Nijmegen, the Netherlands, June 11, 2019 – The Dutch company Xenikos B.V., which develops innovative immunotherapies for treating patients with severe immune disease and post-transplant rejection, announced today that the U.S. Food and Drug Administration (FDA) has approved an investigational new drug (IND) application to initiate a U.S.-based clinical Phase 3 registration trial in order to test the efficacy of T-Guard for treating steroid-refractory acute graft-versus-host disease (SR-aGVHD) in patients following allogeneic stem cell transplantation.

This single-arm, multi-center Phase 3 trial (BMT CTN 1802) is designed to evaluate the efficacy and safety of T-Guard in 47 patients who receive an allogeneic stem cell transplantation and subsequently develop SR-aGVHD. The primary endpoint of the study will be the complete response rate at day 28, and key secondary endpoints will include the duration of complete response and overall survival rate at six months.

“Receiving FDA approval to initiate our U.S. Phase 3 registration trial is a major milestone for us, as it is the next step in advancing T-Guard towards the market,” says Dr. Ypke van Oosterhout, Chief Executive Officer of Xenikos. “Importantly, T-Guard can be administered as a single one-week treatment and was previously shown to promote swift restoration of the immune system, potentially reducing the risk of future infection and relapse. Effective therapies for treating patients with SR-aGVHD are urgently needed, and we believe that T-Guard has the potential to be an important addition to the medical arsenal available for treating patients with this devastating and potentially fatal condition.”

The trial will be conducted in the U.S. by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN), funded by the National Heart, Lung, and Blood Institute (NHLBI) and the National Cancer Institute (NCI), which are co-funding the trial. Drawing on its vast network of leading transplant centers throughout the U.S., the BMT CTN is expected to be able to quickly recruit patients for the trial, thereby helping bring T-Guard to market as quickly as possible.

Dr. Gabrielle Meyers, Study Co-Chair, asserts, “The BMT CTN is the only U.S. clinical trial group focused on improving outcomes for transplant recipients. For many years few studies were conducted for steroid-refractory GVHD due to the difficulty in treating this challenging patient population. We are excited about the opportunity to work with Xenikos to bring novel therapies to benefit patients in greatest need.”

Study Co-Chair Dr. John Levine notes, “Steroid-refractory GVHD is the driver for non-relapse deaths after allogeneic HSCT. Poor immune reconstitution and the resulting infections are major contributors to the high mortality. T-Guard’s novel mechanism of action that preferentially targets activated T cells has shown rapid immune recovery and high response rates. This collaboration with the BMT CTN will hopefully develop the confirmatory evidence needed to move T- Guard towards FDA approval.”