Veloxis Pharmaceuticals Announces Dosing of the First Patient by partner Xenikos in the Global Phase 3 Study Evaluating T-Guard® in Patients with Steroid-Refractory Acute Graft-Versus-Host Disease (SR-aGVHD)

T-Guard®, under development by Xenikos B.V., is being studied in comparison to ruxolitinib in patients with Grade III or IV SR-aGHVD

CARY, N.C., June 28, 2022 /PRNewswire/ -- Veloxis Pharmaceuticals, an Ashai Kasei company, today announced dosing of the first patient by its partner, Xenikos, in a global pivotal Phase 3 clinical study [NCT04934670] designed to evaluate T-Guard® versus ruxolitinib for the treatment of patients with Grade III or IV steroid-refractory acute graft-versus-host disease (SR-aGVHD) following allogeneic hematopoietic stem cell transplant (allo-HSCT). T-Guard is currently being developed by Xenikos B.V., a privately-held biotechnology company that develops innovative immunotherapies for treating patients with severe immune disease and post-transplant rejection.

In September 2021, Xenikos secured €40 million in convertible debt consisting of two equal tranches of €20 million, led by Veloxis Pharmaceuticals with participation from existing investors, Medicxi, RA Capital Management, Oost NL and Sanquinnovate. In connection with the financing, Veloxis has obtained two sequential call options to acquire the company.

"Patients who experience SR-aGVHD have limited treatment options and sadly, their condition is often fatal" said Ulf Meier-Kriesche, chief scientific officer of Veloxis. "This Phase 3 study comparing T-Guard, with its novel mechanism of action, to ruxolitinib, if successful, can help bring a new treatment option to patients who urgently need it."

"Veloxis is looking forward to the potential addition of T-Guard to its pipeline as we develop a broad portfolio of novel drugs to meet the needs of different groups of transplant patients," says Mark Hensley, CEO of Veloxis.

Xenikos reached agreement with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) on the design of this global, pivotal, randomized Phase 3 study, which is planned to enroll 246 patients and has been designed to test for superiority of T-Guard compared to ruxolitinib for the treatment of Grade III or IV SR-aGVHD. The study will be conducted at 75 transplant centers across the U.S. and Europe and will be executed in collaboration with the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). Patients will be randomized 1:1 to receive either T-Guard or ruxolitinib. Participants in the T-Guard arm will receive a one-week course of treatment with T-Guard as a four-hour infusion every other day. Each dose consists of 4mg/m² body surface area (BSA). Participants in the ruxolitinib arm will receive 10mg of ruxolitinib twice daily for a minimum of 56 days.

The primary endpoint of the study is complete response (CR) rate at Day 28, which is an important surrogate for long-term survival in patients with SR-aGVHD. Key secondary objectives include overall survival at Days 60, 90, 180 and 365, duration of complete response (CR), time to CR, overall response rate at Days 14, 28 and 56, GVHD-free survival, incidence of infections and safety.

There will be a safety run-in phase at the beginning of the study whereby the Data and Safety Monitoring Board (DSMB) will evaluate the first 24 patients. Xenikos expects to report results from the safety run-in phase during the first half of 2023. The trial will also include an interim analysis for futility after 46 patients become evaluable for the primary endpoint. A second interim analysis will be performed once 150 participants have reached Day 28.

Xenikos expects the data from this study to support the submission of a Biologics License Application (BLA) in the U.S. based on Day 28 data from the first 150 patients. Data from the full 246 patients is expected to support the submission of a Marketing Authorization Application (MAA) in the EU.

Following allogeneic stem cell transplantation, most patients have a high risk of developing graft-versus-host disease (GVHD). With GVHD, the donor's immune cells attack the patient's cells. Acute GVHD occurs early after transplantation and can be relatively mild or quite severe, even life-threatening, if not treated. Although GVHD can often be treated successfully with steroids, few options are available if the disease progresses or becomes resistant to steroid treatment, and the long-term survival of patients with steroid-refractory acute GVHD (SR-aGVHD) is less than 20%, highlighting the urgent need for effective new therapies.

T-Guard is designed to reset the body's immune system in life-threatening T cell‒mediated conditions, potentially including prevention of transplant-related rejection, treatment of acute solid-organ rejection, and severe autoimmune disease. T-Guard consists of a unique combination of toxin-conjugated monoclonal antibodies that target CD3 and CD7 molecules on immune cells. Preclinical and early clinical testing have shown that T-Guard can specifically identify and eliminate mature T cells and NK cells with tolerable treatment-related side effects. T-Guard's action is believed to be short-lived, which may alter a patient's vulnerability to opportunistic infections compared to currently available therapies. T-Guard has been previously studied in a Phase 1/2 study in comparison to institutional historical controls in patients being treated second-line for SR-aGVHD following hematopoietic stem cell transplantation (HSCT). To learn more about that study, please find it here (link). T-Guard has been granted Orphan Drug Designation in both the EU and the U.S.

The BMT CTN conducts rigorous multi-institutional clinical trials of high scientific merit, focused on improving survival for patients undergoing hematopoietic cell transplantation and/or receiving other cellular therapies. The BMT CTN has completed accrual to more than 50 Phase II and III trials at more than 100 transplant centers and enrolled over 16,000 study participants.

BMT CTN is funded by the National Heart, Lung, and Blood Institute and the National Cancer Institute, both parts of the National Institutes of Health (NIH) and is a collaborative effort of 20 Core Transplant Centers/Consortia, the Center for International Blood and Marrow Transplant Research (CIBMTR), the National Marrow Donor Program (NMDP)/Be The Match and the Emmes Company, LLC, a clinical research organization. CIBMTR is a research collaboration between the NMDP/Be The Match and the Medical College of Wisconsin.

The BMT CTN 2002 study is being led by Drs. Mehdi Hamadani, Protocol Officer, Scientific Director of CIBMTR, Medical College of Wisconsin; John Levine, Protocol Co-chair, Director of BMT Clinical Research, Mt. Sinai School of Medicine; Gérard Socié, Protocol Co-chair, Head of Hematology-Transplantation, Hôpital Saint-Louis; and Gabrielle Meyers, Protocol Co-chair, Associate Professor of Medicine, Oregon Health and Science University. More information about the BMT CTN can be found at bmtctn.net.

Xenikos develops innovative immunotherapies based on conjugated antibodies. This novel therapeutic approach helps reset the immune system in patients who have a severe immune disease or have developed post-transplantation rejection. A randomized Phase 3 registration trial evaluating the company's flagship product, T-Guard® for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) is underway in the U.S. and Europe.

Veloxis Pharmaceuticals, Inc, an Asahi Kasei company, is a fully integrated specialty pharmaceutical company committed to improving the lives of transplant patients. Headquartered in Cary, North Carolina, USA, Veloxis is focused on the global development and commercialization of medications utilized by transplant patients and by patients with serious related diseases. For further information, please visit www.veloxis.com.

The Asahi Kasei Group contributes to life and living for people around the world. Since its foundation in 1922 with ammonia and cellulose fiber business, Asahi Kasei has consistently grown through the proactive transformation of its business portfolio to meet the evolving needs of every age. With more than 40,000 employees around the world, the company contributes to sustainable society by providing solutions to the world's challenges through its three business sectors of Material, Homes, and Healthcare. Its healthcare operations include devices and systems for acute critical care, dialysis, therapeutic apheresis, transfusion, and manufacture of biotherapeutics, as well as pharmaceuticals and diagnostic reagents. For further information, please visit www.asahi-kasei.com.

SOURCE Veloxis Pharmaceuticals