On November 4, 2020 PharmaEssentia Corporation (TPEx: 6446), a global biopharmaceutical innovator leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, reported that data reflecting five years of experience with ropeginterferon alfa-2b in polycythemia vera (PV) will be presented during the virtual 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (Press release, PharmaEssentia, NOV 4, 2020, View Source [SID1234569879]).

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Findings from "Long-Term Use of Ropeginterferon Alpha-2b in Polycythemia Vera: 5-Year Results from a Randomized Controlled Study and Its Extension" (Presentation 481 in Session 634) will be featured as an oral presentation on Sunday, December 6th at 2:30 PM PT.

"We look forward to contributing to the scientific dialogue around opportunities to improve care in the area of myeloproliferative neoplasms, and specifically in PV, during the virtual ASH (Free ASH Whitepaper) meeting," said Meredith Manning, U.S. General Manager for PharmaEssentia. "Our robust research offers a new, innovative perspective in this category and illustrates our goal to reset expectations and improve patient outcomes."

PharmaEssentia has focused its efforts on therapeutic innovation in the category of myeloproliferative neoplasms (MPNs), which are caused by specific genetic mutations that lead to overproduction of blood components including white or red blood cells, or platelets. In PV, the vast majority of which is caused by a JAK2 V617F mutation, the bone marrow produces excessive red blood cells, causing the blood to be thicker than normal and potentially leading to a range of complications.1,2 PV is estimated to affect more than 160,000 people in the U.S. alone,1 who have progressively burdensome symptoms. Without proper management, the disease can progress into myelofibrosis and malignancies including acute myeloid leukemia.3

Follow PharmaEssentia on LinkedIn to get news and updates on our activity at the virtual ASH (Free ASH Whitepaper) Annual Meeting.

About Ropeginterferon alfa-2b

Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon that has been engineered with an optimized profile to support improved pharmacokinetic properties, tolerability and convenience. It is designed for administration once every two weeks, or once every four weeks during long-term maintenance. Ropeginterferon alfa-2b has Orphan Drug designation for treatment of polycythemia vera (PV) in the United States. Marketed as Besremi in Europe, the product was approved by the European Medicines Agency (EMA) in 2019. Ropeginterferon alfa-2b was discovered and is manufactured by PharmaEssentia in its Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018.

About Polycythemia Vera

Polycythemia Vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. This condition may result in cardiovascular complications such as thrombosis and embolism, as well as transformation to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2.3

On November 4, 2020 GlycoMimetics, Inc. (Nasdaq: GLYC) reported that three abstracts including data from the Company’s clinical and research portfolio have been accepted for oral presentations and two abstracts have been accepted for poster presentations at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, to be held virtually December 5-8, 2020 .

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Of particular note are primary and key secondary endpoint data from additional analysis of the Phase 3 RESET study evaluating the efficacy of rivipansel in VOC. These findings point to the potential benefits, clinical improvements and improved outcomes associated with early treatment with GlycoMimetics’ wholly-owned product candidate, which include shorter hospital stays for patients and reduced need for IV opioids to treat pain.

A second presentation includes data from two different preclinical models of VOC using GlycoMimetics’ highly potent and specific E-selectin antagonist, GMI-1687. This presentation will highlight the product candidate’s potential for intravenous and subcutaneous administration to treat VOC by inhibiting occlusions and restoring blood flow.

A third presentation discloses how targeting E-selectin with uproleselan may help patients with AML overcome resistance to venetoclax combined with hypomethylating agent (HMA) based therapy.

Poster presentations convey how GMI-1359, a rationally-designed small molecule that inhibits E-selectin and CXCR4 (a chemokine receptor), enhances sorafenib’s anti-leukemia effect in pre-clinical AML models; and how GMI-1359 can uniquely generate motility-enhancing signals in AML cells and deplete AML cells from protective vascular niches in the bone marrow.

"GlycoMimetics is honored to have five abstracts from across our product candidate portfolio selected for presentations at this year’s ASH (Free ASH Whitepaper) meeting. The data convey new insights into the critical role of E-selectin in both malignant and inflammatory, adhesion-mediated conditions, suggesting novel treatment options for unmet need in sickle cell disease and AML," said Helen Thackray, MD, FAAP, GlycoMimetics’ Chief Medical Officer.

Details on GlycoMimetics presentations at the ASH (Free ASH Whitepaper) Meeting are as follows:

Title: Restoration of Normal Blood Flow in Mouse Models of Sickle Cell Vaso-occlusion Following Intravenous or Subcutaneous Administration of a Highly Potent E-Selectin Specific Inhibitor.
Presenters: Madhan Thamilarasan, William E. Fogler, John L. Magnani and Rahima Zennadi.
Session: 113 Hemoglobinopathies, Excluding Thalassemia—New Genetic Approaches to Sickle Cell Disease: New Insights Into Sickle Cell Disease Pathophysiology.
Date and Time: December 5, 2020. 2:00 – 3:30 p.m. EST
Presentation Time: 2:45 p.m.

Title: Targeting E-selectin with GMI-1271 Overcomes Microenvironment-mediated Resistance to Venetoclax/HMA Therapy.
Presenters: Kyung Hee Chang, Muharrem Muftuoglu, Weiguo Zhang, Mahesh Basyal, Lauren Ostermann, William E. Fogler, John L. Magnani and Michael Andreeff.
Session: 604 Molecular Pharmacology and Drug Resistance in Myeloid Diseases.
Date and Time: Saturday, December 5, 2020. 2:00 – 3:30 p.m.
Presentation Time: 3:15 p.m.

Title: Dual CXCR4 and E-selectin Inhibition (GMI-1359) Rapidly Increases AML Cellular Motility Prior to Intravasation and Vascular Niche Depletion Observed by Intravital Bone Marrow 2-Photon Microscopy.
Presenters: Tomasz Zal, M. Anna Zal, Mateusz Rytelewski, Kyung Hee Chang, Rodrigo Jacamo, William E. Fogler, John L. Magnani and Michael Andreeff.
Session: 616 Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster II.
Date: Sunday, December 6, 2020.
Virtual Poster Hall: 7 a.m. – 3:30 p.m.

Title: Combined Blockage of E-selectin and CXCR4 (GMI-1359) Enhances Anti-Leukemia Effect of FLT3 Inhibition (Sorafenib) and Protects Hematopoiesis in Pre-clinical AML Models.
Presenters: Weiguo Zhang, Kyung Hee Chang, Mahesh Basyal, Yannan Jia, Lauren Ostermann, William E. Fogler, John L. Magnani, M. Anna Zal, Tomasz Zal and Michael Andreeff.
Session: 616 Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster III.
Date: Monday, December 7, 2020.
Virtual Poster Hall: 7 a.m. – 3:30 p.m.

Title: Early Initiation of Treatment with Rivipansel for Acute Vaso-Occlusive Crisis in Sickle Cell Disease (SCD) Achieves Earlier Discontinuation of IV Opioids and Shorter Hospital Stay: Reset Clinical Trial Analysis
Presenter: Helen Thackray
Session Name: 114 Hemoglobinopathies, Excluding Thalassemia—Clinical: Novel Treatments for Sickle Cell Disease
Date and Time: Monday, December 7, 2020. 1:30 – 3:00 p.m.
Presentation Time: 1:45 p.m.

The accepted abstracts are available online through the ASH (Free ASH Whitepaper) meeting website, View Source

About Rivipansel

Rivipansel, the company’s wholly-owned glycomimetic drug candidate that binds to all three members of the selectin family (E-, P- and L-selectin), was GlycoMimetics’ first candidate to enter clinical development. After the Phase 3 RESET trial conducted by Pfizer, GlycoMimetics’ former collaborator, did not meet its primary or key secondary efficacy endpoints in 2019, new efficacy data from an additional analysis of rivipansel were published in June 2020 and subsequently presented at the Foundation for Sickle Cell Disease Research Meeting in September 2020. GlycoMimetics is engaging with the FDA to identify what, if any, next steps to take, with a focus on determining if there is a potential streamlined path forward for this asset in sickle cell disease.

About GMI-1687

Discovered and developed by GlycoMimetics, GMI-1687 is a highly-targeted, highly-potent E-selectin antagonist. It has been shown in preclinical studies to be bioavailable via subcutaneous administration. At the 2018 Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), data presented in a poster about GMI-1687 pointed to the potential for a life-cycle extension for GlycoMimetics’ uproleselan. The investigational drug has also been shown to represent a more highly-potent and subcutaneously bioavailable potential life-cycle extension for rivipansel.

About Uproleselan (GMI-1271)

Discovered and developed by GlycoMimetics, uproleselan is an investigational, first-in-class, targeted inhibitor of E-selectin. Uproleselan (yoo’ pro le’ sel an), currently in a comprehensive Phase 3 development program in AML, has received Breakthrough Therapy Designation from the U.S. FDA for the treatment of adult AML patients with relapsed or refractory disease. Uproleselan is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. In a Phase 1/2 clinical trial, uproleselan was evaluated in both newly diagnosed elderly and relapsed or refractory patients with AML. In both populations, patients treated with uproleselan together with standard chemotherapy achieved better-than-expected remission rates and overall survival compared to historical controls, which have been derived from results from third-party clinical trials evaluating standard chemotherapy, as well as lower-than-expected induction-related mortality rates. Treatment in these patient populations was generally well-tolerated, with fewer than expected adverse effects.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules involved in tumor trafficking and metastatic spread. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as AML and multiple myeloma or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer, as well as in osteosarcoma, a rare pediatric tumor. GMI-1359 has completed a Phase 1 clinical trial in healthy volunteers. The Duke University Phase 1b clinical study in breast cancer patients is designed to enable investigators to identify an effective dose of the drug candidate and to generate initial biomarker data around the drug’s activity. GMI-1359 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for the treatment of osteosarcoma, a rare cancer affecting about 900 adolescents a year in the United States.

On November 4, 2020 Mauna Kea Technologies (Paris:MKEA) (OTCQX:MKEAY) (Euronext: MKEA) inventor of Cellvizio, the multidisciplinary probe and needle-based confocal laser endomicroscopy (pCLE/nCLE) platform, reported the publication of a peer-reviewed meta-analysis and of an international Delphi consensus report based on a systematic, evidence-based review of endoscopic ultrasound (EUS) guided needle-based confocal laser endomicroscopy for pancreatic cystic lesion (PCL) evaluation (Press release, Mauna Kea Technologies, NOV 4, 2020, View Source [SID1234569877]).

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The first publication entitled "Needle-based Confocal Laser Endomicroscopy in Pancreatic Cysts: a Meta-Analysis", was published in the European Journal of Gastroenterology & Hepatology (2020, DOI: 10.1097/MEG.0000000000001728). Ten studies enrolling 536 patients were included and the authors evaluated diagnostic accuracy, pooled sensitivity, specificity, and mean procedural time. The meta-analysis concluded that confocal laser endomicroscopy "clearly outperformed" endoscopic ultrasound fine-needle aspiration (EUS-FNA) in terms of diagnostic accuracy (odds ratio 3.94, 1.58–9.82; P = 0.003) and supports the use of needle-based confocal laser endomicroscopy as a safe and effective tool in the diagnostic algorithm of pancreatic cysts.

The second publication entitled "Confocal Endomicroscopy for the Evaluation of Pancreatic Cystic Lesions: A Systematic Review and an International Delphi Consensus Report", was published in the peer-reviewed journal Endoscopy International Open (2020, DOI: 10.1055/a-1229-4156) and was based on consensus statements from an international panel of 15 experts in pancreatic disease that conducted evidence-based reviews of the applications, outcomes, procedural processes, indications, training, and credentialing of EUS-nCLE in the management of PCLs. The consensus report reflected a high level of agreement pertaining to expert consensus statements and established that EUS-guided nCLE is a minimally invasive procedure that improves evaluation of PCLs and "should be systematically considered when EUS-FNA is indicated for PCL evaluation."

The consensus report also concluded that the use of nCLE as an adjunct to standard EUS-FNA could positively impact patient management and improve healthcare resource utilization by reducing the number of misdiagnoses and preventing redundant follow-up investigations and unnecessary surgery.

"The new meta-analysis and consensus report provide further support that Cellvizio plays a key role in the evaluation of pancreatic cysts and can significantly improve the speed and accuracy of reaching a conclusive diagnosis," said Robert L. Gershon, Chief Executive Officer of Mauna Kea Technologies. "The level of clinical evidence and support suggests that hospitals performing endoscopic evaluation of pancreatic cysts would benefit their patients by incorporating Cellvizio as part of the management of their patients. This represents approximately 75,000 procedures in the US alone. We look forward to the growing adoption of Cellvizio for this important application."

On November 4, 2020 ADC Therapeutics SA (NYSE:ADCT), a late clinical-stage oncology-focused biotechnology company pioneering the development and commercialization of highly potent and targeted antibody drug conjugates (ADCs) for patients with hematological malignancies and solid tumors, reported that eight abstracts have been selected for presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which is being held virtually from December 5-8, 2020 (Press release, ADC Therapeutics, NOV 4, 2020, View Source [SID1234569876]). Presentations will feature data on three of the Company’s pyrrolobenzodiazepine (PBD)-based ADCs – loncastuximab tesirine (Lonca), camidanlumab tesirine (Cami) and ADCT-602.

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"The breadth of data that will be presented at the ASH (Free ASH Whitepaper) Annual Meeting reflects the potential of our product candidates and speaks to our leadership in the development of next-generation ADCs to improve outcomes for patients with blood cancers," said Jay Feingold, MD, PhD, Senior Vice President and Chief Medical Officer of ADC Therapeutics. "Notably, we look forward to sharing subgroup data from the Lonca LOTIS 2 pivotal trial in relapsed or refractory diffuse large B-cell lymphoma, interim results from our LOTIS 3 trial evaluating Lonca in combination with ibrutinib in patients with advanced diffuse large B-cell lymphoma or mantle cell lymphoma, as well as preliminary results from our pivotal Phase 2 trial of Cami in patients with relapsed or refractory Hodgkin lymphoma."

Lonca Presentations

Efficacy and Safety of Loncastuximab Tesirine (ADCT-402) in Relapsed/Refractory Diffuse Large B-Cell Lymphoma
Abstract: 1183
Date and Time: Saturday, December 5, 2020, 7 a.m. – 3:30 p.m. PST
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Poster I
Presenter: Paolo F. Caimi, MD, Case Comprehensive Cancer Center, Case Western Reserve University

Characteristics and Treatment Patterns of Patients with Relapsed/Refractory Diffuse Large B-Cell Lymphoma Who Received ≥3 Lines of Therapies
Abstract: 1651
Date and Time: Saturday, December 5, 2020, 7 a.m. – 3:30 p.m. PST
Session: 905. Outcomes Research—Malignant Conditions (Lymphoid Disease): Poster I
Presenter: Jipan Xie, MD, PhD, Analysis Group, Inc.

Interim Results of Loncastuximab Tesirine Combined with Ibrutinib in Diffuse Large B-Cell Lymphoma or Mantle Cell Lymphoma (LOTIS-3)
Abstract: 2099
Date and Time: Sunday, December 6, 2020, 7 a.m. – 3:30 p.m. PST
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Poster II
Presenter: Julien Depaus, MD, CHU UCL Namur

Safety and Antitumor Activity Study Evaluating Loncastuximab Tesirine and Rituximab Versus Immunochemotherapy in Diffuse Large B-Cell Lymphoma
Abstract: 2107
Date and Time: Sunday, December 6, 2020, 7 a.m. – 3:30 p.m. PST
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Poster II
Presenter: Yuliya Linhares, MD, Miami Cancer Institute, Baptist Health South Florida

Cami Presentations

Preliminary Results of a Phase 2 Study of Camidanlumab Tesirine (Cami), a Novel Pyrrolobenzodiazepine-Based Antibody-Drug Conjugate, in Patients with Relapsed or Refractory Hodgkin Lymphoma
Abstract: 474
Date and Time: Sunday, December 6, 2020, 3 p.m. PST
Session: 624. Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: Clinical Studies in Hodgkin Lymphoma
Presenter: Alex Herrera, MD, City of Hope Medical Center

Pharmacokinetic and Pharmacodynamic Correlates from the Phase 1 Study of Camidanlumab Tesirine (Cami) in Patients with Relapsed or Refractory Hodgkin Lymphoma and Non-Hodgkin Lymphoma
Abstract: 2981
Date and Time: Monday, December 7, 2020, 7 a.m. – 3:30 p.m. PST
Session: 624. Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: Poster III
Presenter: Joseph Boni, ADC Therapeutics

Combination of Camidanlumab Tesirine, a CD25-Targeted ADC, with Gemcitabine Elicits Synergistic Anti-Tumor Activity in Preclinical Tumor Models
Abstract: 1178
Date and Time: Saturday, December 5, 2020, 7 a.m. – 3:30 p.m. PST
Session: 625. Lymphoma: Pre-Clinical—Chemotherapy and Biologic Agents: Poster I
Presenter: Francesca Zammarchi, ADC Therapeutics

ADCT-602 Presentation

Analysis of Adct-602 Pre-Clinical Activity in B-Cell Lymphoma Models and Identification of Potential Biomarkers for Its Activity
Abstract: 3011
Date and Time: Monday, December 7, 2020, 7 a.m. – 3:30 p.m. PST
Session: 625. Lymphoma: Pre-Clinical—Chemotherapy and Biologic Agents: Poster III
Presenter: Francesco Bertoni, MD, Institute of Oncology Research, Università della Svizzera italiana

Online Publication in November Supplemental Issue of Blood
Symptoms, Health-Related Quality of Life, and Tolerability of Loncastuximab Tesirine in Patients with Relapsed or Refractory Diffuse Large B Cell Lymphoma
Abstract Number: 3919

The abstracts are available through the ASH (Free ASH Whitepaper) online meeting program and will be published in the November supplemental issue of Blood.

About Loncastuximab Tesirine (Lonca)

Loncastuximab tesirine (Lonca, formerly ADCT-402) is an antibody drug conjugate (ADC) composed of a humanized monoclonal antibody directed against human CD19 and conjugated through a linker to a pyrrolobenzodiazepine (PBD) dimer cytotoxin. Once bound to a CD19-expressing cell, Lonca is designed to be internalized by the cell, following which the warhead is released. The warhead is designed to bind irreversibly to DNA to create highly potent interstrand cross-links that block DNA strand separation, thus disrupting essential DNA metabolic processes such as replication and ultimately resulting in cell death. CD19 is a clinically validated target for the treatment of B-cell malignancies.

In September 2020, ADC Therapeutics submitted a Biologics License Application to the U.S. Food and Drug Administration seeking accelerated approval for Lonca for the treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL). Lonca is being evaluated in LOTIS 2, a pivotal Phase 2 clinical trial in patients with relapsed or refractory DLBCL, LOTIS 3, a Phase 1/2 clinical trial in combination with ibrutinib in patients with relapsed or refractory DLBCL or mantle cell lymphoma, and LOTIS 5, a Phase 3 confirmatory clinical trial in combination with rituximab in patients with relapsed or refractory DLBCL.

About Camidanlumab Tesirine (Cami)

Camidanlumab tesirine (Cami, formerly ADCT-301) is an antibody drug conjugate (ADC) comprised of a monoclonal antibody that binds to CD25 (HuMax-TAC, licensed from Genmab A/S), conjugated to the pyrrolobenzodiazepine (PBD) dimer payload, tesirine. Once bound to a CD25-expressing cell, ADCT-301 is internalized into the cell where enzymes release the PBD-based warhead killing the cell. This applies to CD25-expressing tumor cells, and also to CD25-expressing Tregs. The intra-tumoral release of its PBD warhead may also cause bystander killing of neighboring tumor cells and PBDs have also been shown to induce immunogenic cell death. All of these properties of Cami may enhance immune-mediated anti-tumor activity. Cami is being evaluated in a pivotal Phase 2 clinical trial in patients with relapsed or refractory Hodgkin lymphoma (HL), as well as in a Phase 1a/1b clinical trial in patients with relapsed or refractory HL and non-Hodgkin lymphoma and a Phase 1b clinical trial as monotherapy and in combination with pembrolizumab in solid tumors.

On November 4, 2020 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported that updated data from the ongoing 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, will be presented in an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) 62nd Annual Meeting, which is being held virtually from December 5-8, 2020. NK cell immunotherapies are thought to offer tremendous potential for transforming the care of hematologic malignancies. Gamida Cell is pioneering a novel approach that harnesses the power of its cell expansion technology, which uniquely improves antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells.

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Additionally, new data from the ongoing Phase 1/2 study of omidubicel in patients with severe aplastic anemia will be shared in a poster presentation during the meeting. Omidubicel is an investigational advanced cell therapy in development as a potential life-saving treatment option for patients in need of a bone marrow transplant.

Omidubicel is also being evaluated in a Phase 3 study in patients with hematologic malignancies. Earlier this year, Gamida Cell reported that its Phase 3 study of omidubicel met its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in recovery from a stem cell transplant. Last month, Gamida Cell also reported that all three secondary endpoints for the study demonstrated statistical significance. The secondary endpoints in the study include outcomes for: platelet engraftment, infections and hospitalizations.

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

Title: 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
Abstract Number: 63
Lead Author: Veronika Bachanova, M.D., Ph.D., Division of Hematology, Oncology and Transplantation, University of Minnesota, Minneapolis, MN
Time: Saturday, December 5, 2020, 7:30 a.m. – 9:00 a.m. PT (session time) and 7:30 a.m. PT (presentation)

Title: Rapid Engraftment, Immune Recovery, and Resolution of Transfusion Dependence in Treatment-Refractory Severe Aplastic Anemia Following Transplantation with Ex Vivo Expanded Umbilical Cord Blood (Omidubicel)
Abstract Number: 1531
Lead Author: Mohamed Samour, M.D., Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD
Time: Saturday, December 5, 2020, 7:00 a.m. – 3:30 p.m. PT

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

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

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

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