Gamida Cell, a leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases, announced today that the company has reached agreements with the FDA and EMA regarding the Phase III study design outline of NiCord. The company 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 Q4/2015. NiCord is in development as an experimental treatment for various types of blood cancers including AML, ALL, MDS and 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. It is intended to fill the crucial clinical need for a therapeutic 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 around 120 patients. It will compare the outcomes of patients transplanted with NiCord to those of patients transplanted with un-manipulated umbilical cord blood.