Phase III PEGASUS study of APL 2 shows consistent impact in paroxysmal nocturnal hemoglobinuria.- Apellis Pharma

Apellis Pharmaceuticals announced results from its Phase III PEGASUS study of APL 2 (pegcetacoplan) in adults with paroxysmal nocturnal hemoglobinuria (PNH). Results showed that in patients with no or low transfusion requirements, pegcetacoplan was associated with an adjusted mean Hb increase of 2.97 g/dL versus Soliris (eculizumab), which had a mean change of -0.01 g/dL from the 8.9 g/dL baseline. In patients with high transfusion requirements, pegcetacoplan had an adjusted mean Hb increase of 2.11 g/dL, while Soliris treatment showed a mean change of -4.02 g/dL from the 8.5 g/dL baseline. Overall, 85% of pegcetacoplan-treated patients were transfusion-free over 16 weeks, compared with 15% for Soliris-treated patients. Among patients with high transfusion requirements, 86% of patients in the pegcetacoplan group were transfusion-free compared to 4% for Soliris, while the rates for those with low or no transfusion requirements, the rates were 85% and 31%, respectively. The findings were presented at the congress of the European Hematology Association (EHA). Apellis Pharma plans to file for approval for PNH in the US and Europe in the second half of 2020.PEGASUS included 80 adults with PNH who had been taking Soliris and were stable for at least three months, with a haemoglobin (Hb) level of less than 10.5 g/dL at screening. After four-weeks, during which patients were given pegcetacoplan twice weekly in addition to Soliris, subjects were randomised to receive either pegcetacoplan twice weekly or the current dose of Soliris.