Apellis planning Phase III PNH program

Apellis Pharmaceuticals Inc. (Crestwood, Ky.) intends to begin a Phase III program in 4Q17 evaluating APL-2 to treat paroxysmal nocturnal hemoglobinuria. The program will include two trials of the complement 3 (C3) inhibitor, including one head-to-head study comparing the therapy with Soliris eculizumab from Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) in patients who have previously responded poorly to that therapy, President and CEO Cedric Francois told BioCentury.

In 4Q17, Apellis plans to start the first Phase III trial, which will evaluate APL-2 as an add-on to Soliris. The second trial, which Apellis expects to begin in 1H18, will evaluate APL-2 among suboptimal responders to Soliris. Francois said the second trial will include a pre-randomization stage in which patients receive both therapies for one month, then a randomized head-to-head stage. Apellis has not yet chosen a primary endpoint for the studies, but Francois said hemoglobin levels will be "a key component."...