T-Guard™ was granted Orphan Designation in the EU and in the US for the treatment of steroid resistant acute Graft-versus-Host Disease (GVHD). This is a frequent and potentially life-threatening complication of bone marrow or blood stem cell transplantation for which currently no registered therapy exists.
In March 2014, Xenikos started a phase 1/2 multi-center trial in 20 patients with severe acute GVHD who had failed corticosteroids. This trial has been completed recently and the results show that T-Guard™ has a good safety and tolerability profile and very promising clinical efficacy. The results will be presented at the upcoming 2017 ASH annual meeting in Atlanta, USA. A scientific publication is to be submitted to a high-ranked peer-reviewed journal shortly. On the basis of the positive preliminary results, preparations for a randomized, controlled pivotal phase 3 trial are ongoing. In this study the safety and efficacy of T-Guard will be compared to the best available, but not registered, therapies. The study is scheduled to start early 2018.
Xenikos aims to further explore T-Guard’s therapeutic potential in alternative indications that might benefit from rebalancing of the immune system, like solid-organ rejection and selected autoimmune diseases.