Gamida Cell Alternative to Bone Marrow Transplant

Gamida Cell announced additional analyses for the Phase II/III, multi-center, multi-national, historical cohort-controlled study to evaluate efficacy and safety of StemEx® as an alternative transplantation treatment for patients with high risk leukemia and lymphoma. Twenty-five bone marrow transplant centers worldwide treating 101 patients with hematologic malignancies following myeloablative therapy who could not find a family related matched bone marrow donor participated in the study.

For the final analyses, the clinical study evaluated the effects of StemEx®, used as part of a transplantation regimen, in comparison with a contemporaneous control group that received double cord blood transplant (DCBT) 2006-2010. This is an updated and more relevant historical control group than the original control (1995-2005). The primary endpoint was defined as the proportion of overall mortality at 100 days after transplantation.

Key findings show that a transplant using StemEx®: Improves survival at 100 days post transplantation. The analysis of this primary endpoint shows 15.8% 100-day mortality in the StemEx® group and 24.5% in the control group (p= 0.035). The primary endpoint was first reported on February 4, 2013. Please click here to see press release. Increases the number of patients with early hematopoietic recovery: neutrophil engraftment failure rates were lower in the StemEx® group (cumulative incidence 8.1%) than in the control group (cumulative incidence 14.5%; p=0.086). Shortens time to neutrophil engraftment. In the regression model for cumulative incidence the median time to neutrophil engraftment was 21 days in the StemEx® group and 28 days in the control group (p<0.0001). Shortens the time to platelet engraftment. In the regression model for cumulative incidence the median time to platelet engraftment was 54 days in the StemEx® group and 105 days in the control group (p=0.008). Provides a graft which is enriched with CD34+ stem/progenitor cells. CD34+ cells from the StemEx® portion correlate with early hematopoietic recovery of neutrophils (p=0.001) and platelets (p=0.007). The survival advantage was not statistically significant by day 180 with mortality of 32.7% in the StemEx® group and 34.7% in the control group (p=0.39). Does not create a significant difference in the level of grade III-IV acute GvHD – 19.4% in the StemEx® group and 16.9% in the control group (p=0.11). Is feasible with a robust manufacturing process and shipment logistics showing delivery within stability limits of 100% of all manufactured units, allowing prompt and reliable transplantation in multiple sites across three continents. Adverse events and serious adverse events in the StemEx® treatment group appear to reflect events expected in a patient population with hematologic malignancies, who are at high risk for concomitant morbidity events and mortality, without increased risk of post-transplant complications. “Phase II/III data suggests that StemEx® can serve as an alternative transplant treatment for patients who cannot find a matched bone marrow donor. The company plans to meet with the FDA this spring and with the EMA this autumn to continue discussions on the regulatory pathway for marketing approvals,” said Dr. Yael Margolin, president and CEO of Gamida Cell. To date, StemEx® has been developed by the Gamida Cell-Teva Joint Venture, equally owned by Gamida Cell and Teva Pharmaceutical Industries. The Joint Venture owns all global rights for the commercialization of StemEx®. The Gamida Cell – Teva Joint Venture is currently seeking a strategic partner for the global commercialization of StemEx®.

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