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ExCellThera announces completion of Phase 2 clinical trials for UM171-expanded cell therapy in high-risk blood cancers

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MONTREAL, Nov. 15, 2022 (GLOBE NEWSWIRE) — ExCellThera Inc. (ExCellThera), a world leader in blood stem cell expansion and rejuvenation, announced today the completion of patient enrollment in its initial Phase 2 studies for high-risk leukemias and myelodysplastic syndromes (MDS). ECT-001 Cell Therapy, ExCellThera’s most advanced investigational drug, combines the proprietary molecule UM171 and an optimized culture system to produce unique UM171-expanded stem cell transplants, and it also enables the use of small cord blood units without compromising engraftment.

The initial Phase 2 studies have enrolled 50 patients treated with ECT-001 Cell Therapy in the United States and Canada and are designed to assess its safety and efficacy in patients with high-risk leukemias and MDS.

In light of the promising data from the ECT-001 Cell Therapy trials, extensions to these initial Phase 2 studies are being initiated in new clinical sites in the United States and Europe, thereby providing the opportunity for additional patients to receive treatment. In addition, ExCellThera is continuing enrollment in its ongoing pediatric study at Memorial Sloan Kettering Cancer Center.

Top-line data across all ECT-001 Cell Therapy Phase 1/2 trials, which will include over 100 patients as of the data lock date of November 30, 2022, are expected in the first half of 2023.

“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 U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and Health Canada, which are important steps in bringing this novel, one-time cell therapy to more patients as quickly as possible. 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,” said Guy Sauvageau, MD, PhD, Co-CEO and Chief Scientific Officer of ExCellThera.

“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,” said Pierre Caudrelier, MD, Chief Medical Officer of ExCellThera. “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.”

Results from the previously completed Phase 1/2 clinical trial published in the peer-reviewed medical journal, The Lancet Haematology, demonstrated that ECT-001 Cell Therapy is feasible, safe and allows for the use of small cords without compromising engraftment. In addition, a recent abstract entitled “Improved Outcomes of UM171-Expanded Cord Blood Transplantation Compared with Other Graft Sources: Real World Evidence” accepted for oral presentation at the upcoming 64th American Society of Hematology (ASH) Annual Meeting and Exposition, concludes, pursuant to a matched-controlled analysis from the CIBMTR® (Center for International Blood and Marrow Transplant Research®), that real-word evidence suggests ECT-001 Cell Therapy recipients have certain improved outcomes compared to cord blood and matched unrelated donor peripheral blood stem cell transplants.

“There are critical unmet needs in the area of high-risk leukemias and MDS, but also for patients in need of an allogeneic stem cell transplant who are unable to find suitable donor-derived stem cells. Current therapies are severely limited in their ability to treat these patients,” said Dr. Sauvageau. “We believe that ECT-001 Cell Therapy has the potential to provide high-risk patients and those without a donor with a potentially curative therapy that can truly make a meaningful difference in their lives. With this in mind, we are excited to review the top-line data of our first 100 patients expected in the first half of 2023, and we are looking forward to working with the regulatory authorities to secure market access and ensure ECT-001 Cell Therapy will be made available to a far greater number of patients in need as quickly as possible.”

About ECT-001 Cell Therapy

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

About ExCellThera Inc.

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 pre-clinical pipeline of next generation cell & gene therapies in adjacent fields of use.

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David Desjardins
Vice President, Business and Corporate Development

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