EXCLUSIVE: Jess Ratcliffe, 31, from Surrey, was diagnosed with paroxysmal nocturnal haemoglobinuria (PNH) in 2016, which causes red blood cells to break down.
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STOCKHOLM and WALTHAM, Mass., Dec. 10, 2020 /PRNewswire/ Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) and Apellis Pharmaceuticals Inc. (Nasdaq: APLS) today announced positive top-line results at week 48 from the phase 3 PEGASUS study, which demonstrated sustained haematological and clinical improvements in patients with paroxysmal nocturnal haemoglobinuria (PNH) who were treated with pegcetacoplan, an investigational, targeted C3 therapy. The safety profile of pegcetacoplan was consistent with previously reported data and no new safety signals were identified.
All patients (n=77) who completed the 16-week randomized controlled period of the PEGASUS study, which evaluated pegcetacoplan compared to eculizumab, entered the open-label period and received pegcetacoplan from week 17 to week 48.