Alexion Pharmaceuticals, Inc. (ALXN) today announced that positive results from one of the two large Phase 3 studies of ALXN1210, the Company’s investigational long-acting C5 complement inhibitor, in patients with paroxysmal nocturnal hemoglobinuria (PNH) were selected for presentation during the Late-Breaking Oral Session (at 11:45 a.m. CET) on Sunday, June 17, 2018 at the Annual Conference of the European Hematology Association (EHA) in Stockholm, Sweden.
“We are very excited about EHA’s recognition of the robustness and importance of these data in complement inhibitor treatment-naïve patients. We enrolled a very broad patient population, representative of patients with PNH in clinical practice, including patients with a history of aplastic anemia, and ‘classic’ PNH, as well as transfused and non-transfused patients,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “We look forward to discussing the data with leading hematologists in Europe at the conference later this week as part of our ambition to make ALXN1210 the new standard of care for patients with PNH.”
As previously announced, weight-optimized treatment every eight weeks with ALXN1210 demonstrated non-inferiority to treatment every two weeks with Soliris® (eculizumab) in complement inhibitor treatment-naïve patients with PNH on the two co-primary endpoints and all four key secondary endpoints. The numeric results for all these endpoints, including breakthrough hemolysis (one of the key secondary endpoints), favored ALXN1210 and are consistent with the immediate and complete C5 inhibition observed by the end of the first infusion of ALXN1210 and sustained throughout the entire 26-week treatment period. The safety profile of ALXN1210, based on almost 180 patient years of experience, was similar to that of Soliris®.
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive, debilitating, and potentially life-threatening ultra-rare blood disorder that can strike men and women of all races, backgrounds, and ages without warning, with an average age of onset in the early 30s.1,2,3 PNH often goes unrecognized, with delays in diagnosis ranging from one to more than 10 years.2 In patients with PNH, chronic, uncontrolled activation of the complement system, a component of the body’s immune system, results in hemolysis (the destruction of red blood cells)4, which in turn can result in progressive anemia, fatigue, dark urine, and shortness of breath.5,6,7 The most devastating consequence of chronic hemolysis is thrombosis (the formation of blood clots), which can damage vital organs and cause premature death.8 Historically, it had been estimated that one in three patients with PNH did not survive more than five years from the time of diagnosis.2 PNH is more common among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndromes (MDS).9,10,11 In certain patients with thrombosis of unknown origin, PNH may be an underlying cause.4
ALXN1210 is an innovative, long-acting C5 inhibitor discovered and developed by Alexion that works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system that, when activated in an uncontrolled manner, plays a role in severe ultra-rare disorders like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG). In Phase 3 clinical studies in complement inhibitor-naïve patients with PNH, and patients with PNH who had been stable on Soliris®, intravenous treatment with ALXN1210 every eight weeks demonstrated non-inferiority to intravenous treatment with Soliris® every two weeks, with numeric results for all primary and key secondary endpoints favoring ALXN1210. ALXN1210 is also currently being evaluated in a Phase 3 clinical study in complement inhibitor-naïve patients with aHUS, administered intravenously every eight weeks. In addition, Alexion plans to initiate a Phase 3 clinical study of ALXN1210 delivered subcutaneously once per week as a potential treatment for patients with PNH and aHUS. Alexion also plans to initiate the development of ALXN1210 as a potential treatment for patients with generalized MG (gMG) and patients with immunoglobulin A nephropathy (IgAN).