Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that researchers have presented preliminary data from a single-arm Phase 2 study of eculizumab (Soliris ®) as an investigational therapy to prevent acute antibody-mediated rejection (AMR) in sensitized deceased-donor kidney transplant recipients. The composite primary endpoint was the nine-week occurrence of post-transplantation treatment failure, which occurred in 10.6% of the 47 patients reported today, including a 6.4% rate of AMR 1 compared to an expected 30% rate of AMR 2 in this highly sensitized population of kidney transplant recipients. The data were presented today in an oral presentation at the 2013 annual congress of the European Society for Organ Transplantation (ESOT) in Vienna, Austria.
Acute AMR can lead to severe kidney allograft damage resulting in rapid loss of function and possible loss of the transplanted kidney, which makes AMR a significant clinical barrier to transplantation in sensitized patients. 3 Research suggests that uncontrolled activation of complement, triggered by the binding of donor-specific antibodies (DSAs) to their target proteins (antigens) of the donor kidney, may be the primary reason for acute AMR in kidney transplant recipients who are sensitized, or have DSAs, to their donors. 3,4 Prophylaxis with a terminal complement inhibitor, such as eculizumab, is thus considered a potential strategy to prevent acute AMR. There are no approved treatments for the prevention of acute AMR.
“Approximately 30% of kidney transplant candidates on waiting lists are sensitized, or have antibodies, to potential donors. Because conventional immunosuppressive therapies are ineffective for the prevention of AMR, sensitized patients often have to wait years for a kidney suitable for transplantation. Thus, AMR has emerged as a significant, long-standing clinical problem in transplantation,” said Denis Glotz, M.D., Ph.D., Chief of the Department of Nephrology and Transplantation at Saint-Louis Hospital in Paris, France. “The data from this study suggest that eculizumab may be an effective prophylaxis against acute AMR in kidney recipients with preexisting antibodies against their donor.”