– UM171 Cell Therapy has been granted an International Nonproprietary Name (INN) of “dorocubicel” on the INN Recommended List issued by the World Health Organization (WHO)
– The U.S. Food and Drug Administration (FDA) has granted UM171 Cell Therapy (INN-dorocubicel) an additional orphan drug designation to enhance cell engraftment and immune reconstitution
MONTREAL, June 4, 2024 – ExCellThera Inc. (ExCellThera), a world leader in enhanced blood stem cell expansion and therapies, announced today that UM171 Cell Therapy has been granted the International Nonproprietary Name (INN) of “dorocubicel” on the INN Recommended List issued by the World Health Organization (WHO). Commonly referred to as a generic name, each INN is a unique name used to identify pharmaceutical substances or active pharmaceutical ingredients.
ExCellThera also announced today that the U.S. Food and Drug Administration (FDA) has granted UM171 Cell Therapy an additional orphan drug designation (ODD) to enhance cell engraftment and immune reconstitution in patients with hematological diseases receiving hematopoietic stem cell transplant. This new designation is in addition to the ODD previously granted to UM171 Cell Therapy by the FDA for the prevention of graft-vs-host-disease (GvHD). UM171 Cell Therapy also previously received an orphan medicinal product designation from the European Medicines Agency (EMA) for treatment in haematopoietic stem cell transplantation.
“Approval of the name dorocubicel is an important step in the commercial development of the UM171 Cell Therapy, signaling to potential partners and collaborators our commitment to the long-term development of this novel cell therapy product,” said David Millette, CEO of ExCellThera. “In addition, today’s announcement of yet another orphan designation speaks to the potential of UM171 Cell Therapy in addressing the medical needs of patients requiring an allogeneic stem cell transplant.”
About UM171 Cell Therapy
UM171 Cell Therapy (INN-dorocubicel), developed by Cordex Biologics, a wholly owned subsidiary of ExCellThera, has been evaluated in 120 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) 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. The initiation of a Phase 3 trial in this patient population is planned for the second half of 2024.
The use of UM171 Cell Therapy in other patient populations, including patients with multiple myeloma, 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 expansion. 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- and best-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.
