ALXN 1210 success for patients switching from Soliris for paroxysmal nocturnal hemoglobinuria .- Alexion Pharma .

Alexion announced positive topline results of a Phase III study of ALXN 1210, the Company�s investigational long-acting C5 complement inhibitor, which show that patients with paroxysmal nocturnal hemoglobinuria (PNH) can be effectively and safely switched from treatment with Soliris (eculizumab) every two weeks to treatment with ALXN 1210 every eight weeks. The study demonstrated non-inferiority of ALXN 1210 to Soliris in patients with PNH who had been stable on Soliris based on the primary endpoint of change in lactate dehydrogenase (LDH) levels, a direct marker of complement-mediated hemolysis in PNH. The study also demonstrated non-inferiority on all four key secondary endpoints: the proportion of patients with breakthrough hemolysis, the change from baseline in quality of life as assessed via the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale, the proportion of patients avoiding transfusion, and the proportion of patients with stabilized hemoglobin levels.

In addition, numeric results for all five endpoints favored ALXN 1210. Notably, no patients treated with ALXN 1210 experienced breakthrough hemolysis compared to five patients treated with Soliris. ALXN 1210 was generally well tolerated with a safety profile that is consistent with that seen for Soliris. The most frequently observed adverse events were headache and upper respiratory infection. The most frequently observed serious adverse events were pyrexia, which occurred in three Soliris patients, and hemolysis, which occurred in two Soliris patients. No patient withdrew from the study due to adverse events. No treatment-emergent anti-drug antibody was observed for ALXN 1210; one was observed for Soliris . No neutralizing antibodies and no apparent effects on efficacy, safety, pharmacokinetics, or pharmacodynamics were detected. There were no cases of meningococcal infection observed in either the ALXN 1210 or Soliris arms. Meningococcal infections are a known risk with terminal complement inhibition, and specific risk-mitigation plans have been in place for ten years for Soliris to minimize the risk for patients. Detailed results from this Phase III study will be presented at a future medical congress.

Comment: ALXN 1210 is a long-acting form of Soliris for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) which will only require treatment once every eight weeks compared with two weeks for patients taking Soliris.