Apellis Pharmaceuticals, Inc. and Sobi presented post hoc data that reinforce the long-term efficacy and safety of EMPAVELI® (pegcetacoplan) in adults with paroxysmal nocturnal hemoglobinuria (PNH) for up to three years. The data were reported during an oral presentation at the American Society of Hematology (ASH) Annual Meeting. The analysis integrated data across the Phase 3 PEGASUS and PRINCE studies and the long-term extension study.

After starting treatment with EMPAVELI, key markers of disease rapidly improved and were sustained in both treatment-naïve patients and patients previously treated with eculizumab. Improvements in hemoglobin reached normal or near-normal levels, and mean lactate dehydrogenase (LDH) was maintained below the upper limit of normal. Additionally, 67% of treatment-naïve patients from PRINCE were transfusion free for up to 2.5 years, and 52% of patients from PEGASUS remained transfusion free for up to 3 years.

Less than 25% of patients were transfusion free in the year prior to entering the PRINCE and PEGASUS studies. The safety profile was consistent with previous clinical study results, and no new or unexpected safety findings were identified. Approximately 4.5% of patients experienced a serious adverse event deemed related to treatment with pegcetacoplan.

No meningococcal infections were reported. The prescribing information for EMPAVELI contains a boxed warning, which states that EMPAVELI may increase the risk of meningococcal and other serious infections caused by encapsulated bacteria that may become rapidly life threatening or fatal if not recognized and treated early. About the Long-Term Efficacy and Safety Extension (APL2-307) Study: The APL2-307 study was a nonrandomized, multicenter, open-label Phase 3 extension study of 137 adults with paroxysmal nocturnal hemoglobinuria (PNH) who completed previous EMPAVELI®/Aspaveli® (pegcetacoplan) Phase 1 (PHAROAH, PADDOCK), Phase 2 (PALOMINO), and Phase 3 (PEGASUS, PRINCE) clinical trials.

Patients in these studies were either anemic despite eculizumab treatment or were naïve to complement inhibitors. During the trial, patients continued to receive 1080 mg of EMPAVELI twice weekly or once every three days (PEGASUS, PRINCE) or switched to 1080 mg of EMPAVELI twice weekly (PHAROAH, PADDOCK, PALOMINO). The primary objective was to establish the long-term efficacy and safety of EMPAVELI.

About the PEGASUS Study: The PEGASUS study (APL2-302; NCT03500549) was a randomized, multi-center, head-to-head Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of EMPAVELI®/Aspaveli® (pegcetacoplan) compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit.

During the four-week run-in, patients were dosed with 1080 mg of EMPAVELI twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of EMPAVELI twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period (n=77) opted to enter the open-label EMPAVELI treatment period.

About the PRINCE Study: The PRINCE study (NCT04085601) was a 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®/Aspaveli® (pegcetacoplan) in patients who had 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 by 2 g/dL or more from their baseline value. About EMPAVELI®/Aspaveli® (pegcetacoplan): EMPAVELI®/Aspaveli® (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body?s immune system, which can lead to the onset and progression of many serious diseases. It is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the United States, European Union, and other countries globally.

The therapy is also under investigation for several other rare diseases across hematology and nephrology.