Benzinga | May 25, 2021 08:21AM EDT

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

* Apellis Pharmaceuticals Inc (NASDAQ: APLS) and Swedish Orphan Biovitrum AB (SOBI) have reported positive top-line results from the Phase 3 PRINCE study evaluating Empaveli (pegcetacoplan) in treatment na?ve 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.