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.>