Omidubicel met the primary end point of time to neutrophil engraftment in patients with high-risk hematologic malignancies undergoing a bone marrow transplant.
Omidubicel met the primary end point of time to neutrophil engraftment in patients with high-risk hematologic malignancies undergoing a bone marrow transplant, according to topline findings from a phase 3 trial announced by Gamida Cell Ltd., the developer of the investigational advanced cell therapy.
In the study, the median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group of patients who received a standard umbilical cord blood transplant (P <.001). Gamida Cell expects to submit a biologics license application to the FDA by the end of the year.
“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,” principal investigator Mitchell Horwitz, MD, a professor of medicine at the Duke Cancer Institute, stated in a press release. “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.”
The intent-to-treat analysis of the multicenter, international, open-label phase 3 study (NCT02730299) included 125 patients aged 12 to 65 years with acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome, or lymphoma. Patients had high-risk disease and were undergoing a bone marrow transplant. The study was conducted at more than 50 clinical locations in the United States, Latin America, Europe, and Asia. Baseline characteristics and demographics were well balanced between the 2 study arms.
Patients were randomized to omidubicel or standard umbilical cord blood transplant. The primary end point was time to neutrophil engraftment. In the omidubicel arm the median time to neutrophil engraftment was 12 days (95% CI, 10-15) compared to 22 days (95% CI, 19-25) in the control arm. Further, among patients who received a transplant per study protocol, successful neutrophil engraftment occurred in 96% of patients treated with omidubicel compared with 88% of patients in the control arm.
“Omidubicel is the first bone marrow transplant product to receive breakthrough therapy designation from the US 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 1 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,” Julian Adams, PhD, chief executive officer of Gamida Cell, stated in the press release. “We deeply appreciate the participation of patients in this trial and the support we have received from investigators and their teams.”
The phase 3 results showed similar efficacy as previously reported data from a phase 1/2 study. In the earlier study, 36 patients with advanced hematologic malignancies were treated with omidubicel and had faster neutrophil engraftment versus a 146-patient concurrent cohort of individuals treated with standard umbilical cord blood per the Center for International Blood and Bone Marrow Transplant Research (CIBMTR). The median time to engraftment was 11.5 days (95% CI, 9-14) versus 21 days (95% CI, 20-23), respectively (P <.001).
“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,” Ronit Simantov, MD, chief medical officer of Gamida Cell, stated in the press release.Gamida Cell Announces Positive Topline Data from Phase 3 Clinical Study of Omidubicel in Patients with High-Risk Hematologic Malignancies. Published May 12, 2020. https://bit.ly/35WbJXO. Accessed May 12, 2020.
Gamida Cell Announces Positive Topline Data from Phase 3 Clinical Study of Omidubicel in Patients with High-Risk Hematologic Malignancies. Published May 12, 2020. https://bit.ly/35WbJXO. Accessed May 12, 2020.