The NEJM publishes phase III PEGASUS study results comparing pegcetacoplan to eculizumab for PNH.- SOBI

Swedish Orphan Biovitrum AB (publ) (Sobi™) and Apellis Pharmaceuticals, Inc. announced that The New England Journal of Medicine (NEJM) published results from the phase III PEGASUS study, which demonstrated the superiority of pegcetacoplan, an investigational targeted C3 therapy, in improving haemoglobin levels and showed improvements in key clinical outcomes compared to eculizumab, a C5 inhibitor, in adults with paroxysmal nocturnal haemoglobinuria (PNH) at 16 weeks who had persistent anaemia following treatment with eculizumab. The results published in NEJM highlight that pegcetacoplan met the study's primary endpoint for efficacy, demonstrating superiority to eculizumab with a statistically significant improvement in adjusted means of 3.8 g/dL of haemoglobin at week 16 (p<0.001). additionally, 85% of pegcetacoplan-treated patients were transfusion free over 16 weeks versus 15% of eculizumab-treated patients. there were meaningful improvements across key markers of disease such as absolute reticulocyte count, lactate dehydrogenase (ldh), and fatigue as measured by the functional assessment of chronic illness therapy (facit)-fatigue score. safety was comparable between pegcetacoplan and eculizumab in this study. seven of 41 patients (17%) in the pegcetacoplan group experienced a sae, and 6 of 39 patients (15%) in the eculizumab group experienced saes. no cases of meningitis and no deaths were reported in either treatment group.>