Apellis Announces Plans to Submit NDA for Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria (PNH)

• Submission of New Drug Application (NDA) planned for second half of 2020 following successful pre-NDA meeting with FDA

WALTHAM, Mass., May 21, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced plans to submit a New Drug Application (NDA) for pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the second half of 2020. Based on feedback from the U.S. Food and Drug Administration (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.  

“We are pleased with the successful outcome of the pre-NDA meeting and committed to working closely with the FDA and other regulatory authorities to bring pegcetacoplan to the PNH community,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. “Based on results from our Phase 3 PEGASUS study, we are excited about the potential for pegcetacoplan to redefine treatment for people living with PNH.”

Regulatory discussions are also ongoing in the EU, and Apellis plans to meet with European regulatory agencies in the second quarter of 2020 to discuss next steps for submission of a marketing authorization application for pegcetacoplan in PNH.

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 25^th Congress of the European Hematology Association and will be available on June 12, 2020.

About the PEGASUS Study
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.