ExCellThera Inc.’s lead technology, ECT-001, will be used in two new U.S. and Canadian clinical trials in patients with high-risk leukemia. In December 2018, ECT-001 received orphan drug designation for the prevention of graft-versus-host disease.
Current treatment for leukemia, which can develop rapidly, may include chemotherapy, radiation, and a hematopoietic stem cell transplant.
Although blood stem-cell transplants are a life-saving option for some patients, they can cause life-threatening complications. ECT-001 expanded blood transplants are reengineered grafts that have greater quantities of stem and immune cells. According to the researchers, clinical data thus far demonstrate that the grafts greatly reduce the risk of post-transplant complications, including chronic graft-versus-host-disease and infection and disease relapse.
The two new studies will be complementary and will each enroll up to 20 patients. Primary endpoints include relapse-free survival and incidence of transplant-related mortality. Secondary endpoints include graft-versus-host disease as well as other infectious complications.
ECT-001 is a combination of the small molecule UM171 and an optimized culture system. The technology, which can expand the number of stem and immune cells exponentially in as little as seven days, is used in novel, curative blood-transplant therapies for patients with blood cancers. This enables faster engraftment, a much-reduced occurrence of transplant-related mortality, and a low risk of chronic graft-versus-host disease and of relapse.
ExCellThera plans to begin additional trials, including a phase III pivotal trial in the U.S. and Canada, over the coming months.
Source: ExCellThera, March 25, 2019