Apellis initiates phase III PRINCE trial of APL 2 to treat paroxysmal nocturnal hemoglobinuria

Apellis Pharmaceuticals Inc. announced the dosing of the first patient in the Phase III clinical study PRINCE (APL2-308) , evaluating the efficacy and safety of APL 2 for treatment-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH). PRINCE is the second Phase III study that Apellis has initiated to investigate the potential of APL 2 to treat PNH.

The PRINCE study is a Phase III, randomized, multicenter, open-label, controlled study that aims to enroll 54 treatment-naïve adult patients with PNH. The primary objective of this study is to evaluate the efficacy of APL 2 in patients with PNH who are currently not being treated with complement inhibitors, as assessed by hemoglobin stabilization from baseline in the absence of transfusion through Week 26 and reduction in lactate dehydrogenase (LDH) level from baseline to Week 26.

PNH is a rare, acquired, potentially life-threatening disease characterized by complement-mediated hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes and/or some degree of bone marrow dysfunction. Some of the prominent symptoms of PNH include severe anemia, abdominal pain, headaches, back pain, excessive weakness, fatigue and recurrent infectionsii. If not treated, PNH results in the death of approximately 35 percent of affected individuals within five years of diagnosis and 50 percent of affected individuals within 10 years of diagnosis/