Apellis reports positive top-line results from phase III PEGASUS head-to-head study of pegcetacoplan compared to eculizumab for paroxysmal nocturnal hemoglobinuria.

Apellis Pharmaceuticals, Inc. announced positive results from the Phase III PEGASUS study evaluating pegcetacoplan (APL-2) in adults with paroxysmal nocturnal hemoglobinuria (PNH). Top-line data show that pegcetacoplan met the study’s primary efficacy endpoint, demonstrating superiority to eculizumab with a statistically significant improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16 (p<0.0001). at week 16 pegcetacoplan-treated patients n="41)" had an adjusted mean hemoglobin increase of 2.4 g dl from a baseline of 8.7 g dl compared to eculizumab-treated patients n="39)" who had a change of -1.5 g dl from a baseline of 8.7 g dl. additionally pegcetacoplan showed promising results in key secondary endpoints. pegcetacoplan met non-inferiority on transfusion avoidance and absolute reticulocyte count. pegcetacoplan also showed positive trends on lactate dehydrogenase ldh and fatigue as measured by the functional assessment of chronic illness therapy facit-fatigue score. in this study the safety profile of pegcetacoplan was comparable to eculizumab. seven of 41 patients 17.1 in the pegcetacoplan group experienced a serious adverse event sae and 6 of 39 patients 15.4 in the eculizumab group experienced saes. no cases of meningitis and no deaths were reported in either treatment group. the most common adverse events reported during the 16-week randomized controlled treatment period in the pegcetacoplan and eculizumab groups respectively were injection site reactions 36.6 vs. 2.6 diarrhea 22.0 vs. 0 headache 7.3 vs. 20.5 and fatigue 4.9 vs. 15.4. another common adverse event was hemolysis which was reported in four patients in the pegcetacoplan group 9.8 and nine patients in the eculizumab group 23.1. this led to the three discontinuations in pegcetacoplan group. all patients who completed the randomization period in both groups 77 80 entered the 32-week open-label pegcetacoplan treatment period.>