ExCellThera Inc., presented data on the company’s Phase I/II clinical trial using its lead technology, ECT-001, for allogeneic stem cell transplant at the 60th American Society of Hematology (ASH) Meeting, this December in San Diego, USA.
Founded in 2015, headquartered in Montréal, Canada, 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 ECT-001 combines a proprietary 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, potentially curative, cord blood transplant therapies for patients with blood cancers, 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.
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.
The clinical trial presentation at ASH detailed results from 21 patients with high-risk hematologic malignancies and multiple comorbidities engrafted with blood cells expanded with ECT-001 as part of a Phase I/II multicentre trial.
The study showed that a 7-day ECT‑001 expanded single cord blood unit provided substantial clinical benefits, including faster engraftment, fewer infectious complications, better HLA matching and very low treatment-related mortality, all the while saving production and hospitalization time and costs. Furthermore, a very low rate of significant chronic graft-versus-host disease (GvHD) was observed and patients’ overall quality of life, as measured by GvHD and relapse free survival (GRFS), was excellent.
With a median follow up of 14 months, there was no moderate-severe chronic GvHD. With a median comorbidity index of 2 and some very high-risk patients, there has been one treatment-related mortality and three cases of relapse.
At 12 months, overall survival, progression-free survival and GRFS were excellent at 95%, 77% and 67%, respectively. Immunosuppressive therapy was discontinued at 12 months in 76% of patients.
“These results further validate our lead technology, ECT-001, which is also being used in an ongoing clinical trial for multiple myeloma and will be used in two new phase II studies scheduled to begin in Q1, 2019.” … “The data presented at ASH shows that rapid engraftment of stem and immune cells expanded with ECT-001 can favorably compete – and improve upon –the current standard of care.”
“These results indicate that ECT-001 expanded single cord transplants combine the advantages of conventional grafts using bone marrow (low treatment-related mortality), peripheral blood (low relapse) and cord blood (accessibility, low relapse and chronic GvHD) in a single, low cost, easy to produce 7-day culture product, which could lead to a paradigm shift in bone marrow transplantation.” – Dr. Guy Sauvageau, CEO and founder of ExCellThera