Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS

Successful blood and marrow transplantation (BMT) requires the infusion of a sufficient

number of hematopoietic stem/progenitor cells (HSPCs), capable of both homing to the bone

marrow and regenerating a full array of hematopoietic cell lineages with early and late

repopulating ability in a timely fashion.

A major drawback of Umbilical Cord Blood (UCB) is the low stem cell dose available for

transplantation, compared to mobilized peripheral blood (PB) or bone marrow. This low

stem cell dose can compromise the chances of engraftment and contributes to delayed

kinetics of neutrophil and platelet recovery, as well as other transplant outcomes.

The aim of ex vivo expansion of cord blood is to provide a graft with sufficient numbers

of cells that have rapid and robust in vivo neutrophil and platelet producing potential

to enable successful transplantation.

NiCord® is a stem/progenitor cell-based product composed of ex vivo expanded allogeneic

cells from one entire unit of UCB. NiCord® utilizes the small molecule nicotinamide

(NAM), as an epigenetic approach to inhibit differentiation and to increase the

migration, bone marrow (BM) homing and engraftment efficiency of Hematopoietic Progenitor

Cells (HPC) expanded in ex vivo cultures. The chief aim of the study is to compare the

safety and efficacy of NiCord® single ex-vivo expanded cord blood unit transplantation to

unmanipulated cord blood unit transplantation in patients with hematological malignancies

following conditioning therapy.