Alexion to present results from ALXN1210 Phase 3 study at EHA
Alexion Pharmaceuticals (ALXN) announced that 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 were selected for presentation during the Late-Breaking Oral Session on Sunday, June 17, 2018 at the Annual Conference of the European Hematology Association in Stockholm, Sweden.
“We are very excited about EHA’s recognition of the robustness and importance of these data in complement inhibitor treatment-naive 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., EVP 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 in complement inhibitor treatment-naive 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, 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.
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.
PNH often goes unrecognized, with delays in diagnosis ranging from one to more than 10 years. 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), which in turn can result in progressive anemia, fatigue, dark urine, and shortness of breath.
The most devastating consequence of chronic hemolysis is thrombosis (the formation of blood clots), which can damage vital organs and cause premature death.
Historically, it had been estimated that one in three patients with PNH did not survive more than five years from the time of diagnosis.
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.
ALXN closed at $118.20.
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