In this Phase I/II study, 19 high-risk leukemia patients were treated with escalating doses of ATIR™ after a haploidentical HSCT. The five-year follow-up data show no transplant related mortality in the nine patients who received an efficacious dose of ATIR™. In addition, no Grade III-IV (life-threatening) acute Graft versus Host Disease (GvHD) was observed at any dose, which again compares favorably to standard of care matched unrelated donor transplantations, where (according to data from the CIBMTR) incidence of life-threatening GvHD is 30%. The strong five-year survival data also suggest that immune cells responsible for the Graft versus Leukemia effect are retained in ATIR™.
The results of the study allowed selecting the optimal ATIR™ dose for further development. Data from this five-year study will be published in due course in a peer-reviewed medical journal.
An international multi-center Phase II study including patients with AML, acute lymphoblastic leukemia (ALL) and myelodysplastic syndrome (MDS), to confirm and extend the data from the Phase I/II study, is now ongoing with topline results of the first phase expected in H1 2014.
Manfred Ruediger , PhD , Chief Executive Officer of Kiadis Pharma , commented: "The results from this study, which spans five years, show that ATIR™ might change the way in which patients with hematological malignancies will be treated. These data show that ATIR™ not only prevents significant infections without eliciting life-threatening GvHD, but also strongly improves survival rates in patients with high-risk malignancies and very poor prognosis. Our currently ongoing international Phase II clinical study is designed to confirm and extend these data to expedite moving ATIR™ towards regulatory approval."Dr. Denis-Claude Roy , Professor of Medicine at the University of Montreal and principal investigator for the study , added: "We are very excited about the long-term effects of ATIR™. In demonstrating very strong efficacy in minimizing post-transplantation risks and improving overall survival, these long-term data represent a potential major advancement in providing patients for whom a suitable matched donor is not available, with the opportunity to receive an HSCT from a mismatched family member with ATIR™ added as an adjunctive treatment." About ATIR™