Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU.
In May 2020, we reported topline results from an international, multi-center, randomized Phase 3 clinical study in 125 patients demonstrating that omidubicel was generally well tolerated and the median time to neutrophil engraftment was significantly shorter for patients who were randomized to omidubicel than those in the comparator group. In October 2020, we reported that the study met all three of its secondary endpoints related to platelet engraftment, infections, and hospitalizations, which are key measures in bone marrow transplant. These secondary endpoints were the proportion of patients who achieved platelet engraftment by day 42, the proportion of patients with grade 2 or grade 3 bacterial or invasive fungal infections in the first 100 days following transplant, and the number of days alive and out of the hospital in the first 100 days following transplant. All three secondary endpoints demonstrated statistical significance in an intent-to-treat analysis.
We anticipate reporting the full data set in a peer-reviewed setting in the first half of 2021 and expect to initiate the submission of a biologics license application (BLA) filing, on a rolling basis, in the fourth quarter of 2020.
For more information about the details of the secondary endpoints, please see here.