On November 3, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematological and solid cancers and other serious diseases, reported an oral presentation of a real-world analysis comparing the effectiveness of omidubicel to other allo-HCT donor sources from the Center for International Blood and Marrow Transplant Research (CIBMTR) database (Press release, Gamida Cell, NOV 3, 2022, View Source [SID1234623035]). These data are being presented at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which is being held in New Orleans from December 10-13, 2022.

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"These data reinforce the clinical relevance of the rapid time to neutrophil engraftment observed in patients transplanted with omidubicel, and support the potential use of omidubicel in patients with hematologic malignancies requiring transplant. We are diligently preparing to bring this important therapy to patients upon potential FDA approval."

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"Data from our phase 3 study compared the safety and efficacy of omidubicel to standard cord blood. We are delighted to have the opportunity, in a podium presentation at ASH (Free ASH Whitepaper), to share the work we have done with CIBMTR, exploring their expansive real-world database and performing the first comparative efficacy analyses between omidubicel and other donor sources for patients undergoing allogeneic stem cell transplant," said Ronit Simantov, M.D., Chief Medical and Scientific Officer of Gamida Cell. "These data reinforce the clinical relevance of the rapid time to neutrophil engraftment observed in patients transplanted with omidubicel, and support the potential use of omidubicel in patients with hematologic malignancies requiring transplant. We are diligently preparing to bring this important therapy to patients upon potential FDA approval."

Details about the ASH (Free ASH Whitepaper) presentation are as follows:

Title: Clinical Outcomes Following Allogeneic Hematopoietic Cell Transplantation with Omidubicel or Other Donor Sources in Patients with Hematologic Malignancies: Comparison of Clinical Trial Results to Center for International Blood and Marrow Transplant Research Database Controls
Session Title: 732. Allogeneic Transplantation: Disease Response and Comparative Treatment Studies: Clinical Outcome: Real World Studies Based on Database Analyses
Lead Author: Smitha Sivaraman, PhD.
Time: Saturday, December 10, 2022, 2:00 p.m. – 3:30 p.m. EST (session time) and 2:30 p.m. EST (presentation time)
Location: Ernest N. Morial Convention Center, 391 – 392

A prospective cohort analysis study to compare the effectiveness of omidubicel versus other allo-HCT donor sources (MUD, MMUD and haploidentical) used in clinical practice is being presented. The study compared data from the omidubicel (n=52) and control arms (n=56) of the phase 3 study with a cohort of similar patients derived from the CIBMTR database (n = 807) who had undergone transplant with matched unrelated donors, mismatched unrelated donors, or haploidentical donors. The analysis showed that omidubicel was associated with more rapid neutrophil recovery (median: 10 days) compared to all other donor sources (median 15-20 days; p<0.001). While platelet recovery took longer in the omidubicel cohort, rates of severe acute and chronic graft versus host disease (GVHD) were comparable. Importantly, analyses of non-relapse mortality, disease-free survival, and overall survival showed similar results among all donor sources. While the phase 3 study compared omidubicel to standard cord blood, these real-world data reinforce the clinical utility of omidubicel as a graft source in patients in need of an allogeneic stem cell transplant.

About Omidubicel

Omidubicel is an advanced cell therapy candidate developed as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment in comparison to standard umbilical cord blood in an international, multi-center, randomized Phase 3 study (NCT0273029) in patients with hematologic malignancies undergoing allogeneic bone marrow transplant. The Phase 3 study also showed reduced time to platelet engraftment, reduced infections, and fewer days of hospitalization. One-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the US and EU.

The BLA for omidubicel has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 30, 2023. If approved, omidubicel will be the first allogeneic advanced stem cell therapy donor source for patients with blood cancers in need of a stem cell transplant.

Omidubicel is an investigational stem cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority. For more information about omidubicel, please visit View Source

About NAM Technology

Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.

On November 3, 2022 Pfizer Inc. (NYSE:PFE) reported its investigational cancer immunotherapy, elranatamab, received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of people with relapsed or refractory multiple myeloma (RRMM) (Press release, Pfizer, NOV 3, 2022, View Source [SID1234623033]). Elranatamab is a B-cell maturation antigen (BCMA)-CD3-targeted bispecific antibody (BsAb).

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"The FDA’s Breakthrough Designation recognizes the potential of elranatamab as an innovative medicine for people with multiple myeloma whose disease has relapsed or is refractory to existing treatments, which at present leaves very few avenues for staving off this currently incurable cancer," said Chris Boshoff, M.D., Ph.D., Chief Development Officer, Oncology and Rare Disease, Pfizer Global Product Development. "This marks Pfizer’s twelfth FDA Breakthrough Therapy Designation in Oncology, a testament to our relentless commitment to developing transformational cancer medicines in areas of high unmet need. We look forward to working with the FDA to accelerate the development of this therapy."

The FDA’s Breakthrough Therapy Designation is intended to expedite the development and review of a medicine that is intended to treat a serious or life-threatening disease and preliminary clinical evidence indicates the drug may demonstrate substantial improvement over existing therapies.1

BsAbs are a novel form of cancer immunotherapy that bind to and engage two different targets at once. One arm binds directly to specific antigens on cancer cells and the other arm binds to T-cells, bringing both cell types together. Elranatamab is designed to bind to BCMA, which is highly expressed on the surface of multiple myeloma (MM) cells, and the CD3 receptor found on the surface of T-cells, bridging them together and activating the T-cells to kill the myeloma cells. The binding affinity of elranatamab for BCMA and CD3 has been engineered to elicit potent T-cell mediated anti-myeloma activity. Elranatamab is administered subcutaneously, which offers more convenience over intravenous administration, and may mitigate the risk of potential adverse events, such as cytokine release syndrome (CRS).

The Breakthrough Therapy Designation is based on six-month follow-up data from cohort A (n=123) of MagnetisMM-3, an open-label, multicenter, single arm, Phase 2 study evaluating the safety and efficacy of elranatamab monotherapy in patients with RRMM. Patients received subcutaneous (SC) elranatamab 76 mg weekly (QW) with a 2-step-up priming dose regimen administered during the first week. The study showed elranatamab demonstrated a manageable safety profile, and at a median follow-up of 6.8 months, patients achieved an overall response rate (ORR) of 61.0%. Among responders, there was 90.4% probability of maintaining a response ≥6 months. The most common treatment-emergent adverse event (TEAE) regardless of causality was CRS (57.9%), with the majority of events reported being either Grade 1 (43.2%) or Grade 2 (14.2%). Updated data from MagnetisMM-3 will be presented at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition 2022 (ASH 2022), taking place December 10-13, 2022, in New Orleans.

MagnetisMM-3 is part of the robust MagnetisMM clinical research program, which has registration-intent trials planned or ongoing that explore elranatamab both as monotherapy and in combination with standard or novel therapies, spanning multiple patient populations from newly diagnosed multiple myeloma (NDMM), double-class exposed disease and RRMM.

In addition to the Breakthrough Therapy Designation, elranatamab has been granted Orphan Drug Designation by the FDA and the European Medicines Agency (EMA) for the treatment of MM. The FDA and EMA have granted elranatamab Fast Track Designation and the PRIME scheme, respectively, for the treatment of patients with RRMM. The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has also granted elranatamab Innovative Medicine Designation and the Innovation Passport, for the treatment of MM.

About Multiple Myeloma

MM is a blood cancer that affects plasma cells made in the bone marrow. Healthy plasma cells make antibodies that help the body fight infection. According to the latest figures available, there are over 34,000 new cases of MM diagnosed annually in the U.S. and 176,000 globally.2, 3 Despite treatment advances, MM remains incurable. The median overall survival is just over five years, and most patients receive four or more lines of therapy.4

About Pfizer Oncology

At Pfizer Oncology, we are committed to advancing medicines wherever we believe we can make a meaningful difference in the lives of people living with cancer. Today, we have an industry-leading portfolio of 24 approved innovative cancer medicines and biosimilars across more than 30 indications, including breast, genitourinary, colorectal, blood and lung cancers, as well as melanoma.

On November 3, 2022 Gilead Sciences, Inc. (Nasdaq: GILD) and Kite, a Gilead Company, reported that it will support 30 data presentations, including seven oral presentations in large B-cell lymphoma (LBCL), and two oral presentations on investigator-sponsored studies in acute myeloid leukemia (AML) during the 64th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (December 10-13) (Press release, Gilead Sciences, NOV 3, 2022, View Source [SID1234623032]).

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"Every year, we see more patients benefitting in profound ways from CAR T-cell therapy," said Frank Neumann, MD, PhD, Senior Vice President, Global Head of Clinical Development, Kite. "The breadth of real-world evidence and multi-year follow-up in pivotal studies we present this year can reinforce physicians’ confidence in the durability and reliability of Kite’s CAR T-cell therapies."

Key presentations for Kite’s CAR T-cell therapies include three-year results from ZUMA-5 in indolent non-Hodgkin lymphoma (iNHL) and exploratory data from the three-year results from ZUMA-2 in mantle cell lymphoma (MCL) and two-year results from ZUMA-3 in B-cell acute lymphoblastic leukemia (ALL). Additional Kite research will focus on new sub-analyses of ZUMA-7 in LBCL, and real-world experience studying the impact of time from leukapheresis to infusion on patient outcomes.

"We continue to grow the body of evidence for new therapeutic options that improve how we treat blood cancers, which include some of the most challenging tumors," said Bill Grossman, MD, PhD, Senior Vice President, Therapeutic Area Head, Gilead Oncology. "With our recently announced collaboration with MacroGenics to develop their second-generation CD123 bispecific antibody, we are excited about our presence at ASH (Free ASH Whitepaper) and the potential of our pipeline to improve patient outcomes across a variety of hematologic cancers."

An oral presentation showcasing results from a Phase 1/2 study of our investigational, anti-CD47 immunotherapy, in combination with azacitidine and venetoclax in patients with newly diagnosed older/unfit AML and relapsed/refractory AML also will be presented.

On November 3, 2022 Schrödinger, Inc. (Nasdaq: SDGR), whose physics-based computational platform is transforming the way therapeutics and materials are discovered, reported that new preclinical data on its CDC7 inhibitor, SGR-2921, will be presented during a poster session at the American Society of Hematology (ASH) (Free ASH Whitepaper) 64th Annual Meeting taking place virtually and in New Orleans, Louisiana, December 10-13, 2022 (Press release, Schrodinger, NOV 3, 2022, View Source [SID1234623031]).

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CDC7 is a cell cycle kinase involved in DNA replication and is an important activator of replication stress and DNA damage responses. CDC7 inhibition is considered a promising therapeutic approach for the treatment of cancers, including acute myeloid leukemia (AML). Schrödinger is advancing SGR-2921 through investigational new drug (IND)-enabling studies with plans to initiate a Phase 1 trial in patients with relapsed/refractory acute myeloid leukemia (AML) in the second half of 2023.

On November 3, 2022 MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company creating innovative medical solutions utilizing the latest biotechnology, reported that three abstracts highlighting data and information from the clinical development program evaluating the intermittent dosing of zandelisib, an investigational phosphatidylinositol 3-kinase delta ("PI3Kδ") inhibitor for the treatment of B-cell malignancies, will be presented at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) 2022 Annual Meeting to be held December 10 – 13, 2022 (Press release, MEI Pharma, NOV 3, 2022, View Source [SID1234623030]).

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