Gamida Cell said today that the 1st patient has been treated in the Phase III registration trial of its NiCord cell graft for patients with blood cancer. The treatment is a graft derived from umbilical cord blood which has been expanded and enriched with stem and progenitor cells, according to the company.
The Jerusalem-based company’s cell graft was designed as an alternative to bone marrow transplantation for patients with blood cancer who do not have a fully matched donor.
“NiCord can provide a transplantation solution for patients in need. With clinical outcomes that potentially compare to a matched donor, NiCord is positioned to become the graft of choice for patients who do not have a high probability of rapidly finding a match,” president & CEO Yael Margolin said in prepared remarks. “This registration study is the last stage in the clinical development of NiCord towards regulatory approval and market introduction, with the goal of saving lives, curing disease and reducing treatment costs.”
The phase III trial, which is slated to enroll 120 patients, will evaluate the safety and efficacy of NiCord compared to un-manipulated cord blood in patients with hematological malignancies, Gamida Cell reported. The study’s primary endpoint is the time to neutrophil engraftment.
“We are very excited to start the Phase III study where we hope to reproduce the encouraging results demonstrated in the Pilot and Phase 1/2 data in a broader population,” principal investigator Dr. Guillermo Sanz said.
Data from the pilot and phase 1/2 studies demonstrated stronger clinical outcomes and improved pharmaco-economic measures for NiCord compared to un-manipulated umbilical cord blood, according to the company.
“There is a clinical need for a transplant option for the many patients who do not have a fully matched donor. Data from the Phase 1/2 study strongly support the further study of NiCord in this important Phase III trial,” principal investigator Dr. Mitchell Horwitz added.
In October last year, Gamida Cell won breakthrough therapy designation from the FDA for NiCord.