Omidubicel: An investigational advanced cell therapy ... · hematologic malignancies, bone marrow...
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Each year, approximately 8,000 patients in the U.S. with a hematologic malignancy receive an allogeneic bone marrow transplant using a donor source. 5 An estimated 40 percent of patients eligible for bone marrow transplant do not receive one for many reasons, including the inability to find a matched donor. 2 The transplant process can lead to serious complications, including infections and graft- versus-host-disease (GVHD) Omidubicel: An investigational advanced cell therapy designed to enhance the life-saving benefits of bone marrow transplant O midubicel, Gamida Cell’s lead investigational product candidate, is an advanced cellular therapy in Phase 3 development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution. 1 For certain high-risk hematologic malignancies, bone marrow transplant is the only potentially curative treatment. However, an estimated 40 percent of eligible patients do not receive a transplant for a number of reasons, including the inability to find a matched donor. 2 For those who receive a transplant, the process itself may result in serious complications including graft-versus-host disease and infections. Omidubicel has the potential to address these challenges and improve treatment outcomes for patients. Data from a Phase 1/2 clinical study published in the Journal of Clinical Oncology demonstrated that treatment with omidubicel resulted in rapid and durable engraftment — a crucial treatment milestone in which donor cells begin producing healthy cells — and was generally well tolerated. 3 Bone marrow transplant: Fast facts ~ ~ About the Phase 3 clinical study Gamida Cell is currently conducting a multi-center, international, randomized Phase 3 clinical study to evaluate the safety and efficacy of omidubicel compared to standard umbilical cord blood transplant in patients with high-risk blood cancers who do not have a suitable matched donor. 4 The study includes patients living with acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome and lymphoma. This clinical study is enrolling approximately 120 patients aged 12-65 at over 40 clinical centers in the U.S., Europe and Asia. gamida-cell.com United States Europe Asia For more information, including a complete list of study centers, please visit https://bit.ly/2EBhmOQ.
Transcript of Omidubicel: An investigational advanced cell therapy ... · hematologic malignancies, bone marrow...
Each year, approximately 8,000 patients in the U.S. with a
hematologic malignancy receive an allogeneic bone marrow
transplant using a donor source.5
An estimated 40 percent of patients eligible for bone marrow
transplant do not receive one for many reasons, including the
inability to find a matched donor.2
The transplant process can lead to serious complications, including infections and graft-versus-host-disease (GVHD)
Omidubicel: An investigational advanced cell therapy designed to enhance the life-saving benefits of bone marrow transplant
Omidubicel, Gamida Cell’s lead investigational product candidate, is an advanced cellular therapy in Phase 3 development as a potential life-saving allogeneic
hematopoietic stem cell (bone marrow) transplant solution.1 For certain high-risk hematologic malignancies, bone marrow transplant is the only potentially curative treatment. However, an estimated 40 percent of eligible patients do not receive a transplant for a number of reasons, including the inability to find a matched donor.2 For those who receive a transplant, the process itself may result in serious complications including graft-versus-host disease and infections.
Omidubicel has the potential to address these challenges and improve treatment outcomes for patients. Data from a Phase 1/2 clinical study published in the Journal of Clinical Oncology demonstrated that treatment with omidubicel resulted in rapid and durable engraftment — a crucial treatment milestone in which donor cells begin producing healthy cells — and was generally well tolerated.3
Bone marrow transplant: Fast facts
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About the Phase 3 clinical study
Gamida Cell is currently conducting a multi-center, international, randomized Phase 3 clinical study to evaluate the safety and efficacy of omidubicel compared to standard umbilical cord blood transplant in patients with high-risk blood cancers who do not have a suitable matched donor.4 The study includes patients living with acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome and lymphoma. This clinical study is enrolling approximately 120 patients aged 12-65 at over 40 clinical centers in the U.S., Europe and Asia.
gamida-cell.com
United States
Europe
Asia
For more information, including a complete list of study centers, please visit https://bit.ly/2EBhmOQ.
For media inquiries, please contact:
Max StendahlTen Bridge Communications [email protected]
For other corporate or investor relations inquiries, please contact:
Jaren Irene MaddenGamida Cell Ltd. [email protected]
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.
References:
1 Gamida Cell’s lead development candidate consists of omidubicel (expanded hematopoietic stem cells) and differentiated immune cells, including T cells. Gamida Cell refers to the two components collectively as “omidubicel.”
2 Besse, K. et al. Estimating Demand and Unmet Need for Allogeneic Hematopoietic Cell Transplantation in the United States Using Geographic Information Systems. Journal of Oncology Practice Vol. 11, Issue 2. 2015.
3 Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo with Nicotinamide. Horwitz M.E., Wease S., Blackwell B., et al. Journal of Clinical Oncology. 2018 DOI: 10.1200/JCO.18.00053.
4 ClinicalTrials.gov identifier NCT02730299
5 D’Souza A., Fretham C. Current Uses and Outcomes of Hematopoietic Cell Transplantation (HCT): CIBMTR Summary Slides, 2018. Available at https://www.cibmtr.org.
What is omidubicel?
Gamida Cell’s proprietary nicotinamide-based, or NAM-based, expansion technology enables donor cells to grow while maintaining functionality. Omidubicel consists of NAM-expanded hematopoietic stem cells and differentiated immune cells, including T cells. The final cell therapy product may be cryopreserved until the patient is ready to begin the transplant, when it is thawed and infused.
Anticipated development timeline
Omidubicel is the first bone marrow transplant product to receive FDA Breakthrough Therapy Designation and has also received Orphan Drug Designation in the U.S. and EU. Gamida Cell expects to complete patient enrollment in the Phase 3 study by the end of 2019 with topline data anticipated in the first half of 2020. Based on the results of the Phase 3 clinical study, the company expects to submit a biologics license application to the FDA by the end of 2020.
Omidubicel: An investigational advanced cell therapy designed to enhance the life-saving benefits of bone marrow transplant
