Phase III study of omidubicel in patients with high-risk hematologic malignancies meets all 3 secondary endpoints

On October 6, 2020, positive topline secondary endpoint data were released from the randomized, multicenter, phase III omidubicel study (NCT02730299) in 125 patients with high-risk hematological malignancies. In May, the AML Hub reported on the results regarding the primary endpoint of this international study, which showed that treatment with omidubicel significantly reduced the median time to neutrophil engraftment by 10 days compared to standard umbilical cord blood transplant (p < 0.001). It was generally well tolerated, with a higher percentage of patients achieving successful neutrophil engraftment.^1,2

In the new announcement, all three secondary endpoints have shown a statistically significant improvement for patients treated with omidubicel compared to the comparator group.¹ These secondary endpoints were:

• The proportion of patients who achieved platelet engraftment by Day 42,
• The proportion of patients with Grade 2/3 bacterial or invasive fungal infections in the first 100 days posttransplant, and
• The number of days alive and out of hospital in the first 100 days posttransplant.

The full data sets are due to be presented at a medical meeting by the end of the year, as well as submitted to the U.S. Food and Drug Administration (FDA) for a biologics license. The positive primary and secondary endpoints of this study show omidubicel to be a promising treatment for patients who do not have a matched related donor of suitable age.