Gamida Cell Presents Analysis of Observational Data Demonstrating the Impact of Donor Age in Hematopoietic Stem Cell Transplant Outcomes at the Virtual Cord Blood Connect Meeting
— Study shows younger donor age is associated with improved outcomes following bone marrow transplant —
BOSTON--(BUSINESS WIRE)--Sep. 10, 2020-- Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious blood diseases, today announced data from an observational study demonstrating that younger donor age is associated with more rapid time to neutrophil engraftment and increased overall survival in patients who received hematopoietic stem cell transplant (HSCT, or bone marrow transplant) for the treatment of hematological malignancies. These data are being presented at the Cord Blood Connect Meeting, which is taking place virtually today and on September 17.
The study is the result of a research agreement between Gamida Cell and the CIBMTR® (Center for International Blood and Marrow Transplant Research®) designed to collect and analyze health outcomes data in patients with hematologic malignancies who receive a hematopoietic stem cell transplant or cellular therapy infusion, including bone marrow transplant graft from various donor sources. The study evaluated clinical outcomes for 660 patients in the CIBMTR registry who underwent a bone marrow transplant with a matched unrelated, mismatched unrelated or haploidentical graft source contemporaneous to the Phase 3 study of omidubicel, Gamida Cell’s investigational advanced cell therapy in development as a treatment option for patients in need of a bone marrow transplant. Key clinical outcomes, including time to neutrophil engraftment and overall survival, were improved for patients with donors under the age of 30.
“As new graft options evolve for bone marrow transplant, and we as a field learn more about the long-term patient outcomes of these graft options, the selection algorithms that are used to match patients with donors must also evolve to take into account the most current clinical data,” said Ronit Simantov, M.D., chief medical officer at Gamida Cell. “These data indicate that donor age is a factor in clinical outcomes and that donor age should be considered when matching patients with a graft source.”
In May, Gamida Cell reported that its Phase 3 study of omidubicel achieved its primary endpoint, demonstrating a highly statistically significant reduction (p < 0.001) in time to neutrophil engraftment, a key milestone in recovery from a bone marrow transplant. Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA). Gamida Cell expects to begin submitting the biologics license application for omidubicel to the FDA on a rolling basis in the fourth quarter of 2020.
“This analysis reinforces the importance of considering donor age when selecting a bone marrow graft source for patients, as donors aged 30 or younger resulted in improved overall survival,” said Julian Adams, Ph.D., chief executive officer at Gamida Cell. “As we look toward our anticipated regulatory submission of omidubicel to the FDA and potential approval, given that omidubicel is derived from cord blood, we believe these findings could potentially have future implications for considering omidubicel for any patient who does not have an available related or unrelated donor of suitable age.”
More Details About the Study
This observational study utilizes data from the CIBMTR® registry to analyze long-term safety and efficacy data for patients with hematologic malignancies who underwent a bone marrow transplant with a matched unrelated, mismatched unrelated or haploidentical graft source. The criteria for inclusion of patients and the outcomes evaluated in the analyses were consistent with those in the Phase 3 study of omidubicel. The median donor age was 30 (range 2-74) years.
Statistical analyses, performed by Gamida Cell, compared important clinical outcomes for patients with donors ≤ 30 years old (n=334) to patients with donors > 30 years of age (n=326). Patient demographics were well-balanced across the two donor age groups. The study demonstrated that neutrophil recovery was more rapid in patients with donors ≤ 30 years old (p = 0.013). Additionally, patients with donors ≤ 30 years of age showed improved overall survival at one year (p = 0.016).
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 and NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.1,2 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.
