Omidubicel Shows Clinical Benefit at 3 Years for Individuals With Hematologic Malignancies

New long term follow-up data and health-related quality of life scores of individuals with omidubicel from Gamida Cell Ltd were presented at the 10th Annual Meeting of the Society of Hematologic Oncology, held in Houston, Texas.

“These data reinforce our commitment to advance transformational cell therapy research and underscore the potential of our [nicotinamide] technology platform. Our lead stem cell therapy candidate, omidubicel, addresses the unmet need for patients with hematologic malignancies, demonstrated by the robust and growing body of encouraging clinical evidence, including the long-term follow up data and quality of life improvement,” Ronit Simantov, MD, chief medical officer of Gamida Cell, said in a statement.

“As we approach the [Prescription Drug User Fee Amendment] date of January 30, 2023, and upon potential FDA approval, we are prepared to execute our plan that ensures access to those patients who can benefit from omidubicel as quickly as possible,” she said.

Investigators observed that the clinical benefit of omidubicel at 3 years, across the patient population, was durable and long-term.

In the study, 105 individuals were transplanted with omidubicel between 2006 and 2020, with a median follow-up of 22 months. The results showed disease-free and overall survival of 56% and 63%, respectively, at 3 years.

Additionally, the data show durable long-term hematopoiesis and immune competence.

Further, the overall well-being health-related quality-of-life scores also showed a clinical benefit for patients who were treated with omidubicel compared with the standard of care.

The functional and physical well-being and other scores from the survey were more favorable for individuals who were treated with omidubicel, investigators said.

This analysis was based on 108 individuals who completed and validated health-related quality of life surveys on the screening day, as well as on days 42, 100, 180, and 365, post-transplant.

These data show clinically meaningful and sustained improvement in functional, physical, and overall well-being compared with umbilical cord blood transplantation.

Omidubicel is an advanced cell therapy candidate that was developed as a potential life-saving allogenic hematopoietic stem cell transplant for individuals with blood cancer.

The drug has shown a statistically significant reduction in time to neutrophil engraftment by comparison with standard umbilical cord blood in a randomized phase 3 study (NCT0273029) for individuals with hematologic malignancies undergoing allogeneic bone marrow transplant.

The study’s findings showed a reduction in time-to-platelet engraftment, reduction in infections, and fewer days of hospitalization.

Omidubicel is the first stem cell transplant donor source to receive breakthrough therapy designation from the FDA and orphan drug designation in the European Union and the United States.

Gamida Cell’s NAM technology was designed to enhance the number and functionality of targeted cells, which has helped the company create innovative approaches that differ from existing therapies, according to the statement.

Reference

Long-term data from omidubicel phase 3 trial demonstrates overall survival and sustainable durable outcomes for patients with blood cancers at the Society of Hematologic Oncology Meeting. News release. Gamida Cell. September 29, 2022. Accessed September 29, 2022. https://investors.gamida-cell.com/news-events/press-releases/news-release-details/long-term-data-omidubicel-phase-3-trial