Omidubicel Improves Survival in Hematologic Malignancies With HSCT

The umbilical cord blood (UCB) cell therapy omidubicel (NiCord; Gamida Cell) improved survival in patients with hematological malignancies receiving allogeneic hematopoietic stem cell transplantation (HSCT).¹

These data, from a phase 3 trial (NCT02730299) were presented at the 10th Annual Meeting of the Society of Hematologic Oncology (SOHO), taking place September 28- October 1 in Houston, Texas, by Chenyu Lin, MD, hematology/oncology fellow, malignant hematologist, Duke University.¹

“These data reinforce our commitment to advance transformational cell therapy research and underscore the potential of our NAM 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, Gamida Cell, said in a statement.² “As we approach the PDUFA 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.”

Lin and colleagues analyzed data from 105 participants across 2 academic transplant centers worldwide from the phase 3 study either as a standalone unit (n = 92) or together with an unmanipulated UCB (n = 24).¹ Eleven participants were excluded from an initial pool of 116. Most patients (n = 97; 93%) engrafted fully, 2 (2%) had mixed chimerism between omidubicel and UCB, 5 (5%) had primary graft failure, and 1 patient was not evaluable. All patients received myeloablative conditioning regimens and standard graft versus host disease (GVHD) prophylaxis with a calcineurin inhibitor and mycophenolate mofetil. There were no late cases of secondary graft failure reported after the 5 patients reported in the primary analysis.

READ MORE: Omidubicel BLA Accepted With Priority Review in Hematological Malignancies

Median follow-up was 22 months (range, 0.3-122.5) overall and 35.7 months (range, 11.7-122.5) among survivors.Treated participants had an overall survival of 63% (95% CI, 53%-73%) and disease-free survival of 56% (95% CI, 47%-67%). Quality of life assessments revealed that overall well-being scores were higher in these patients when compared to standard of care.

In terms of safety, most chronic GVHD cases were mild (54%), with 33% moderate and 13% severe cases. No deaths were primarily attributed to chronic GVHD. One participant developed donor-derived MDS which is consistent with previous reports of donor-derived myeloid neoplasms after cord blood transplants (0.6–2%). A case of donor-derived AML was also observed in the phase 3 control group.

Omidubicel has demonstrated fast engraftment, few infections, and short durations of hospitalization in previous trials. It is an ex vivo expanded stem cell therapy derived from UCB.

“In addition to the known early benefits, omidubicel demonstrated long-term graft durability, with preserved trilineage hematopoiesis and immune competence at up to 10 years of follow-up,” Lin and colleagues wrote in the poster.¹

REFERENCES
1. 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. https://www.businesswire.com/news/home/20220929005316/en/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
2. Lin C, Schwarzbach A, Montesinos P, et al. Multicenter long-term follow-up of allogeneic hematopoietic cell transplantation with omidubicel: A pooled analysis of five prospective clinical trials. Presented at: 10th Annual Meeting of the SOHO, September 28- October 1, Houston, Texas.