ExCellThera, Inc.

- Market Authorisation Application (MAA) for UM171 Cell Therapy accepted under an accelerated assessment procedure by the European Medicines Agency (EMA), for adult patients with hematological malignancies requiring a stem cell transplant who lack a readily available suitable donor June 25, 2024 -- ExCellThera Inc. (ExCellThera), a world leader in enhanced blood stem cell expansion and therapies, announced today that the Market Authorisation Application (MAA) for UM171 Cell Therapy (INN-dorocubicel) has been accepted under an accelerated assessment procedure by the European Medicines Agency (EMA), for adult patients with hematological malignancies requiring a stem cell transplant who lack a readily available suitable donor. The EMA’s Committee for Medicinal Products for Human Use (CHMP) granted UM171 Cell Therapy accelerated assessment as it was deemed of major interest from a public health point of view. Accelerated assessment aims to reduce the timeframe for the CHMP to review a MAA compared to the standard procedure and follows the EMA granting Orphan Medicinal Product designation and access to the Priority Medicines scheme (PRIME) in 2020. “We are excited that the EMA has accepted this regulatory submission under an accelerated assessment procedure, as UM171 Cell Therapy has immense potential to help address a high unmet medical need for patients with hematological malignancies who require a stem cell transplant but lack a readily available suitable donor,” said David Millette, CEO of ExCellThera. “We now look forward to working closely with the EMA in the coming months to bring this therapy to the patients who need it.” 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. 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. The content above comes from the network. if any infringement, please contact us to modify.

MONTREAL, Jan. 02, 2024 (GLOBE NEWSWIRE) -- ExCellThera Inc. (ExCellThera), a world leader in blood stem cell therapies, announced today that its senior leaders will be holding investor meetings during the 42nd Annual J.P. Morgan Healthcare Conference, January 8-11, 2024, in San Francisco, CA, and will be participating in the following investor conferences in January: 2024 Biotech Showcase (Presentation) Presentation Date and Time: January 8, 2024 at 4:45 PM PT Speaker: David Millette, Chief Executive Officer Location: San Francisco, CA, Ballroom Franciscan D, Hilton Union Square ExCellThera will also hold one-on-one investor meetings during the 42nd Annual J.P. Morgan Healthcare Conference, January 8-11, 2024, in San Francisco, CA. To request a one-on-one meeting with ExCellThera, please contact sellinwood@kendallir.com. B Riley 4th Annual Oncology Conference (Fireside Chat) Presentation Date and Time: January 18, 2024 at 10:00 AM ET Speakers: Guy Sauvageau, Founder and Chief Scientific Officer and David Millette, Chief Executive Officer Location: Virtual 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. High Unmet Medical Need UM171 Cell Therapy 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. UM171 Cell Therapy has successfully completed Phase 2 trials in high and very high-risk acute leukemias and myelodysplasias. A Phase 3 trial is planned in 2024 for UM171 Cell Therapy for the treatment of high and very high-risk acute leukemias and myelodysplasias. Patients with hematological malignancies requiring a stem cell transplant who lack a readily available suitable donor, including familial, unrelated or haploidentical donor, or cord blood of sufficient quantity. A Market Authorization Application (MAA) submission was completed and is under accelerated assessment with the European Medicines Agency (EMA) for the licensure of UM171 Cell Therapy for adult patients with hematological malignancies requiring a stem cell transplant who lack a readily available suitable donor. 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. Investor and Media Contact Sarah Ellinwood Kendall Investor Relations sellinwood@kendallir.com

- 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.