Achillion Pharmaceuticals has initiated patient dosing in a Phase 2 open-label study of ACH-4471, Achillion’s first orally-administered, small molecule factor D inhibitor, for patients with paroxysmal nocturnal hemoglobinuria (PNH). This proof of concept study will assess the efficacy, safety, and pharmacokinetics of ACH-4471 in untreated patients with PNH.

“We are pleased to have begun this trial in PNH patients to evaluate ACH-4471, the first orally-administered factor D inhibitor to have demonstrated complement alternative pathway (AP) inhibition in humans, which represents a potentially novel and unique mechanistic approach to treating this life-threatening disease,” commented Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion.