Kiadis Pharma today provided an update on its cell therapy product ATIR™ developed for mismatched bone marrow transplants.
ATIR™ continues to show excellent clinical results in patients who received a mismatched bone marrow transplantation from a family member donor. The two year follow up data from the phase I/II study show no Transplant Related Mortality (TRM) in a group of 10 high risk leukemia patients who received an efficacious dose of ATIR™. The overall survival of this group after two years is 70%. These results compare favorably to outcomes of bone marrow transplantations from a full matching donor.
In addition, the FDA has granted ATIR™ an Orphan Drug Designation as a cell based therapy for the reduction of Transplant Related Mortality caused by Graft versus Host Disease (GvHD) and/or infections following allogeneic bone marrow transplantation. Transplant related mortality is predominantly caused by acute Graft versus Host Disease (GvHD) or infections. Today, bone marrow transplants require a full match between patient and donor, and prophylactic use of immune suppressants post transplantation is essential to limit and manage risk of acute GvHD. However, this approach leaves patients without an active immune system for a prolonged period post transplantation, resulting in increased mortality due to infections. ATIR™ is a cell based product of mismatched donor immune cells from which the acute GvHD causing cells have been removed. By eliminating the risk of severe acute GvHD, the prophylactic use of immune suppressants can be avoided post transplantation, quickly giving patients a functioning immune system and ultimately resulting in a reduced Transplant Related Mortality (TRM).
“The FDA’s decision to grant ATIR an Orphan Drug Designation underscores the significant potential of our program for bone marrow transplant patients and their families. We are excited and encouraged by the clinical success to date, and are focused on rapidly developing ATIR to better serve this patient community.” said Manja Bouman, CEO of Kiadis Pharma.
ATIR™ is a cell based therapy of donor T-lymphocytes depleted of alloreactive T-cells and is infused after a patient receives a mismatched bone marrow transplantation to reduce Transplant Related Mortality. ATIR™ is designed to prevent severe acute GvHD while providing an early immune reconstitution to fight infections and remaining tumor cells, thereby reducing TRM and improving overall survival. Moreover, it enables the use of a mismatched, related (haplo-identical) donor, thus providing virtually everyone with an immediately available donor and treatment and addressing a high unmet medical need. Today patients eligible for an allogeneic transplantation, but without a matched donor available, have no treatment options left. ATIR™ can be considered a truly individualized medicine since it is prepared for each patient specifically.
ATIR™ is currently in a multinational, registration clinical study, with sites in Europe and Canada open for enrollment.