Alexion Pharmaceuticals’s data from two Phase 1b/2 studies of ALXN1210, the company’s investigational long-acting C5 complement inhibitor, in patients with paroxysmal nocturnal hemoglobinuria (PNH), show that treatment for up to eight months resulted in rapid and sustained reduction of plasma lactate dehydrogenase (LDH) levels from 73% to 88%.
ALXN1210 was generally well tolerated with a safety profile that is consistent with that seen historically in patients with complement inhibition. All patients from the Phase 1b study and from Cohorts 1, 2, and 3 of the Phase 2 study have been successfully transitioned to the Phase 3 dosing regimen, after which plasma LDH levels have remained suppressed.
“It is encouraging to see rapid and sustained reduction in plasma LDH levels in these dose optimization studies,” said an investigator in the Phase 1b/2 studies Alexander Röth, M.D. from the Department of Hematology, University Hospital Essen. “These comprehensive results provide robust preliminary evidence for the efficacy and safety of ALXN1210 as a future treatment for patients with PNH.”
John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion, said: “The strength of these data and exposure-response analyses, along with the totality of data for ALXN1210 and discussions with global regulators, allowed us to determine an eight-week, weight-based dosing regimen that targets complete C5 inhibition and rapid and sustained suppression of LDH. We have completed enrollment in our two multinational Phase 3 PNH studies, with nearly 450 patients enrolled, and expect data from these studies in the second quarter of 2018.”