Gamida Cell: StemEx® overview – clinical trials and commercial partners

Yael Margolin, President & CEO of Gamida Cell Ltd., provided this overview of StemEx®. Gamida Cell is currently seeking a strategic partner to commercialize the product.

Each year, approximately 60,000 patients with leukemia or lymphoma can potentially be cured by a bone marrow transplantation. Of these, only approximately 25,000 are transplanted, primarily because they could not find a fully matched bone marrow donor in time. Cord blood transplantation (CBT) does not require full tissue matching and is considered a good alternative for transplantation. However, CBT is limited by a low number of TNC and CD34+ cells, influencing the incidence and rate of hematopoietic recovery and risk of early transplant-related mortality. Ex-vivo expansion is a strategy employed to increase the number of progenitor cells and improve clinical outcomes.

StemEx is a graft of stem/progenitor cells isolated and expanded from a portion of a single unit of umbilical cord blood (CBU) and transplanted in combination with non-expanded cells from the same unit. The portion of umbilical stem cells is expanded using Gamida Cell's proprietary copper chelator technology.

StemEx is poised to answer the dire unmet clinical need in the field of bone marrow transplantation by supplying alternative cell grafts to 50,000 patients with hematological malignancies, who are indicated for transplantation but do not have a matched family related bone marrow donor. The market potential of StemEx is estimated at more than one billion USD.

StemEx is now being studied as a treatment for hematological malignancies in an international, Phase III, pivotal registration clinical trial. This is an open study with a historical control cohort: the design is approved by the FDA under SPA. The pivotal study has completed recruitment. Safety and efficacy clinical outcomes of the study will be available in Q4/2012. Gamida Cell

More than 100 StemEx batches were manufactured in 3 centralized GMP facilities. Data available for the first 88 batches shows a median fold expansion of TNC, CD34+ cells and CFU over culture input were 399 (range 52-764), 75 (6-280) and 107 (43-662), respectively. Since only a portion of the CBU isexpanded, patients were infused with a median 8.4 fold increase (0.8-90.3) in the number of CD34+ cells infused over the number that would be infused from the entire CBU without expansion. The CFU potential of culture seeded CD133+ cells measured at day-0 of production indicates the expansion potential of the cryopreserved CB cells. In all six batches which failed to expand, day-0 CFU was low, while day-0 CFU of all successfully expanded batches was within specification ranges. This information, available before patient myeloablation, strengthens the clinical applicability of StemEx.

Gamida Cell has developed a robust logistical infrastructure to support the clinical implementation of StemEx. All StemEx batches were successfully delivered and infused to the patients.

StemEx is developed in a 50/50 Joint Venture between Gamida Cell and Teva Pharmaceutical Industries. The JV is now seeking a strategic partner to support the global commercialization of StemEx.

Dr. Margolin will be presenting at this year's Stem Cells USA & Regenerative Medicine Congress, on the topic, ‘StemEx pivotal trial completed – preparing for commercialization.' Register now to hear her share her insights on the stem cells sector.

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