Apellis reports data from Phase Ib PADDOCK trial of C3 inhibitor in PNH

Apellis Pharmaceuticals Inc. (Crestwood, Ky.) reported data from 3 evaluable patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not received Soliris eculizumab in the Phase Ib PADDOCK trial showing that once-daily 270 mg subcutaneous APL-2 reduced average lactate dehydrogenase (LDH) levels to 275 U/L from 1,615 U/L during the first month. APL-2 was well tolerated and no episodes of breakthrough hemolysis were reported.

The open-label, New Zealand trial is evaluating once-daily 180, 270 and 360 mg doses of APL-2 in about 6 patients. The primary endpoint is safety...