APLS - Apellis Sobi's pegcetacoplan shows positive action in rare blood disorder

Apellis Pharmaceuticals (APLS) and Sobi Swedish Orphan Biovitrum AB (BIOVF) announce positive top-line results at Week 48 from the Phase 3 PEGASUS study, which demonstrated sustained hematological and clinical improvements in patients with paroxysmal nocturnal hemoglobinuria ((PNH)) who were treated with pegcetacoplan, an investigational, targeted C3 therapy.The primary objective of this study was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. At Week 48, hemoglobin increases were sustained in pegcetacoplan-treated patients with a mean improvement from baseline of 2.7 g/dL, same as the one seen at Week 16.Additionally, eculizumab-treated patients who switched to pegcetacoplan experienced sustained improvements in hemoglobin and other hematological and clinical measures. Throughout the 48-week study, 73% of patients treated with pegcetacoplan remained transfusion free.Improvements across additional markers of disease, such as reticulocyte count, lactate dehydrogenase levels, and the Functional Assessment of Chronic Illness Therapy fatigue scores, were maintained. Overall, the safety profile of

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Apellis, Sobi's pegcetacoplan shows positive action in rare blood disorder