission after Intensive Chemotherapy Independent of Presence of Measurable Residual Disease at Study Entry: Results from the QUAZAR-AML-001 Maintenance Trial
Presenter: G. Roboz
Presentation: #692
Date/Time: Monday, December 7, 2:30 pm EST
CC-486 Improves Overall Survival and Relapse-free Survival for Patients with Acute Myeloid Leukemia in First Remission after Intensive Chemotherapy, Regardless of Amount of Consolidation Received: Results from the Phase III QUAZAR AML-001 Maintenance Trial
Presenter: A. Wei
Poster: #1036
Date: Saturday, December 5
Gastrointestinal Events and Management Strategies for Patients with Acute Myeloid Leukemia (AML) in First Remission Receiving CC-486 in the Randomized, Placebo-controlled, Phase III QUAZAR AML-001 Maintenance Trial ​
Presenter: F. Ravandi
Poster: #1917
Date: Sunday, December 6
Real-World Use of Enasidenib in Relapsed or Refractory (RR) Acute Myeloid Leukemia (AML) Is Associated with Reduced Risk of Disease Progression and Death
Presenter: A. Klink
Poster: #1912
Date: Sunday, December 6
Beta Thalassemia

Health-Related Quality of Life Outcomes for Patients with Transfusion-Dependent Beta-Thalassemia Treated With Luspatercept in the BELIEVE Trial
Presenter: M. Cappellini
Presentation: #364
Date/Time: Sunday, December 6, 9:30 am EST
Longitudinal Effect of Luspatercept Treatment on Iron Overload and Iron Chelation Therapy in Adult Patients with β-Thalassemia in the BELIEVE Trial
Presenter: O. Hermine
Poster: #1697
Date: Sunday, December 6
Sustained Reductions in Red Blood Cell (RBC) Transfusion Burden and Events in β-Thalassemia With Luspatercept: Longitudinal Results of the BELIEVE Trial
Presenter: A. Taher
Poster: #1695
Date: Sunday, December 6
Lymphoma and Chronic Lymphocytic Leukemia

Safety and Preliminary Efficacy in Patients with Relapsed/Refractory Mantle Cell Lymphoma Receiving Lisocabtagene Maraleucel in TRANSCEND NHL 001
Presenter: M. Palomba
Presentation: #118
Date/Time: Saturday, December 5, 9:45 am EST
Subgroup Analyses of Elderly Patients Aged ≥ 70 years in MAGNIFY: A Phase IIIb Interim Analysis of Induction R2 Followed by Maintenance in Relapsed/Refractory Indolent Non-Hodgkin Lymphoma
Presenter: F. Lansigan
Presentation: #340
Date/Time: Sunday, December 6, 10:30 am EST
Frontline Brentuximab Vedotin as Monotherapy or in Combination for Older Hodgkin Lymphoma Patients ​
Presenter: C. Yasenchak
Presentation: #471​
Date/Time: Sunday, December 6​, 2:15 pm​ EST
TRANSCEND CLL 004: Phase 1 Cohort of Lisocabtagene Maraleucel (liso-cel) in Combination With Ibrutinib for Patients with Relapsed/Refractory (R/R) Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL)
Presenter: W. Wierda
Presentation: #544
Date/Time: Monday, December 7, 7:30 am EST
Updated Follow-up of Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma Treated with Lisocabtagene Maraleucel in the Phase 1 Monotherapy Cohort of TRANSCEND CLL 004, Including High-Risk and Ibrutinib-Treated Patients
Presenter: T. Siddiqi
Presentation: #546
Date/Time: Monday, December 7, 8:00 am EST
Outcomes of Treatment With the Chimeric Antigen Receptor (CAR) T Cell Therapy Lisocabtagene Maraleucel (liso-cel) in the Nonuniversity Setting: Initial Results from the OUTREACH Study
Presenter: J. Godwin
Poster: #1196
Date: Saturday, December 5
Patients With Relapsed/Refractory Marginal Zone Lymphoma in the MAGNIFY Phase IIIb Interim Analysis of Induction R2 Followed by Maintenance
Presenter: M. Coleman
Poster: #1123
Date: Saturday, December 5
Costs of Postinfusion Monitoring by Site of Care for Patients with Relapsed/Refractory (R/R) Large B-Cell Lymphoma (LBCL) Who Received Third-Line or Later Treatment with Lisocabtagene Maraleucel (liso-cel) in the TRANSCEND NHL 001 and OUTREACH Trials
Presenter: M. Palomba
Poster: #2514
Date: Sunday, December 6
Matching-Adjusted Indirect Comparison (MAIC) of Lisocabtagene Maraleucel (Liso-cel) vs Axicabtagene Ciloleucel (Axi-cel) and Tisagenlecleucel in Relapsed/Refractory (R/R) Large B‐Cell Lymphoma (LBCL)
Presenter: D. Maloney
Poster: #2116
Date: Sunday, December 6
Nivolumab Combined with Brentuximab Vedotin (BV) for Relapsed/Refractory Mediastinal Gray Zone Lymphoma (R/R MGZL): Primary Efficacy and Safety Analysis of Phase 2 CheckMate 436 Study
Presenter: A. Santoro
Poster: #2045
Date: Sunday, December 6
Qualitative Analysis of the Treatment Experience and Well-Being of Patients with Relapsed/Refractory Large B-Cell Lymphoma Enrolled in 2 Trials of Lisocabtagene Maraleucel (liso-cel) during the Initial Stages of Therapy
Presenter: T. Siddiqi
Poster: #2565
Date: Sunday, December 6
Long-Term Results from a Phase 1b Study of Avadomide in Combination with Obinutuzumab in Patients with Relapsed and/or Refractory B-Cell Non-Hodgkin Lymphoma
Presenter: J. Michot
Poster: #2939
Date: Monday, December 7
Multiple Myeloma

Updated results from the Phase I CRB-402 study of anti-BCMA CAR-T cell therapy bb21217 in patients with relapsed and refractory multiple myeloma: correlation of expansion and duration of response with T cell phenotype
Presenter: M. Alsina
Presentation: #130
Date/Time: Saturday, December 5, 9:45 am EST
Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-Directed CAR T Cell Therapy, in Patients with Relapsed and Refractory Multiple Myeloma: Updated Results from Phase 1 CRB-401 Study
Presenter: Y. Lin
Presentation: #131
Date/Time: Saturday, December 5, 10:00 am EST
Secondary Quality of Life Domains in Patients with Relapsed and Refractory Multiple Myeloma treated with the BCMA Directed CAR T cell therapy Idecabtagene Vicleucel (ide-cel, bb2121): results from the KarMMa Clinical Trial
Presenter: N. Shah
Presentation: #437
Date/Time: Sunday, December 6, 12:15 pm EST
First results of Iberdomide (IBER; CC-220) in Combination With Dexamethasone (DEX) and Daratumumab (DARA) or Bortezomib (BORT) in Patients (Pts) with Relapsed/Refractory Multiple Myeloma (RRMM)
Presenter: N. Van de Donk
Presentation: #724
Date/Time: Monday, December 7, 1:30 pm EST
Characterization of Cytokine Release Syndrome in the KarMMa Study of Idecabtagene Vicleucel (ide-cel, bb2121) for Relapsed and Refractory Multiple Myeloma
Presenter: A. Kansagra
Poster: #1378
Date: Saturday, December 5
Efficacy and Safety of Idecabtagene Vicleucel (ide-cel, bb2121) in Elderly Patients With Relapsed and Refractory Multiple Myeloma: KarMMa Subgroup Analysis
Presenter: J. Berdeja
Poster: #1367
Date: Saturday, December 5
Molecular and Phenotypic Profiling of Drug Product and Post-Infusion Samples from CRB-402, an Ongoing: Phase I Clinical Study of bb21217 a BCMA Directed CAR T Cell Therapy
Presenter: O. Finnery
Poster: #1401
Date: Saturday, December 5
Association of Baseline and Postinfusion Biomarkers with Safety and Efficacy Endpoints in Patients Treated with Orvacabtagene Autoleucel (orva-cel; JCARH125) in the Phase 1/2 EVOLVE Study
Presenter: P. McCarthy
Poster #2350
Date: Sunday, December 6
Dose- and Schedule-Dependent Immunomodulatory Effects of the Novel CELMoD Agent CC-92480 in Patients with Relapsed/Refractory Multiple Myeloma
Presenter: L. Wong
Poster: #2295
Date: Sunday, December 6
Orvacabtagene Autoleucel (orva-cel; JCARH125): A Fully Human BCMA-Targeted Second-Generation CAR T Cell Product Characterized by a Predominant Central Memory Phenotype with High In Vitro and In Vivo Proliferative Potential and Sustained In Vivo Persistence
Presenter: L. Colonna
Poster: #2350
Date: Sunday, December 6
Pomalidomide, Dexamethasone, and Daratumumab after Lenalidomide Treatment in Relapsed Refractory Multiple Myeloma: Updated Results from an Open-Label, Multicenter, Phase 2 Trial
Presenter: N. Bahlis
Poster: #2314
Date: Sunday, December 6
Idecabtagene Vicleucel (ide-cel, bb2121) in Relapsed and Refractory Multiple Myeloma: Analyses of High-Risk Subgroups in the KarMMa Study
Presenter: N. Raje
Poster: #3234
Date: Monday, December 7
Myelodysplastic Syndromes

Health-Related Quality of Life Outcomes in Patients with Myelodysplastic Syndromes with Ring Sideroblasts Treated with Luspatercept in the MEDALIST Study
Presenter: E. Olivia
Poster: #1611
Date: Saturday, December 5
Efficacy and Safety of Luspatercept Treatment in Patients with Myelodysplastic Syndrome/ Myeloproliferative Neoplasm with Ring Sideroblasts and Thrombocytosis (MDS/MPN-RS-T): A Retrospective Analysis from the MEDALIST Study
Presenter: R. Komrokji
Poster: #3111
Date: Monday, December 7
Physicians’ Experience in Blood Supply Shortages and the Top Factors That Impact the Clinical, Economic, and Humanistic Outcomes of Myelodysplastic Syndrome (MDS) Patients in Five European Countries
Presenter: J.Tang
Poster: #3492
Date: Monday, December 7
Myelofibrosis

Long-Term Safety of Fedratinib in Patients with Intermediate- or High-Risk Myelofibrosis
Presenter: A. Pardanani
Poster: #3006
Date: Monday, December 7
Preclinical Presentations

FEIBA and NovoSeven Neutralize the Anticoagulant Effects of a Novel Small Molecule FXIa Inhibitor BMS-986177/JNJ-70033093 in Human Plasma and Whole Blood In Vitro
Presenter: M. Bunce
Poster: #1809
Date: Sunday, December 6
Bristol Myers Squibb: Creating a Better Future for People with Cancer
Bristol Myers Squibb is inspired by a single vision—transforming people’s lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine, and through innovative digital platforms, are turning data into insights that sharpen their focus. Deep scientific expertise, cutting-edge capabilities and discovery platforms enable the company to look at cancer from every angle. Cancer can have a relentless grasp on many parts of a patient’s life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. Because as a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.

On December 5, 2020 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported new data highlighting progress on its AUTO1 program, the company’s CAR T cell therapy being investigated in the ongoing ALLCAR Phase 1 study in relapsed / refractory adult B-Acute Lymphocytic Leukemia (ALL), during the American Society of Hematology (ASH) (Free ASH Whitepaper) All-Virtual Annual Meeting, held between December 5-8, 2020 (Press release, Autolus, DEC 5, 2020, View Source [SID1234572235]).

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As of the November 12, 2020 data cut-off date, 20 patients with r/r ALL had received AUTO1. AUTO1 was well tolerated, with no patients experiencing ≥ Grade 3 cytokine release syndrome (CRS). Three patients (15%), all of whom had high leukemia burden (>50% blasts), experienced Grade 3 neurotoxicity (NT) that resolved swiftly with steroids.

Of the 19 patients evaluable for efficacy, 16 (84%) patients achieved minimum residual disease (MRD)-negative complete response (CR) at one month. Most notably, the durability of remissions is highly encouraging. Across all treated patients, event free survival (EFS) at six and 12 months is 69% and 52% respectively. Median EFS and overall survival (OS) has not been reached at a median follow up of 16.9 months (range up to 30.5 months).

"The high level of sustained CRs observed with AUTO1 in adult ALL, achieved without subsequent stem cell transplant, point to a potentially transformational treatment for adult ALL," said Dr. Claire Roddie, Consultant Hematologist, UCL Cancer Institute and University College London Hospital. "Despite high disease burden and despite this being a heavily pre-treated patient population on study, AUTO1 remains well tolerated. It’s encouraging to also observe promising early activity and safety in indolent NHL."

"Adult ALL is a disease with high unmet need, whereby approximately 60% of patients relapse or are refractory to first line therapy," said Dr. Elias Jabbour, Professor of Leukemia at The University of Texas MD Anderson Cancer Center. "AUTO1 is a novel CD19 CAR T candidate with an impressive clinical profile. This profile has the potential to change standard of care as a curative therapy for r/r ALL."

Dr. Christian Itin, chairman and chief executive officer of Autolus, added "We are excited about the long-term remissions observed without a need for an additional stem cell transplant. Remarkably, this outstanding result was achieved with a well-tolerated safety profile in this fragile adult ALL population. We believe the unique characteristics of AUTO1, seen in the ALLCAR study, point to the potential for AUTO1 as a standalone and transformational therapy in r/r ALL. Our Phase 1b/2 pivotal study is under way, however, with the escalating COVID-19 pandemic, enrolment projections have had to be adjusted. We now expect to enroll patients throughout 2021 with a full data set in 2022."

In addition to adult ALL, the ALLCAR study was extended to patients with indolent B cell Non-Hodgkin Lymphoma (NHL) (Cohort 1), high grade B-NHL (Cohort 2) and chronic lymphocytic leukemia (CLL) (Cohort 3). As of the data cut-off date of November 12, 2020, four patients in Cohort 1 had been infused with AUTO1. AUTO1 was well tolerated, with no patients experiencing ≥ Grade 2 CRS and no patients experiencing NT of any grade. All four patients achieved a Complete Metabolic Response (CMR).

Investor call on Monday December 7, 2020
Management will host a conference call and webcast on Monday, December 7, 2020 at 4:00 pm ET/9:00 pm GMT to discuss the ASH (Free ASH Whitepaper) data. To listen to the webcast and view the accompanying slide presentation, please go to: View Source

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID 9188389. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID 9188389.

About Autolus Therapeutics plc
Autolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer. Using a broad suite of proprietary and modular T cell programming technologies, the company is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognize cancer cells, break down their defense mechanisms and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies and solid tumors. For more information please visit www.autolus.com.

About AUTO1
AUTO1 is a CD19 CAR T cell investigational therapy designed to overcome the limitations in safety – while maintaining similar levels of efficacy – compared to current CD19 CAR T cell therapies. Designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, AUTO1 may reduce toxicity and be less prone to T cell exhaustion, which could enhance persistence and improve the ability of the programmed T cells to engage in serial killing of target cancer cells. AUTO1 is currently being evaluated in two Phase 1 studies, one in pediatric ALL and one in adult ALL. The company has also now progressed the program to a potential pivotal study, AUTO1-AL1.

About AUTO1-AL1 pivotal study
The AUTO1-AL1 study will enroll patients with relapsed / refractory ALL. The study will have a short Phase1b component prior to proceeding to a single arm Phase 2 study. The primary end point is overall response rate and the key secondary end points include duration of response, MRD negative CR rate and safety. The study will enroll approximately 100 patients across 30 of the leading academic and non-academic centers in the US, UK and Europe.