Gamida Cell Ltd., a leading cellular and immune therapeutics company, has announced the publication of data from a multi-center Phase 1/2 clinical study evaluating the safety and efficacy of NiCord® as a stand-alone, hematopoietic stem cell (bone marrow) transplant in the Journal of Clinical Oncology.
Founded in 1998, headquartered in Jerusalem, Israel, Gamida Cell is a clinical stage biopharmaceutical company leveraging its proprietary technology to develop cell therapies that are designed to cure cancer and rare, serious hematologic diseases. The company is using its nicotinamide-, or NAM-, based cell expansion technology to develop a pipeline of products designed to address the limitations of cell therapies.
Despite the curative potential of bone marrow transplants, it is estimated that more than 40 percent of eligible patients do not receive one for various reasons, including finding a matched donor.
While umbilical cord blood provides a source of stem cells for patients who do not have a matched related donor, it provides a smaller number of stem cells, which can delay engraftment and put patients at a greater risk for prolonged hospitalizations and life-threatening infections.
NiCord® is designed to address these limitations by offering a therapeutic dose of expanded cells while preserving the functional characteristics of stem cells.
NiCord®, the company’s lead clinical program (phase III), is under development as a universal bone marrow transplant solution for patients with high-risk hematologic malignancies. It has been granted breakthrough status by the U.S. Food and Drug Administration, making it the first bone marrow transplant alternative to receive this designation. It has also received U.S. and EU orphan drug designation.
Results from the published Phase 1/2 study showed that patients transplanted with NiCord® had rapid and durable engraftment of neutrophils and platelets, as well as prompt immune reconstitution. The median time to neutrophil recovery was shortened by nearly 50 percent for patients who received NiCord® compared to a retrospective cohort of patients who received standard umbilical cord blood. NiCord® also demonstrated an acceptable safety profile for patients undergoing bone marrow transplant.
“In this study, patients who received NiCord had a clinically meaningful reduction in their time to neutrophil and platelet recovery compared to a retrospective cohort of patients who received a standard umbilical cord blood transplant. The neutrophil recovery observed with NiCord also resulted in fewer days spent in the hospital compared to the comparator cohort.” … “These data suggest a potential step toward making stem cell transplantation safer and more accessible to patients with lethal blood cancers.” – Mitchell Horwitz, M.D.,principal investigator and professor of medicine at the Duke Cancer Institute.