Gamida Cell announces results from NiCord Phase I/II study on hematological malignancies

Gamida Cell announced today the successful results of the Phase I/II study of its second pipeline product NiCord, umbilical cord derived stem cells expanded using the company's proprietary NAM technology. Study data was announced today during an oral presentation titled Nicord® Expanded Hematopoietic Progenitor Cells (HPC) Are Capable of Outcompeting the Unmanipulated (UM) Cord Blood Unit and of Prolonged Myeloid and Lymphoid Engraftment Following Myeloablative Dual Umbilical Cord Blood (UCB) Transplantation. The presentation was delivered at the prestigious 2013 BMT Tandem Meetings in Salt Lake City by Dr. Mitchell E. Horwitz, associate professor of medicine at Duke Medicine and a principal investigator of the NiCord study for hematological malignancies (HM).

Eleven patients, ages 21-61, with high-risk hematological malignancies received NiCord and an un-manipulated graft of umbilical cord blood. Eight patients engrafted with NiCord. The median time to neutrophil engraftment was 10.5 (7-18) days for those engrafting with NiCord. Two patients engrafted with the un-manipulated UCB and one patient experienced primary graft failure. There were no cases of Grade III/IV acute GvHD. No safety concerns surrounding the use of NiCord were raised. With a median follow-up of 8 months, the progression-free and overall survival are both 90%.

Dr. Horwitz said, "The results from this early study suggest that NiCord expanded hematopoietic progenitor cells (HPCs) reduce the time to hematopoietic recovery and are capable of long term (>22 months) neutrophil and T-cell engraftment. Co-transplantation of NiCord and an un-manipulated cord blood unit is feasible and safe. Further studies are planned to confirm that the NiCord product can be transplanted as a single expanded unit, without co-infusion of un-manipulated cells. This would mean that only one cord blood unit would be used for transplantation, rather than two units, as are used today in most adult umbilical cord blood transplantations."

"We view NiCord as a paradigm shift in cord blood transplantation as this is the first technology that allows for durable engraftment of ex vivo expanded hematopoietic stem cells. It is also significant that NiCord engrafted unusually fast with apparent robust immune reconstitution," said Dr. David Snyder, vice president of clinical development and regulatory affairs at Gamida Cell. "It is a sincere pleasure to work with the esteemed clinical team at Duke Medicine led by Dr. Horwitz and Dr. Joanne Kurtzberg. We look forward to continuing this very important research together."

Dr. Snyder continued, "Gamida Cell opted to begin clinical research of NiCord in hematological malignancies as cancer serves as a good bench mark. The early results of the HM study further supports the company's ambitions to continue its initiative to develop NiCord for other indications including sickle cell disease, thalassemia, severe autoimmune diseases and genetic metabolic diseases. In fact, a phase I/II study is currently enrolling pediatric patients with sickle cell disease at Duke Medicine and at the Steven and Alexandra Cohen Children's Medical Center of New York."