Gamida Cell, a cell therapy company based in Jerusalem, Israel, has reached agreements with the US Food and Drug Administration (USFDA) and the European Medicines Agency (EMA) with regards to a Phase III study design outline for testing their NiCord product. NiCord is a blood cancer treatment derived from a single umbilical cord blood until expanded in culture and enriched with stem cells by means of the company’s proprietary NAM technology.
Gamida Cell is moving forward now with plans to commence an international, multi-center, Phase III study of NiCord in 2016. Phase I/II data of 15 patients are expected in the fourth quarter of 2015. NiCord is in development as an experimental treatment for various types of blood cancers including Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Myelodysplastic Syndrome (MDS), and Chronic Myelogenous Leukemia (CML).
NiCord® is derived from a single cord blood unit which has been expanded in culture and enriched with stem cells using Gamida Cell’s proprietary NAM technology. NAM technology proceeds from the observation that nicotinamide, a form of vitamin B3, inhibits the loss of functionality that usually occurs during the culture process of umbilical cord blood stem cells, when added to the culture medium. Pre-clinical studies have also shown that the expanded cell grafts manufactured using NAM technology demonstrate improved functionality following infusion in a living animal. These stem cells show improved movement, home to the bone marrow, and show higher rates of engraftment, or durable retention in the bone marrow. Based on these results, Gamida Cell is currently testing in clinical trials (in patients) cells expanded in culture with the NAM platform to determine their safety and effectiveness as a treatment for blood cancers, sickle-cell anemia and thalassemia. NiCord is intended to fill the crucial clinical need for a treatment for the vast majority of blood cancer patients indicated for bone marrow transplantation, with insufficient treatment options. This segment has a multi-billion dollar market potential.
“The FDA and EMA feedback is a major regulatory milestone for NiCord. NiCord is a life-saving therapy intended to provide a successful treatment for the large number of blood cancer patients who do not have a family related matched donor. Gamida Cell is dedicated to changing the paradigm in transplantation by bringing this therapy to market as soon as possible,” said Dr. Yael Margolin, president and CEO of Gamida Cell.
“The positive regulatory feedback confirms that Gamida Cell’s NiCord program is on a clear path to approval both in the U.S. and EU. We look forward to continuing the development of this very important product in cooperation with sites of excellence in cord blood transplantation worldwide,” said Dr. David Snyder, V.P. of Clinical Development and Regulatory Affairs at Gamida Cell.
The Phase III study will be a randomized, controlled study of approximately 120 patients. It will compare the outcomes of patients transplanted with NiCord to those of patients transplanted with un-manipulated umbilical cord blood.