Positive results support the initiation of a Phase III study of NiCord
Jerusalem, Israel, December 7, 2015 — Gamida Cell, a leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases, announced today positive top line results from the Phase I/II study of NiCord, in development as an experimental treatment for patients with blood cancers. NiCord is an ex vivo cell graft derived from a single cord blood unit, expanded and enriched with highly functional stem cells and immune modulatory cells, utilizing the Company’s proprietary NAM technology.
The currently available set of results are documented in a report submitted to the U.S. Food and Drug Administration (FDA) in preparation for an End-of-Phase II meeting, and the initiation of a Phase III, randomized controlled, registration study of NiCord in hematological malignancies. NiCord aims to provide a superior transplant solution for patients without a suitable matched donor for bone marrow transplantation.
“We are very enthusiastic about the positive results of this NiCord study, which we believe support the initiation of our Phase III registration study. Assuming regulatory support, we intend to commence this trial in mid-2016 to evaluate the safety and efficacy of NiCord compared to un-manipulated cord blood transplantation. Final Phase I/II data will be released in H2 2016, although the protocol remains open for additional sites qualification,” said Gamida Cell president and CEO Dr. Yael Margolin.
Data was reported on the clinical outcomes of 16 patients with high risk hematological malignancies after transplantation of NiCord as the sole graft source. NiCord demonstrated rapid short term engraftment and stable, long term hematopoiesis of multiple cellular lineages. Neutrophil engraftment was achieved for all patients, at a median 10 days post-transplant. Historical reports of un-manipulated cord blood transplants estimate that neutrophil engraftment usually takes between 23-27 days on average. The top line data reported shows that NiCord is comparable in terms of time to engraftment to peripheral blood transplantation, which is considered the gold standard. Results also indicated a good safety profile with potentially reduced morbidity and shorter hospitalization of patients.
Mitchell Horwitz MD, associate professor of medicine at Duke Cancer Institute and principal investigator of the NiCord study, said, “Though still early, the survival readout from this Phase I/II study of NiCord looks encouraging. We have observed rapid and durable engraftment, which is important because time to engraftment has been consistently reported to impact transplant outcomes and survival for these patients. We are eager to start the upcoming Phase III study for NiCord.”
About Gamida Cell
Gamida Cell is a world leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases. The company’s pipeline of products are in development to treat a wide range of conditions including cancer, genetic hematological diseases such as sickle cell disease and thalassemia, bone marrow failure syndromes such as aplastic anemia, genetic metabolic diseases and refractory autoimmune diseases. Gamida Cell’s current shareholders include: Novartis, Elbit Imaging, Clal Biotechnology Industries, Israel Healthcare Venture, Teva Pharmaceutical Industries, Denali Ventures and Auriga Ventures. For more information please visit www.gamida-cell.com.