Achillion Pharmaceuticals, Inc. reported top-line data from a dose-finding Phase 2 trial assessing the safety and effectiveness of its oral small molecule factor D inhibitor danicopan (ACH-4471) in combination with intravenous eculizumab in paroxysmal nocturnal hemoglobinuria (PNH) patients who have an inadequate response to C5 monotherapy. Data from the Phase 2 trial were presented in a poster presentation at the 61stAmerican Society of Hematology (ASH) Annual Meeting in Orlando, FL. The primary endpoint of the trial was an increase in hemoglobin from baseline. A mean increase of 2.4 g/dL at 24 weeks of treatment was achieved in this proof-of-concept trial. Danicopan, in combination with eculizumab, resulted in a significant reduction in blood transfusions with 10 patients receiving 34 transfusions (58 units) in the 6 months prior to screening to 1 patient receiving 1 transfusion (2 units) during the 24-week trial. In addition to improvements in hemoglobin and transfusions, there were also meaningful improvements in markers of hemolysis including bilirubin, reticulocytes, and PNH red blood clone size (%). In this clinical trial, danicopan was generally well tolerated. All treatment emergent adverse events were considered mild to moderate in severity except for Grade 3 severe adverse events that occurred in two patients. Both patients had resolution of their events, remained on danicopan, and completed the study. The Company was granted Breakthrough Therapy designation by Food & Drug Administration (FDA) in September and PRIME designation by the European Medicines Agency (EMA) in November for danicopan for the treatment of PNH in combination with a C5 monoclonal antibody for patients who are suboptimal responders to a C5 inhibitor therapy alone. In addition, the Company met with the FDA during an End of Phase 2 Meeting in the fourth quarter and is progressing to Phase 3. The Company plans to initiate a global Phase 3 trial in early 2020.