Gamida Cell Ltd. (NASDAQ: GMDA) today announced positive topline results from its Phase 3 clinical study evaluating the safety and efficacy of omidubicel, an investigational advanced cell therapy in development as a potential life-saving treatment option for patients in need of bone marrow transplant. The median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p<0.001). Neutrophil engraftment is a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells, and rapid neutrophil engraftment has been associated with fewer infections and shorter hospitalizations.1
Despite the curative potential of bone marrow transplant, it is estimated that more than 40 percent of eligible patients in the United States do not receive a transplant for various reasons, including the lack of a matched donor.2 Even for patients who do receive a transplant, treatment is not always effective and can lead to serious complications that can dramatically affect their quality of life.3 Omidubicel is intended to address the current limitations of bone marrow transplant by providing a therapeutic dose of stem cells while preserving the cells' functional therapeutic characteristics.
"I'm very encouraged by the data from this rigorous, Phase 3 study that was conducted at more than 50 centers around the world, as there is a significant need for new bone marrow transplant graft modalities," said Mitchell Horwitz, M.D., principal investigator and professor of medicine at the Duke Cancer Institute. "These results have the potential to substantially move the field forward and represent an important step toward making stem cell transplantation more accessible and more successful for patients with lethal blood cancers. Shortening the time to engraftment is clinically meaningful, as it can reduce a patient's time in the hospital and decrease likelihood of infection."
"Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has the potential to be the first FDA-approved bone marrow transplant graft. We are very pleased with the results of the Phase 3 data reported today, which move us one step closer toward bringing potentially curative therapies to patients. We expect to begin to submit our biologics license application for omidubicel to the FDA on a rolling basis in the fourth quarter of this year," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We deeply appreciate the participation of patients in this trial and the support we have received from investigators and their teams."
Topline Phase 3 Data
The international, multi-center, randomized Phase 3 study (NCT02730299) was designed to evaluate the safety and efficacy of omidubicel in patients with high-risk hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. The primary endpoint was time to neutrophil engraftment. The intent-to-treat analysis included 125 patients aged 12–65 years with acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma and was conducted at more than 50 clinical centers in the United States, Latin America, Europe and Asia. The demographics and baseline characteristics were well-balanced across the two study groups.
The study achieved its primary endpoint (p<0.001). In the intent-to-treat analysis, the median time to neutrophil engraftment was significantly shorter for patients who received omidubicel (12 days; 95% CI: 10-15 days) compared to the comparator group (22 days; 95% CI: 19-25 days). Omidubicel was generally well tolerated. Among patients who were transplanted per protocol, 96 percent of patients who received omidubicel achieved successful neutrophil engraftment, compared to 88 percent of patients in the comparator group.
"We are pleased with the outcome of this global, well-designed study in patients with life-threatening blood cancers who were in need of a bone marrow transplant and did not have an available matched donor," said Ronit Simantov, M.D., chief medical officer of Gamida Cell. "Importantly, these data confirmed the results from our earlier Phase 1/2 clinical study and demonstrated that patients who received omidubicel had more rapid recovery of neutrophils, which are key infection-fighting white blood cells."
The data reported today are consistent with results from a multi-center, Phase 1/2 study in 36 patients with advanced hematologic malignancies, which showed that patients treated with omidubicel demonstrated more rapid neutrophil engraftment compared to a concurrent cohort of 146 patients treated with standard umbilical cord blood as reported by the Center for International Blood and Bone Marrow Transplant Research.4 In the Phase 1/2 study, the median time to engraftment was 11.5 days (95% CI: 9-14 days) for omidubicel recipients compared to 21 days (95% CI: 20-23 days) for the CIBMTR cohort (p<0.001).
Gamida Cell expects to report full efficacy and safety results at a medical conference later this year.