Positive top-line results from Phase III PRINCE study of pegcetacoplan in PNH

Positive top-line results have been reported from the Phase III PRINCE study evaluating the efficacy and safety of pegcetacoplan in adults with paroxysmal nocturnal hemoglobinuria (PNH) who are treatment naïve, meaning they had not received a complement inhibitor within three months before entering the study.

The drug was originated by US biopharma company Apellis Pharmaceuticals (Nasdaq: APLS) and licensed to Swedish Orphan Biovitrum (STO: SOBI; also known as Sobi) for exclusive ex-US commercialization under a deal that provided a $250 million upfront payment plus eligibility for a further $1.25 billion for the US firm.

Pegcetacoplan, which was approved in the USA under the trade name Empaveli earlier this month and with a European decision expected shortly, demonstrated statistical superiority on the co-primary endpoints of hemoglobin stabilization and reduction in lactate dehydrogenase (LDH) compared to standard of care, which did not include complement inhibitors, at week 26.