Positive top-line results from the phase III PRINCE study of pegcetacoplan in treatment-naïve patients with paroxysmal nocturnal haemoglobinuria. SOBI + Apellis

Pegcetacoplan demonstrated statistical superiority on the co-primary endpoints of haemoglobin stabilization and reduction in lactate dehydrogenase (LDH) compared to standard of care, which did not include complement inhibitors, at week 26. i.86 per cent of pegcetacoplan treated patients achieved haemoglobin stabilisation compared to 0 per cent of patients on standard of care (p<0.0001). haemoglobin stabilisation was defined as an avoidance of a greater than 1 g dl decrease in haemoglobin levels in the absence of transfusions. ii. mean ldh in the pegcetacoplan group decreased by 90 per cent from a baseline of 2151 u l [9.5x upper limit of normal (uln)] to 211 u l, which is within the normal range, compared to a 14 per cent reduction on standard of care from a baseline of 1946 u l (8.6x uln) to 1681 u l (7.4x uln) (p><0.0001). pegcetacoplan also achieved statistical superiority on several secondary endpoints, including improvements in haemoglobin levels and transfusion avoidance, compared to standard of care, which did not include complement inhibitors. i mean haemoglobin levels in the pegcetacoplan group increased from 9.4 g dl to 12.1 g dl compared to an increase from a baseline of 8.7 g dl to 9.4 g dl on standard of care (p="0.0019)." ii. 91 per cent of patients on pegcetacoplan were transfusion free compared to 22 per cent on standard of care (p><0.0001). the safety profile of pegcetacoplan was consistent with previous studies. at week 26, 9 per cent of patients in the pegcetacoplan group experienced a serious adverse event (sae) compared to 17 per cent on standard of care. one death was reported in each group, and neither were related to treatment. no cases of meningitis or thrombosis were reported in either group. the most common adverse events reported during the study in the pegcetacoplan and standard of care groups, respectively, were injection site reaction (30 per cent vs. 0 per cent), hypokalemia (13 per cent vs. 11 per cent), and fever (9 per cent vs. 0 per cent).></0.0001).></0.0001).></0.0001).>