APLS - Apellis reports positive Empaveli data from late-stage study in rare blood disorder

Apellis Pharmaceuticals (APLS) and Swedish Orphan Biovitrum AB (BIOVF) report positive top-line results from the Phase 3 PRINCE study evaluating the efficacy and safety of Empaveli (pegcetacoplan) in adults with paroxysmal nocturnal hemoglobinuria ((PNH)) who are treatment naïve. At week 26, 86% of Empaveli-treated patients achieved hemoglobin stabilization compared to 0% of patients on standard of care (p<0.0001).Mean lactate dehydrogenase ((LDH)) in the Empaveli group decreased by 90% from a baseline of 2151 U/L to 211 U/L, compared to a 14% reduction on standard of care from a baseline of 1946 U/L to 1681 U/L (p<0.0001). Empaveli also achieved statistical superiority on several secondary endpoints.Mean hemoglobin levels in Empaveli group increased from 9.4 g/dL to 12.1 g/dL compared to an increase from 8.7 g/dL to 9.4 g/dL on standard of care (p=0.0019).91% of patients on Empaveli were transfusion free compared to 22% on standard of care (p<0.0001). The safety profile

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Apellis reports positive Empaveli data from late-stage study in rare blood disorder