ExCellThera Announces Clearance by FDA & Health Canada for New Clinical Trials

ExCellThera Inc. recently announced its lead technology, ECT-001, will be used as part of two new clinical trials in patients with high-risk leukemia. The announcement follows the recent clearance of the company’s Investigational New Drug (IND) application by the U.S. Food and  Drug Administration (FDA) and of a clinical trial application by Health Canada, in each case for the use of ECT-001 to treat patients with high-risk leukemia and myelodysplasia. The US-based study will be conducted at the Fred Hutchinson Cancer Centre in Seattle, Washington, with the Canadian study principally taking place at the Hôpital Maisonneuve-Rosemont in Montreal, Quebec.

Leukemia is a cancer that begins in blood stem cells and can develop very quickly. It results in the over-production of abnormal cells, which crowd out healthy blood cells and leave the patient tired, short of breath and prone to bleeding, bruising and infection. Current treatment may include chemotherapy, radiation and a hematopoietic (blood) stem cell transplant.

“Blood stem cell transplants present a life-saving option for some patients; however, they can lead to life-threatening complications,” said Dr. Guy Sauvageau, CEO and founder of ExCellThera. “ECT-001 expanded blood transplants are reengineered grafts with higher quantities of stem and immune cells. Our clinical data thus far show the grafts greatly reduce the risks of post-transplant complications, including chronic graft-versus-host-disease, infection and disease relapse, resulting in better overall well-being for patients.”

The two new studies are designed to be complementary and will each enrol up to 20 patients, representing geographically distinct populations. Primary endpoints include relapse free survival and incidence of transplant related mortality. Secondary endpoints include graft-versus-host disease and other infectious complications.

ExCellThera plans to initiate additional trials, including a Phase III pivotal trial in the United States and Canada, in the coming months. In December 2018, ECT-001 received FDA orphan drug designation for the prevention of graft-versus-host disease.

The ECT-001 technology 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 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.

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. For more information, visit excellthera.com.