Gamida Cell Announces the Successful Transplantation of the First Patient in The Company’s Phase I/II Study of NiCord® in a Single Cord Configuration

We are witnessing a paradigm shift in transplantation. The first patient was successfully transplanted with a stem cell graft entirely expanded in ex vivo cultures and without the support of a second unit of un-manipulated stem cells. Gamida Cell CEO Dr. Yael Margolin

Jerusalem, Israel, September 9, 2013 — Gamida Cell announced today that the first patient has been successfully transplanted in the company’s second Phase I/II study of NiCord®, as an alternative, experimental treatment for blood cancers. The transplant took place at Duke University Medical Center. Of great significance: This is the first study researching the outcome of a whole umbilical cord blood unit (UCBU) expanded in culture and transplanted in myeloablated patients without the support of un-manipulated stem cells derived from a second UCBU. The approach using NiCord®, as a single stem cell graft, has the potential to broaden accessibility, reduce toxicity and improve the clinical and economic outcomes of cord blood transplantation.

NiCord® is an expanded cell graft derived from an entire CBU and enriched with stem cells. NiCord® was developed based on Gamida Cell’s proprietary NAM technology.

Until NiCord®, even though expanded stem cells have been demonstrated to decrease the time to neutrophil engraftment, they disappeared shortly thereafter and the long-term engraftment was always provided by the second un-manipulated unit. Thus, the clinical benefit could be achieved only when two UCBUs were transplanted: one unit expanded in culture to shorten time to neutrophil engraftment and the second un-manipulated unit to provide the long and durable engraftment. Then came the Phase I/II study of NiCord® in a double cord configuration. In this study, for the first time, a robust short and long-term engraftment (over two years) was achieved entirely from the expanded stem cell graft. The reduced time to neutrophil and platelet engraftment and the durable, long-term engraftment provided by NiCord® also led to a shorter time of hospitalization, which also led to reduced costs for both the patient and the hospital. These outcomes provided the scientific basis for the second Phase I/II study of NiCord® in a single UCBU transplant modality.

Clinical sites will enroll up to 20 patients, ages 18-65, with hematological malignancies (blood cancers) following myeloablative therapy in the study: Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Stem and Progenitor Cells, in Adult Patients with high risk Hematological Malignancies. The principal investigator is Dr. Mitchell E. Horwitz, associate professor of medicine at Duke Medicine. Dr. Horwitz was also a principal investigator of the first Phase I/II study of NiCord® in a double cord configuration as an alternative, experimental treatment for blood cancers.

Dr. Horwitz said, “The protracted time to neutrophil and platelet engraftment and prolonged hospitalization has been a critical limitation of umbilical cord blood transplantation. The results of the first Phase I/II study were encouraging and suggest that NiCord has the potential to address both of these limitations. It also suggested that transplantation of a second, un-manipulated cord blood unit was not necessary, so we are interested in pursuing this ‘single cord’ Phase I/II study of NiCord® to determine whether those results bear out.”

“Gamida Cell is a world pioneer in cord blood transplantation, and with this new single cord approach we have indeed turned a corner and reached an intended milestone on the road to achieving a market paradigm change,” said Gamida Cell CEO Dr. Yael Margolin.

“We have witnessed in the clinic a small glimpse of the potential of NiCord® that we have extensively studied in the lab and animal models. The novel finding of the first NiCord® Phase I/II study is that cord blood-derived hematopoietic stem cells that are expanded in culture are capable of providing both, fast and robust long-term engraftment. This outstanding clinical outcome inspires us to continue developing our entire pipeline of therapeutic stem cell treatments,” said Tony Peled, VP of R&D and co-founder of Gamida Cell.

“The board is proud of Gamida Cell’s ongoing ability to maintain its leadership role in the cell therapy industry and is confident and steadfast in its support of the company’s important and extremely valuable work,” said chairman of the board of Gamida Cell Ruben Krupik.