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.