Clinical Trials

* On June 6, 2016, Gamida Cell announced the presentation of results from the Company’s international, multi-center, Phase 1/2 study of NiCord at the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting. NiCord is in development as a novel transplant modality for high risk hematological malignancies (blood cancers). These results demonstrated statistically significant improvements across key measures including time to engraftment of neutrophils and platelets. The data were highlighted in an oral presentation, entitled NiCord®: results of phase 1/2 trials set the stage for a definitive phase 3 clinical trial, by Mitchell Horwitz M.D. of Duke Cancer Institute, and co-chair of the NiCord study, along with Professor Guillermo Sanz of Hospital Universitario La Fe in Valencia, Spain. The data comprised the results of 16 patients, ages 12-65, with high-risk hematological malignancies, who were transplanted with NiCord as a standalone graft following myeloablative therapy. The outcomes of these 16 patients were evaluated at one year post-transplantation. In this analysis, the study outcomes were compared to a retrospective cohort, provided by the CIBMTR registry, of 125 similar patients transplanted with cord blood. Highlights of the Phase 1/2 Study Results of NiCord demonstrate:
· A 101-fold increase in the number of CD34+ (stem and progenitor) cells in NiCord compared to unmanipulated cord blood.
· Faster neutrophil engraftment for NiCord recipients (p<0.0001) at a median of 10 days post-transplantation compared to 21 days in registry controls.
· By 16 days post-transplant (the usual median time to engraftment after standard peripheral blood transplantation), 75% of NiCord recipients had achieved neutrophil engraftment, compared to only 18% in registry controls (p<0.0001).
· Faster platelet engraftment for patients engrafting with NiCord (p<0.001) at a median of 32 days, compared to 46 days in registry controls.
· At 42 days post-transplant, 56% of NiCord recipients achieved platelet engraftment, compared to 27% in registry controls (p=0.015).
· Transplant related mortality at one year was 19% compared to 39% in registry controls (p=0.12).
The findings announced are consistent with data presented earlier this year at the 42nd annual meeting of the EBMT where patients transplanted with NiCord demonstrated a significantly lower frequency of grade 2-3 bacterial infections and a shorter hospital stay.

* On September 9, 2013, Gamida Cell has announced 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. 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.
In this study, 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.

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.”