Gamida Cell Announces the Successful Transplantation of the First Patient in the Company’s Phase I/II Study of NiCord® …

JERUSALEM--(BUSINESS WIRE)--

Gamida Cell announced today that the first patient has been successfully transplanted in the companys 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 Cells 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 ofmedicine 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 Cells ongoing ability to maintain its leadership role in the cell therapy industry and is confident and steadfast in its support of the companys important and extremely valuable work, said chairman of the board of Gamida Cell Ruben Krupik.

About Gamida Cell Gamida Cell is a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine. The companys pipeline of stem cell therapy products are in development to treat a wide range of conditions including blood cancers, solid tumors, non-malignant hematological diseases such as hemoglobinopathies, neutropenia and acute radiation syndrome, autoimmune diseases and genetic metabolic diseases as well as conditions that can be helped by regenerative medicine. Gamida Cells therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood, bone marrow and peripheral blood, which are expanded in culture. Gamida Cells current shareholders include: Elbit Imaging (NASDAQ: EMITF), Clal Biotechnology Industries (TASE: CBI), Israel Healthcare Venture, Teva Pharmaceutical Industries, Amgen, Denali Ventures and Auriga Ventures. For more information please visit http://www.gamida-cell.com.

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Gamida Cell Announces the Successful Transplantation of the First Patient in the Company’s Phase I/II Study of NiCord® ...

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