Based on these data, Apellis has launched a large phase III trial in PNH patients with suboptimal hematological response to eculizumab, defined as Hb level <10.5 g/dL; in this study, after a short period of concomitant treatment (to reach APL-2 steady-state), patients will be randomized to continue either eculizumab or APL-2 monotherapy (120). This evidence concerns the gene GSTM1 and paroxysmal nocturnal hemoglobinuria.