JERUSALEM--(BUSINESS WIRE)--Gamida Cell announced today that while the current single arm Phase II/III study, incorporating a historical cohort as the study control, provides evidence of activity of StemEx®, the FDA has advised Gamida Cell to conduct another trial – a Phase III, randomized, controlled clinical study.
StemEx® is in development as an alternative transplantation treatment for patients with high risk leukemia and lymphoma, who could not find a family related matched bone marrow donor. The data and analysis of the Phase II/III, multi-center, multi-national, historical cohort-controlled study of StemEx® were announced in a press release on April 2013.
“The use of cord blood for transplantation is limited by the small number of stem/progenitor cells, resulting in a delay in engraftment and increased morbidity and mortality during the first 100 days post transplantation as compared with bone marrow as a source of stem cells for transplantation. Of note, StemEx® study data showed that the increased dose of CD34+ stem and progenitor cells provided by StemEx® was directly associated with a shorter time to neutrophil and platelet engraftment, which in turn, was associated with improved survival at 100 days post transplantation. Therefore, the clinical outcomes from the Phase II/III study clearly demonstrated a proof of concept of StemEx®’s mode of action,” said Tony Peled, V.P. of research and development and chief scientific officer.
“Indeed we had expectations that the completed Phase II/III study will support a BLA submission and allow us to launch the product in the market in a couple of years. Nevertheless, we know from the Phase II/III study data as well as the FDA’s recommendation, that the StemEx® study provides evidence of activity that warrants an additional clinical trial for registration,” said Gamida Cell CEO Dr. Yael Margolin. “We will now regroup and determine how to proceed with our clinical development program of StemEx®. We will also continue to advance the clinical development programs of NiCord® for hematological malignancies as well as for sickle cell disease and thalassemia.”
About StemEx®
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 infusion 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 treated patients 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.
About Gamida Cell
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, solid tumors, non-malignant hematological diseases such as hemoglobinopathies, neutropenia and acute radiation syndrome, autoimmune diseases and metabolic diseases as well as conditions that can be helped by regenerative medicine. Gamida Cell’s therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood, bone marrow and peripheral blood, which are expanded in culture. Gamida Cell’s current shareholders include: Elbit Imaging (NASDAQ: EMITF), Clal Biotechnology Industries (TASE: CBI), Israel Healthcare Venture, Teva Pharmaceutical Industries, Amgen, Denali Ventures and Auriga Ventures. For more information please visit www.gamida-cell.com.
