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Phase III study of omidubicel in patients with high-risk hematologic malignancies meets primary endpoint

May 15, 2020

Positive results from a phase III study of omidubicel, a cell therapy product of expanded umbilical cord blood (UCB) cells, ( NCT02730299 ) have been announced. 1The study investigated the safety and efficacy of omidubicel, compared to standard UCB transplant, in patients with high-risk hematological malignancies without a suitable matched donor. 2

  Key features 3,4

Omidubicel

  • An advanced cell therapy product consisting of nicotinamide (NAM)-expanded stem cells and differentiated immune cells from UCB
    • NAM inhibits differentiation and enhances the functionality of cultured hematopoietic stem and progenitor cells, which substantially increases bone marrow homing and engraftment potential
  • Can be cryopreserved and transplanted after myeloablative conditioning as a standalone hematopoietic stem cell graft
  • The first bone marrow transplant product to receive the United States Food and Drug Administration (FDA) breakthrough therapy designation and has also received orphan drug designation in the United States and European Union.
  • Proven in a phase I/II study, published in the Journal of Clinical Oncology, to induce rapid and durable engraftment while being well tolerated

  Study design 2

  Open-label, controlled, multicenter, international, randomized, phase III trial comparing stem cell transplantation with omidubicel vs standard UCB in patients with high-risk hematologic malignancies

  • Started in November 2016 and completed in December 2019
  • Included 125 patients aged 12–65 years with acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome, lymphoma, or acute leukemia 1
  • Primary endpoint: Time to neutrophil engraftment 

  Top-line results from the study demonstrated that treatment with omidubicel significantly reduced the median time to neutrophil engraftment by 10 days compared to standard UCB transplant (p < 0.001) and was generally well tolerated. Furthermore, a greater percentage of patients achieved successful neutrophil engraftment compared with patients in the comparator group (96% vs88%, respectively). 1 

  Full efficacy and safety results are expected to be presented later this year, and a biologics license application is due to be submitted to the FDA by the end of the year.

  1. gamidaCell. Gamida Cell announces positive topline data from phase 3 clinical study of omidubicel in patients with high-risk hematologic malignancies. https://investors.gamida-cell.com/node/7236. Published May 12, 2020. Accessed May 12, 2020.
  2. Clinicaltrials.gov. Stem cell transplantation with NiCord® (omidubicel) vs standard umbilical cord blood in patients with leukemia, lymphoma, and myelodysplastic syndrome (MDS). https://www.clinicaltrials.gov/ct2/show/study/NCT02730299Updated April 14, 2020. Accessed May 12, 2020.
  3. gamidaCell. Omidubicel: An investigational advanced cell therapy designed to enhance the life-saving benefits of bone marrow transplant. http://www.gamida-cell.com/wp-content/uploads/Omidubicel-Fact-Sheet.pdf. Accessed: May 12, 2020.
  4. Horwitz ME, Wease S, Blackwell B, et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivowith nicotinamide. J Clin Oncol. 2019;37(5):367-374. DOI: 1200/JCO.18.00053