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Seven accepted abstracts, including an oral presentation, reinforce the potential of pegcetacoplan, an investigational, targeted C3 therapy, to redefine treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH)
PDUFA action date of May 14, 2021 set by the U.S. Food and Drug Administration (FDA)
WALTHAM, Mass., May 12, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that seven abstracts were accepted for presentation and publication at the European Hematology Association (EHA) Virtual Congress to be held June 9-17, 2021. Data support the long-term durability and consistent safety profile of pegcetacoplan, an investigational, targeted C3 therapy, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
“The breadth of data presented at EHA add to a growing body of evidence that reinforces the potential of pegcetacoplan to redefine treatment for the PNH community,” said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis.
Marketing applications for pegcetacoplan for the treatment of PNH are under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA granted the application Priority Review designation and set a target action date of May 14, 2021. The application has the potential for a European Commission decision in the second half of 2021.
Pegcetacoplan Oral Presentation:
Forty-eight week efficacy and safety of pegcetacoplan in adult patients with paroxysmal nocturnal hemoglobinuria and suboptimal response to prior eculizumab treatment – S174 – Friday, June 11 at 9:00 CEST
Pegcetacoplan E-Poster Presentation:
Effect of pegcetacoplan on quality of life in patients with paroxysmal nocturnal hemoglobinuria: week 48 of PEGASUS phase 3 trial comparing pegcetacoplan to eculizumab – EP595 – Friday, June 11 at 9:00 CEST
Paroxysmal nocturnal hemoglobinuria’s humanistic and economic burden in patients receiving C5 inhibitors in Europe – EP1191 – Friday, June 11 at 9:00 CEST
Categorized hematologic response to pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal hemoglobinuria: post hoc analysis of PEGASUS phase 3 randomized trial data
Comparative effectiveness of pegcetacoplan versus ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: a matching-adjusted indirect comparison using 26 week PEGASUS phase 3 trial data
Injection-site reactions at week 48 in the randomized phase 3 PEGASUS trial of pegcetacoplan compared with eculizumab for individuals with paroxysmal nocturnal hemoglobinuria
Long-term effects in subgroups of patients with paroxysmal nocturnal hemoglobinuria treated with pegcetacoplan versus eculizumab: 48-week analysis of PEGASUS phase 3 trial
About the PEGASUS Study
The PEGASUS study (APL2-302; NCT03500549) was a multi-center, randomized, active-comparator controlled 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 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 pegcetacoplan 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 pegcetacoplan twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period entered the open-label pegcetacoplan treatment period.
About Pegcetacoplan (APL-2)
Pegcetacoplan is an investigational, 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. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan is being evaluated in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was granted Fast Track designation by the FDA for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3G by the FDA and EMA. For additional information regarding pegcetacoplan clinical studies, visit https://apellis.com/our-science/clinical-trials.
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening blood disorder caused by an acquired mutation which leads to uncontrolled complement activation and the destruction of red blood cells through hemolysis. A retrospective analysis shows that, even on eculizumab, approximately 72% of people with PNH have anemia, a key indicator of ongoing hemolysis.1 The analysis also finds that 36% of patients require one or more transfusions a year and 16% require three or more.1
About the Apellis and Sobi Collaboration
Apellis and Sobi entered a collaboration to develop and commercialize systemic pegcetacoplan in October 2020. The companies have global co-development rights for systemic pegcetacoplan. Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy (GA). Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan.
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.
Apellis Forward-Looking Statement
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements in respect of the expected closing of the exchanges. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether the conditions for the closing of the exchanges will be satisfied and other factors discussed in the “Risk Factors” section of Apellis’ Quarterly Report on Form 10-Q with the Securities and Exchange Commission on April 28, 2021 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
1. McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.