The Gamida Cell-Teva Joint Venture (JV) announced that the Data Monitoring Committee (DMC) has independently reviewed data collected from the first 70 patients enrolled in the pivotal registration, phase III clinical trial of StemEx and has recommended that the JV continue to enroll patients in the StemEx study. This marks the last protocol mandated DMC review of the StemEx study.
StemEx is being evaluated as a therapeutic treatment for adolescents and adults with blood cancers such as leukaemia and lymphoma, who cannot find a family related matched bone marrow donor.
The DMC’s primary charge is to ensure that the study is conducted in an ethical manner that does not expose participants to undue risk. In addition, the Committee monitors the quality and overall conduct of the study and assesses whether to recommend that the sponsor modify or terminate an ongoing study, or whether to request further information for review. The DMC for the phase III StemEx study was comprised of an independent group of medical and scientific experts who reviewed safety and efficacy data from participating sites to assess treatment benefit (or harm) to study participants. The DMC had no major safety concerns and recommended that accrual on the study continue.
Dr Yael Margolin, president and chief executive officer of Gamida Cell said, “The DMC’s recommendation to continue the StemEx study represents a very significant milestone. We are on track to complete enrollment of patients in a few months and to submit the regulatory files in the US and in Europe. The DMC has confirmed our resolve and confidence in the potential of StemEx to provide a good solution for patients with leukaemia or lymphoma who need a bone marrow transplant but who do not have a family related matched bone marrow donor.”
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 unit of non-manipulated cells. The StemEx study is currently enrolling adolescents and adults with high-risk hematologic malignancies, at clinical sites in the US, Italy, Spain and Israel.
The Gamida Cell-Teva Joint Venture expects to complete the enrollment of 100 patients in the StemEx study in Q4 2011 and plans to submit a Biologic License Application to the FDA in 2012 as well as a Marketing Authorization Application to the EMA thereafter. A first market launch is planned for 2013.
Gamida Cell is a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine. The company’s pipeline of stem cell therapy products are in development to treat a wide range of conditions including blood cancers such as leukaemia and lymphoma, solid tumours, autoimmune diseases, metabolic diseases and non-malignant haematological diseases.