ExCellThera announces new UM171 presentation on advances in stem cell transplant at the EBMT Annual Meeting

• Dr. Guy Sauvageau, Chief Scientific Officer at ExCellThera will present on improving stem cell quality with UM171 for better transplant outcome

MONTREAL, March 25, 2025 – ExCellThera Inc. (ExCellThera), a world leader in blood stem cell expansion and metabolic fitness, announced today that Dr. Guy Sauvageau, the company’s Chief Scientific Officer, will present on significant advances in hematopoietic stem cell (HSC) expansion during the 51st Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT).

Presentation details:

Over the years, Dr. Guy Sauvageau’s group at the Institute of Research in Immunology and Cancer (IRIC) in Montreal identified major determinants of HSC self-renewal that are central to HSC expansion and transplant. The mechanism of action of small molecule UM171 involves a new E3 ligase complex which is key to the epigenetic control of HSC expansion and metabolic fitness. This novel molecule and its mechanism of action has been used to expand and increase metabolic fitness of HSCs prior to their transplantation, leading to impressive clinical results that appear to outperform standard of care HSC transplants in patients with blood cancers. The potential of UM171 and its unique mechanism of action is being further investigated to make human HSC transplants safer and to make them more widely available and clinically efficacious for patients in need.

About UM171 Cell Therapy
UM171 Cell Therapy (INN/USAN-dorocubicel), developed by Cordex Biologics, a wholly owned subsidiary of ExCellThera, has been evaluated in 120 patients with 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) designations 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 patients with high and very high-risk acute leukemias and myelodysplasias who have limited treatment options with low survival outcomes and high incidence of relapse under the current standard of care, including patients with refractory or active disease, patients requiring a second transplant, and patients with TP53 mutations or other genetic abnormalities. A pivotal Phase 3 trial in this patient population will be initiated as soon as possible.

A Market Authorisation Application (MAA) for UM171 Cell Therapy is currently undergoing review by the European Medicines Agency (EMA) for adult patients with hematological malignancies requiring an allogeneic stem cell transplant for whom no suitable donor cells is are available.

The use of UM171 Cell Therapy in other patient populations, including 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 and UM171 Technology
ExCellThera is a world leader in enhanced blood stem cell therapies. ExCellThera’s proprietary EnhanceTM 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 biopharmas to help them develop best-in-class cell and gene therapies by leveraging the technologies that form the EnhanceTM platform, including the 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.