Gamida Cell, a leader in adult stem cell expansion technologies and products, announced today that it has completed enrollment for a Phase I/II clinical trial of NiCord, the company's second pipeline product.
NiCord is in development as an experimental treatment for a series of indications that potentially could be cured with a bone marrow transplantation including hematological malignancies (blood cancer), sickle cell disease, thalassemia, severe autoimmune diseases and metabolic diseases. The clinical trial announced today (clinicaltrials.gov identifier NCT01221857) is studying NiCord as an alternative investigational treatment for hematological malignancies (HM). A combined total of 11 patients were transplanted at Duke University Medical Center and at Loyola University Medical Center. Dr. Mitchell E. Horwitz of Duke University Medical Center is the principal investigator. Final results of the Phase I/II study are expected within 6 months. The company is also actively enrolling for a Phase I/II study of NiCord as an experimental treatment for sickle cell, a genetic blood disease (clinicaltrials.gov identifier NCT01590628).
NiCord is an expanded cell graft derived from an entire unit of umbilical cord blood enriched with stem cells. NiCord was developed based on Gamida Cell's proprietary NAM technology. As the Phase I/II trial for HM is a first in man safety and efficacy study, for this stage, NiCord was transplanted with a second un-manipulated cord blood unit in a double cord blood configuration.
Dr. Tony Peled, chief scientific officer and vice president of research & development at Gamida Cell, said, "Pre-clinical data demonstrated the uniqueness of NAM technology in not only decreasing the aging process but also preserving the characteristics and functions of ex vivo expanded stem cells (Experimental Hematology 2012;40:342-355). Of significance, the Phase I/II clinical trial data have already shown that many of the patients in the study engrafted with the expanded cells of NiCord rather than with the second un-manipulated unit. This is the first time, in a situation where two units are transplanted, that the cultured stem cells demonstrated prompt and durable long-term engraftment (over one year) in the clinic. We look forward to sharing the complete results of this study in the coming months."
Dr. Yael Margolin, CEO of Gamida Cell, said, "The clinical progress of the company's second pipeline product reaffirms Gamida Cell's leadership role in the stem cell industry and the company's expertise in bone marrow transplantation. In the near future we plan to not only release the NiCord Phase I/II data but also the long awaited complete results of the Phase III study of StemEx, also for hematological malignancies, but clearly further ahead in development. The company remains on course with the development of StemEx and is considering strategic partners to develop its expanding pipeline of products and to bring StemEx to market."