BioCentury
ARTICLE | Clinical News

Achillion gains on early factor D inhibitor data

August 10, 2017 12:15 AM UTC

Achillion Pharmaceuticals Inc. (NASDAQ:ACHN) rose $0.80 (20%) to $4.81 on Wednesday after announcing late Tuesday proof-of-concept data for ACH-4471 to treat paroxysmal nocturnal hemoglobinuria (PNH). Interim data from the first four patients with untreated PNH in a Phase II trial showed that oral ACH-4471 led to “meaningful” dose-related improvements in lactate dehydrogenase (LDH) levels, hemoglobin levels and fatigue score.

The data include three patients with classic PNH and one patient with PNH and aplastic anemia. Two patients have completed the three-month trial and entered into the study’s long-term extension phase, one patient withdrew for reasons unrelated to safety, and one patient continues dosing. ACH-4471 was well tolerated with no significant elevations in liver enzymes reported...