- Data presented in an oral session at ASH 2023, building on strong data from prior Phase 1/2 trial and comparative registry studies
- UM171 cell therapy achieved the primary endpoint of progression-free survival (63% at 2 years) in high and very high-risk cohort and demonstrated an excellent safety profile
- Company plans to initiate a registrational Phase 3 trial in in high and very high-risk leukemias and myelodysplasias in 2024
MONTREAL, Dec. 12, 2023 (GLOBE NEWSWIRE) -- ExCellThera Inc. (ExCellThera), a world leader in blood stem cell expansion and metabolic fitness, announced today positive results from 50 patients in two multi-center Phase 2 trials for UM171 cell therapy conducted at transplant centers in the U.S., Canada and Europe, building on prior strong data from a Phase 1/2 trial and comparative registry studies (CIBMTR and EBMT).
The data were presented for the first time in an oral session at the 65th American Society of Hematology Annual Meeting & Exposition (ASH) by Filippo Milano, M.D., Ph.D., Associate Professor, Translational Science and Therapeutics Division, Fred Hutchinson Cancer Center, and demonstrated excellent clinical outcomes at two years for this high and very high-risk cohort, including strong rates of overall survival (67%) and progression-free survival (63%), as well as low rates of non-relapse mortality (19%), relapse (18%) and moderate-to-severe chronic GVHD (7%).
“There is an unmet need for new treatment options for patients with high and very high-risk leukemias and myelodysplasias who have low survival outcomes and high incidence of relapse,” said Filippo Milano, M.D., Ph.D., Director of Fred Hutch’s Cord Blood Transplant Program. “In these UM171 Phase 2 studies, about 70% of patients survived two years post-transplant without evidence of disease recurrence. These outcomes are encouraging considering that 30% of the enrolled patients did not benefit from previous stem cell transplantation. In addition, 20% of the study population had an identified TP53 genetic mutation, which has been associated with particularly dismal outcomes.”
“Patients in high-risk categories, including those with refractory or active disease, with genetic abnormalities, or who have relapsed after a first transplant, have limited treatment options under the current standard of care,” said David Millette, Chief Executive Officer of ExCellThera. “We are very excited to share the positive Phase 2 results for UM171 cell therapy, building on strong data from our Phase 1/2 trial and comparative registry studies. We believe this establishes UM171 cell therapy as a potential treatment options for high-risk leukemias and myelodysplasias based on its combined efficacy and safety profile. Based on these highly compelling data, we are looking forward to advancing UM171 cell therapy forward to a registrational Phase 3 trial in high and very high-risk leukemias and myelodysplasias in 2024.”
About UM171 Cell Therapy
UM171 cell therapy has been evaluated in over 100 patients to treat hematologic malignancies in clinical trials in the United States, Europe and Canada. UM171 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. UM171 cell therapy has successfully completed Phase 2 trials in high and very high-risk acute leukemias and myelodysplasias, and a Phase 3 trial is expected as soon as possible.
Patients with high and very high-risk acute leukemias and myelodysplasias, including patients with refractory or active disease, patients requiring a second transplant, and patients with TP53 mutations or other genetic abnormalities, have limited treatment options with low survival outcomes and high incidence of relapse under the current standard of care.
The use of UM171 cell therapy in other patient populations, including patients unable to find a stem cell donor, pediatric patients and patients with non-malignant hematological diseases, is also being explored.
UM171 cell therapy 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.
About ExCellThera
ExCellThera is a world leader in blood stem cell expansion and metabolic fitness. ExCellThera’s proprietary Enhance™ platform for cell expansion and metabolic fitness is designed to deliver a greater dose of functional therapeutic stem cells by expanding HSCs from any source and counteracting the effects of culture or gene editing induced stress. ExCellThera partners with bio-pharmas to help them develop best-in-class cell and gene therapies by leveraging the technologies that form the EnhanceTM platform, including proprietary molecule UM171 which has a first-in-class mechanism of action for ex vivo expansion and metabolic fitness of HSCs. For additional information, visit excellthera.com, and follow us on LinkedIn.
