Apellis-Sobi Partnered Pegcetacoplan Shows Clinically Meaningful Improvements in Rare Blood Disorder Study

Apellis Pharmaceuticals Inc APLS and Swedish Orphan Biovitrum AB (SOBI) have reported positive top-line results from the Phase 3 PRINCE study evaluating Empaveli (pegcetacoplan) in treatment naïve paroxysmal nocturnal hemoglobinuria (PNH) patients.
Empaveli 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.
86% of Empaveli-treated patients achieved hemoglobin stabilization compared to 0% of patients on standard of care.
Mean LDH in the Empaveli group decreased by 90% from a baseline, compared to 14%.
Mean hemoglobin levels in the 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 the standard of care.
91% of patients on Empaveli were transfusion-free compared to 22% on the standard of care.
On the safety front, 9% of patients in the Empaveli group experienced a serious adverse event compared to 17% on the standard of care.
One death was reported in each group, and neither was related to treatment.
Detailed results from the PRINCE study will be presented at medical congresses.
Empaveli is approved in the U.S. for PNH, a rare blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis.
Price Action: APLS shares are trading higher by 9.75% at $51.80 in the premarket session on the last check Tuesday.

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