Tag: APLS

Apellis update in ph 3 study of Pegcetacoplan in Treatment-Naïve Patients with PNH

Apellis Pharmaceuticals, Inc. () completes enrollment in the global phase 3 PRINCE study evaluating pegcetacoplan (APL-2) in treatment-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH), meaning they had not received a complement inhibitor within three months before entering the trial. The trial is designed to further establish the potential of pegcetacoplan, a targeted C3 therapy, in all patients living with PNH. Top-line results are expected in early 2021.
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The PRINCE study is a phase 3, randomized, multicenter, open-label, controlled trial. The study was designed to evaluate the efficacy of pegcetacoplan in 48 adult patients who are treatment-naïve, showed evidence of hemolysis (elevated LDH ≥ 1.5x ULN) and had hemoglobin levels that were less than the lower limit of normal at the time of their screening. Primary outcome measures, to be evaluated after 26 weeks of treatment, include hemoglobin stabilization in the absence of transfusion and reduction in lactate dehydrogenase (LDH) level. A total of 53 patients were enrolled in the study.

Based on positive results from the pivotal PEGASUS study of pegcetacoplan in PNH, the company plans to submit marketing applications for pegcetacoplan for the treatment of PNH both in the United States and the European Union in the second half of 2020.


Press release

Apellis to submit NDA for Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria (PNH) in 2H-2020

Apellis Pharmaceuticals () to submit NDA for pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in 2H-2020. Based on feedback from the U.S. FDA during a pre-NDA meeting to discuss the registration strategy for pegcetacoplan, Apellis plans to submit the NDA with results from the Phase 3 PEGASUS trial comparing pegcetacoplan to eculizumab in patients with PNH. PNH is a rare blood disease in which the immune system destroys red blood cells.
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The PEGASUS study met its primary endpoint, demonstrating the superiority of pegcetacoplan to eculizumab with a statistically significant improvement in hemoglobin levels at 16 weeks. Top-line results also showed that the safety profile of pegcetacoplan was comparable to eculizumab in the study. As previously announced, results from the PEGASUS study have been selected for an oral presentation at the 25th Congress of the European Hematology Association and will be available on June 12, 2020.

The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, open-label, active-comparator controlled Phase 3 study in 80 adults with PNH. The primary objective of this study was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Participants must have been on eculizumab (stable for at least 3 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 pegcetacoplan twice weekly in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of pegcetacoplan twice weekly or their current dose of eculizumab. All participants completing the randomized controlled period entered the open-label pegcetacoplan treatment period where they received pegcetacoplan, regardless of the prior treatment received in the randomized, controlled period.

https://investors.apellis.com/news-releases/news-release-details/apellis-announces-plans-submit-nda-pegcetacoplan-paroxysmal

https://seekingalpha.com/article/4316161-apellis-pharmaceuticals-phase-3-success-and-news-good-bad-and-ugly-of-biopharma