UM171 Cell Therapy Demonstrates Improved Outcomes Compared to Cord & Matched Unrelated Donor Peripheral Blood Transplants in Real-World Setting

ExCellThera Inc. recently announced findings from a safety and efficacy retrospective analysis comparing outcomes of patients treated with ECT-001 (UM171-expanded) Cell Therapy in a Phase 1/2 trial to those treated with cord blood and matched unrelated donor peripheral blood stem cell transplants. In this real-world analysis using matched-controlled data from the CIBMTR (Center for International Blood and Marrow Transplant Research), ECT-001 Cell Therapy demonstrated improved outcomes compared to cord blood (CB) and matched unrelated donor (MUD) peripheral blood stem cell (PBSC) transplants, including overall survival (OS), progression-free survival (PFS), and graft-versus-host disease (GVHD) and relapse-free survival (GRFS). The findings were recently presented in an oral session during the 2022 American Society of Hematology (ASH) Annual Meeting (Abstract No. 665).

“This matched-controlled real-world analysis strongly suggests that UM171-expanded Cell Therapy improves key long-term clinical outcomes for patients suffering from blood cancers who need a matched unrelated donor or a cord blood transplant. More specifically, we observed very significant reductions in transplant related mortality and improvement in graft-versus-host disease and relapse-free survival, or GRFS, over these two graft sources,” said Guy Sauvageau, MD, PhD, CEO and Chief Scientific Officer of ExCellThera.

“This is another significant achievement for ExCellThera and our most advanced technology,” said Pierre Caudrelier, MD, Chief Medical Officer at ExCellThera. “With a GRFS hazard ratio (HR) below 0.3 and 0.4 in favor of ECT-001 Cell Therapy versus standard of care matched unrelated donor or cord blood transplants, respectively (p <0.01 and 0.02), it is apparent that ECT-001 Cell Therapy is significantly less hazardous. It is urgent that we get our technology in the hands of clinicians that share our enthusiasm in providing long-lasting complication-free solutions to these patients.”

Overall, 137 CIBMTR control patients and 22 ECT-001 Phase 1/2 trial patients were included in the study. Full details are available at: https://excellthera.com/selected-publications/. Selected results are shown in the table below.

ECT-001 Cell Therapy combines proprietary molecule UM171, and an optimized culture system. ECT‑001 Cell Therapy has been evaluated in over 100 adult and pediatric patients with severe blood disorders in several clinical trials in the United States and Canada (with additional clinical trials in Europe expected soon). ECT-001 Cell Therapy has received orphan drug designation and regenerative medicine advanced therapy (RMAT) designation from the FDA as well as orphan medicinal product designation, advanced therapy medicinal product (ATMP) classification and priority medicines (PRIME) designation from the EMA.

ECT-001 is an investigational cell therapy, and its safety and efficacy have not been established by the FDA, EMA, Health Canada or any other health authority. For more information about ECT-001 Cell Therapy, please visit excellthera.com.

ExCellThera is a world leader in blood stem cell expansion and rejuvenation. ExCellThera’s lead proprietary molecule, UM171, has become the standard for hematopoietic stem cell (HSC) expansion in cell and gene therapy. UM171’s first-in-class mechanism of action has been shown to expand and rejuvenate HSCs, and to counteract exhaustion from culture and gene modification which represent important limitations in cell and gene therapies. ExCellThera has developed commercial GMP grade UM171 which can be integrated into the manufacturing process of various cell and gene therapy products as a reagent and is supported by a Type II Drug Master File (DMF) filed with the FDA. In addition to ECT-001 Cell Therapy, ExCellThera also has a preclinical pipeline of next-generation cell & gene therapies in adjacent fields of use. For more information, visit excellthera.com.