High-risk Leukemia and MDS Expanded Cell Therapy Trial Completes Phase 2 Enrollment


ExCellThera is in the process of the expanding the studies into new trial sites in the United States and Europe.

The phase 2 clinical trial (NCT04103879) for ExCellThera’s ECT-001, an investigational cell therapy intended to treat patients with high-risk leukemias and myelodysplastic syndromes (MDS), has completed patient enrollment.1

ECT-001 is a cord blood transplant comprised of stem cells from an optimized culture system that have been expanded with the use of hematopoietic stem cell self-renewal agonist UM171.1,2 It is able to be produced using small cord blood units, with UM171 intended to reverse the exhaustion from culture and gene modification typically seen in cell therapies. ECT-001 has previously been evaluated for the treatment of severe blood disorders in several clinical trials in the United States and Canada, with over 100 adults and children having received the therapy to date.

“We are pleased to announce the completion of enrollment in these important initial phase 2 studies in high-risk patients as it moves us closer to key meetings with, and submissions to, the US FDA, the European Medicines Agency (EMA) and Health Canada, which are important steps in bringing this novel, 1-time cell therapy to more patients as quickly as possible,” Guy Sauvageau, MD, PhD, co-chief executive officer and chief scientific officer, ExCellThera, said in a statement regarding the news.1 “Based on the first-in-class mechanism of action of UM171 in ex vivo cell culture, we believe the compound has the potential to represent a breakthrough in the expansion and rejuvenation of stem cells by offering patients a better and healthier graft. We are eager to see if the findings of these initial phase 2 studies validate that belief and provide support for the ongoing advancement of ECT-001 Cell Therapy as a potentially life-saving treatment for high-risk leukemias and MDS patients.”

Data from a phase 1/2 trial (NCT02668315) completed in 2018 was published in The Lancet Haematology in 2020.2 It showed that for the 22 patients who received a single UM171-expanded cord blood transplantation as of December 31, 2018, the median time to engraftment of 100 neutrophils per μL was 9·5 days (IQR 8-12), the median time to engraftment of 500 neutrophils per μL was 18 days (12·5-20·0), and the median platelet recovery time was 42 days (IQR 35-47). There was no graft failure reported, and the most common non-hematological adverse events included grade 3 febrile neutropenia (16 of 22 patients) and bacteremia (9 of 22 patients). A treatment-related diffuse alveolar hemorrhage lead to 1 patient death.

ECT-101 has been granted both orphan drug and regenerative medicine advanced therapy designations by the FDA and orphan medicinal product designation, advanced therapy medicinal product classification, and priority medicines designation by the European Medicines Agency.1 The phase 2 studies have already treated 50 patients with ECT-001, and ExCellThera is in the process of the expanding the studies into to new trial sites in the United States and Europe.

“The data ExCellThera is collecting from clinical trials to date demonstrate that ECT-001 Cell Therapy is safe, reduces the incidence of severe complications and improves outcomes for patients who require blood stem cell transplants,” Pierre Caudrelier, MD, chief medical officer, ExCellThera, added to the statement.1 “With the treatment of 50 patients in the initial Phase 2 trials in high-risk patients completed, we are looking forward to presenting the clinical data for these studies, together with the body of clinical data for ECT-001 Cell Therapy to the regulatory authorities, with a view to bringing this curative therapy to a much greater number of severely ill patients.”

Continued enrollment in a separate study (NCT04990323) of ECT-001 for children with high-risk myeloid malignancies is also taking place at Memorial Sloan Kettering Cancer Center. The company expects to report topline data from across the phase 1/2 trials of ECT-001 in the first half of next year. ExCellThera was also previously recruiting patients for a phase 1 clinical trial (NCT04594031) of ECT-001 for the treatment of sickle cell disease, however the study was withdrawn due to community preference being given to gene therapy approaches.

1. ExCellThera announces completion of Phase 2 clinical trials for UM171-expanded cell therapy in high-risk blood cancers. News release. ExCellThera Inc. November 15, 2022. https://excellthera.com/press_releases/excellthera-announces-completion-of-phase-2-clinical-trials-for-um171-expanded-cell-therapy-in-high-risk-blood-cancers/ 
2. Cohen S, Roy J, Lachance S, et al. Hematopoietic stem cell transplantation using single UM171-expanded cord blood: a single-arm, phase 1–2 safety and feasibility study. The Lancet Haematology. 2020;7(2):e134-e145. doi: 10.1016/S2352-3026(19)30202-9
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