Gamida Cell Phase II/III Stem Cell Success

Gamida Cell announced that its flagship product, StemEx, reached its primary endpoint of improving overall survival in a Phase II/III study which compared the use of StemEx as part of a transplantation regimen to historical controls in the treatment of patients with hematological malignancies such as leukemia and lymphoma.

The primary endpoint is defined as the rate of mortality (%) within 100 days after transplantation. For

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the final analysis, the historical control cohort was comprised of a similar group of patients each transplanted with double cord blood during the years 2006-2010. The analysis shows 15.8% mortality in the StemEx group and 24.5% in the control group (p=0.034). A complete analysis of the data will be available in a few weeks.

Twenty-five bone marrow transplantation centers worldwide treating 101 patients with hematologic malignancies following myleoablative therapy who could not find a family related matched bone marrow donor participated in the study.

StemEx is a graft of an expanded population of stem/progenitor cells, derived from part of a single unit of umbilical cord blood and transplanted by IV administration along with the remaining, non-manipulated cells from the same unit. Cord blood has less matching requirements than bone marrow or peripheral blood transplants, providing the potential to increase the number of suitable transplant matches and to shorten the time it can take to find a match. However, there are a limited number of stem/progenitor cells in cord blood, enabling a quantity sufficient generally only for pediatric treatment. StemEx employs a technology that expands this small number of cord blood stem/progenitor cells, increasing their therapeutic capacity for transplantation in adolescents and adults.

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. Gamida Cell is currently seeking a strategic partner for the global commercialization of StemEx

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