Preliminary data from Phase 2 study shows that ECT-001 demonstrates excellent leukemia free survival.
Enrolment in Phase 2 trials of ECT-001 Cell Therapy expected to be competed in 2020.
ExCellThera to continue dialogue with the FDA on a registration-enabling study under the RMAT designation, and with the European Medicines Agency for development in Europe.
MONTREAL, February 25, 2020 – ExCellThera Inc., a clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, recently announced preliminary data from the Phase 2 trial of its ECT-001 Cell Therapy in patients with high-risk leukemia and myelodysplasia (MDS). The preliminary data was presented at the 2020 Transplant and Cellular Therapy (TCT) Annual Meeting in Orlando, Florida, and demonstrates excellent leukemia free survival in patients with high-risk leukemia and myelodysplasia.
ECT-001 Cell Therapy is a combination of a small molecule, UM171, and an optimized culture system. The technology, capable of expanding the number of stem and immune cells exponentially in as little as seven days, is used in novel curative cord blood transplant therapies for patients with hematological malignancies, allowing more rapid engraftment, greatly reduced incidence of transplant-related mortality, low risk of chronic graft-versus-host disease and low risk of relapse, resulting in better outcomes for patients.
“The preliminary data from the ECT-001 Cell Therapy Phase 2 trial suggests excellent leukemia free survival for high-risk patients with leukemias and myelodysplasia; this data is extremely encouraging as these leukemia free survival results are not seen for this high-risk patient population under the current standard or care,” said Dr. Guy Sauvageau, CEO and founder of ExCellThera. “This preliminary Phase 2 data in high-risk leukemias and myelodysplasia adds to the body of excellent clinical data for ECT-001, which has been dosed in over 50 patients to date. It further validates the introduction of the cryopreserved ECT-001 product, which will enable us to perform multi-centre trials and eventually provide global patient access to our cell therapy product.”
ExCellThera expects that enrolment in Phase 2 trials of its ECT-001 Cell Therapy will be competed in 2020 and intends to continue dialogue with the FDA under the auspices of the RMAT designation on a pivotal registration-enabling study, and to have discussions with the European Medicines Agency for development in Europe.
Data presented at the 2020 Transplant and Cellular Therapy (TCT) Annual Meeting
Presenter: Sandra Cohen, MD, Hôpital Maisonneuve-Rosemont/Université de Montréal
Updated data from Phase 1/2 trial (completed) of ECT-001 Cell Therapy in patients with hematological malignancies:
- Clinically viable with a rapid 7-day expansion and small culture volume
- Accelerates clinically relevant engraftment (100 neutrophils at D+9.5) regardless of cord blood unit size
- Reduced hospitalization length and fever (last day of fever D+7.5)
- A single expanded cord is possible for all patients, with better HLA matching
- Access to up to 50% of cord blood units in banks for 70kg patient
- Low incidence of transplant related mortality (5%)
- Excellent chronic GVHD-relapse-free survival (CRFS) of 74% at 33 months
Preliminary Data from Phase 2 trial (ongoing) of ECT-001 Cell Therapy in patients with high-risk leukemia and myelodysplasia
- To date, seven (7) high-risk patients dosed with ECT-001 Cell Therapy
- Preliminary data suggests excellent leukemia free survival
About ExCellThera Inc.
ExCellThera is a clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for use in novel one-time curative therapies for patients with hematologic malignancies, autoimmune and other diseases. ExCellThera’s lead solution combines a proprietary small molecule, UM171, and an optimized culture system. In pursuit of better treatments for patients, the company is building out its portfolio of products, as well as supporting best-in-class clinical trials. www.excellthera.com