On September 11, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, and the CIBMTR (Center for International Blood and Marrow Transplant Research) reported the entry into a research agreement to collect and analyze health outcomes data in patients with hematologic malignancies who receive an allogeneic hematopoietic stem cell transplant (HSCT, or bone marrow transplant) from various donor sources (Press release, Gamida Cell, SEP 11, 2019, View Source [SID1234539444]). The CIBMTR is an organization that collaborates with the global scientific community to advance hematopoietic cell transplantation (HCT) and cellular therapy worldwide.
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The newly launched observational study by the CIBMTR and Gamida Cell will include both retrospective and prospective data contemporaneous to the international, randomized, Phase 3 study of omidubicel, Gamida Cell’s investigational advanced cell therapy designed to enhance the life-saving benefits of bone marrow transplant. Topline data from the ongoing Phase 3 study is anticipated in the first half of 2020, and initial data from the multi-year observational study is anticipated next year. Omidubicel has not yet been approved for marketing in the United States or any other jurisdiction.
"We are pleased to announce this collaboration with Gamida Cell, a company aiming to make bone marrow transplant an option for more patients facing life-threatening blood diseases," said Mary M. Horowitz, M.D., M.S., chief scientific director of the CIBMTR. "This collaboration will leverage the CIBMTR’s deep experience collecting and analyzing data on both the short- and long‐term outcomes of patients undergoing a bone marrow transplant. We look forward to contributing to efforts to better understand real-world clinical outcomes."
"This agreement marks the beginning of Gamida Cell’s health outcomes research initiatives, and we are excited to partner with the CIBMTR, an organization with deep expertise in bone marrow transplantation and cellular therapy," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We are committed to improving outcomes for patients who are in need of a bone marrow transplant and look forward to better understanding the variables that influence health outcomes, as well as elucidating how omidubicel may fit into the treatment landscape."
Randomized clinical trials comparing different donor types suggest that clinical outcomes may vary significantly depending on the donor type. The goal of this real-world, observational study is to better understand the variables that influence the health outcomes of patients receiving a transplant from a source other than a fully matched family donor. As part of the research agreement, the CIBMTR will use its registry, which consists of clinical outcomes data on more than 500,000 stem cell transplants, to analyze long‐term safety and efficacy data for patients with hematologic malignancies who underwent a bone marrow transplant with an alternative donor source following myeloablative conditioning. The criteria for inclusion of patients and the endpoints evaluated in the analysis will be consistent with the design of the Phase 3 study of omidubicel.
About Omidubicel
Omidubicel (formerly known as NiCord), the company’s lead clinical program, 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). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In a Phase 1/2 clinical study, omidubicel demonstrated rapid and durable time to engraftment and was generally well-tolerated.1 A Phase 3 study evaluating omidubicel in patients with leukemia and lymphoma is ongoing in the U.S., Europe and Asia.2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.3 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 has not been evaluated by the U.S. Food and Drug Administration or any other health authority.