Achillion reports interim Phase II data for factor D inhibitor in PNH

Achillion Pharmaceuticals Inc. (NASDAQ:ACHN) reported interim data from 4 patients with untreated paroxysmal nocturnal hemoglobinuria (PNH) in a Phase II trial showing that oral ACH-4471 led to “meaningful” dose-related improvements in lactate dehydrogenase (LDH) levels, hemoglobin levels and fatigue score. The data include 3 patients with classic PNH and 1 patient with PNH and aplastic anemia. Two patients have completed the 3-month trial and entered into the study’s long-term extension phase, 1 patient withdrew for reasons unrelated to safety, and 1 patient continues dosing. ACH-4471 was well tolerated with no significant elevations in liver enzymes reported.

Patients in the open-label, dose-escalation, New Zealand trial received ACH-4471 at a starting dose of 100 mg thrice daily, which was titrated up to 200 mg thrice daily. Achillion said it will begin an add-on Phase II trial to support a "switch-strategy" for patients with a suboptimal response to Soliris eculizumab from Alexion Pharmaceuticals Inc. (NASDAQ:ALXN)...