Apellis Pharmaceuticals (APLS) and Swedish Orphan Biovitrum Reports Positive Top-line Results from the Phase 3 PRINCE Study of EMPAVELI (pegcetacoplan)

Apellis Pharmaceuticals, Inc. (NASDAQ: APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today reported 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, meaning they had not received a complement inhibitor within three months before entering the study.

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 (p<0.0001). Hemoglobin stabilization was defined as an avoidance of a >1 g/dL decrease in hemoglobin levels in the absence of transfusions.
• Mean LDH in the EMPAVELI group decreased by 90% from a baseline of 2151 U/L [9.5x upper limit of normal (ULN)] to 211 U/L, which is within the normal range, compared to a 14% reduction on standard of care from a baseline of 1946 U/L (8.6x ULN) to 1681 U/L (7.4x ULN) (p<0.0001).

“The positive PRINCE data showed that EMPAVELI provided clinically meaningful improvements across multiple measures that are important for patients and build on our recent FDA approval of EMPAVELI in PNH,” said Federico Grossi, M.D., Ph.D., chief medical officer, Apellis. “Combined with previous studies, these results emphasize the potential of EMPAVELI to provide disease control for all adults with PNH regardless of prior treatment.”

EMPAVELI also achieved statistical superiority on several secondary endpoints, including improvements in hemoglobin levels and transfusion avoidance, compared to standard of care, which did not include complement inhibitors.

• Mean hemoglobin levels in the EMPAVELI group increased from 9.4 g/dL to 12.1 g/dL compared to an increase from a baseline of 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 of EMPAVELI was consistent with previous studies. At Week 26, 9% of patients in the EMPAVELI group experienced a serious adverse event (SAE) compared to 17% on standard of care. One death was reported in each group, and neither were related to treatment. No cases of meningitis or thrombosis were reported in either group. The most common adverse events reported during the study in the EMPAVELI and standard of care groups, respectively, were injection site reaction (30% vs. 0%), hypokalemia (13% vs.11%), and fever (9% vs. 0%).

“The PRINCE study results reinforce the efficacy and safety profile of EMPAVELI in PNH,” said Ravi Rao, head of research and development and chief medical officer at Sobi. “Our hope is to contribute to an improvement of care and to make a difference in the lives of people with this rare blood disease.”

Detailed results from the PRINCE study will be presented at medical congresses.

About the PRINCE StudyThe PRINCE study (NCT04085601) is a 2:1 (EMPAVELI: standard of care) randomized, multi-center, open-label, controlled Phase 3 study in 53 treatment-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of EMPAVELI™ (pegcetacoplan) in patients who have not received treatment with any complement inhibitor within three months prior to screening. During the 26-week randomized, controlled period, patients received either 1080 mg of EMPAVELI twice weekly or standard of care therapy, which did not include complement inhibitors. Patients in the standard of care group had the option to escape to the EMPAVELI group if their hemoglobin decreased 2 g/dL or more from their baseline value.

The study was conducted in collaboration with SFJ Pharmaceuticals, who supported the development of EMPAVELI in PNH. SFJ is a global drug development company, which provides a unique and highly customized co-development partnering model for the world’s top pharmaceutical and biotechnology companies.

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