Apellis to Present Pivotal Paroxysmal Nocturnal Hemoglobinuria (PNH) Data at 25th EHA Annual Congress

Rhea-AI Summary

Apellis Pharmaceuticals announced positive results from its Phase 3 PEGASUS study, demonstrating that pegcetacoplan significantly improved hemoglobin levels in patients with paroxysmal nocturnal hemoglobinuria (PNH) compared to eculizumab. This pivotal study was selected for an oral presentation at the 25th European Hematology Association Congress, validating the innovative research and commitment to improve standards of care for PNH patients. Pegcetacoplan has received Fast Track designation from the FDA for treating PNH and is also being evaluated for other conditions.

• Results from positive Phase 3 PEGASUS study in PNH selected for oral presentation at the 2020 European Hematology Association Congress
• PEGASUS showed superiority of pegcetacoplan to eculizumab in improving hemoglobin levels as well as clinically meaningful improvements in key additional hematologic and clinical outcomes at 16 weeks

WALTHAM, Mass., May 14, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced that results from the Phase 3 PEGASUS study were selected for oral presentation at the 25^th Annual Congress of the European Hematology Association. The abstract has been published on the EHA website, and the oral presentation, featuring additional analysis of the 16-week PEGASUS data, will be available online during the virtual congress.

“We are excited to present our positive 16-week data from the Phase 3 PEGASUS study at the EHA Annual Congress,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. “The recognition of our pivotal study by EHA with an oral presentation validates years of innovative research and underscores our commitment to elevating the standard of care for people living with PNH.”

Featured Abstract
Results of the PEGASUS Phase III Randomized Trial Demonstrating Superiority of the C3 Inhibitor, Pegcetacoplan, Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (S192). The presentation will be made available on-demand starting on Friday, June 12, 8:30 CET.

About Pegcetacoplan (APL-2)
Pegcetacoplan is an investigational, targeted C3 inhibitor designed to regulate excessive complement activation, 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. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), cold agglutinin disease, and C3 glomerulopathy. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit www.apellis.com/clinical-trials.html.¹

About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is a rare, chronic, life-threatening blood disorder associated with abnormally low hemoglobin levels due to the destruction of oxygen-carrying red blood cells (hemolysis). Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue and difficulty breathing (dyspnea). Retrospective studies show that, even on eculizumab, approximately 70% of people with PNH have low hemoglobin levels,^1,2 and 36% require one or more transfusions a year.²

About Apellis
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. By pioneering targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. 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 relating to the anticipated closing date. 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 the uncertainties related to market conditions and the completion of the private placement on the anticipated terms or at all, the uncertainties related to the satisfaction of closing conditions for the sale of the notes, the other factors discussed in the “Risk Factors” section of Apellis’ Quarterly Report on Form 10-Q filed with the SEC on April 29, 2020 and the risks described in other filings that Apellis may make with the SEC. 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.

Media Contact:
Mark Dole
media@apellis.com
617.997.3484

Investor Contact:
Sam Martin / Maghan Meyers
Argot Partners
sam@argotpartners.com / maghan@argotpartners.com
212.600.1902

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1.      Risitano AM, Notaro R, Marando L, et al. (2009) Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab. Blood. 2009 Apr 23;113(17):4094-100. 
2.      McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.

FAQ

What were the results of the PEGASUS study for APLS?

The PEGASUS study showed that pegcetacoplan significantly improved hemoglobin levels in PNH patients compared to eculizumab.

When were the PEGASUS study results presented?

The results were selected for oral presentation at the 25th European Hematology Association Congress, held in June 2020.

What is pegcetacoplan and its relevance to APLS?

Pegcetacoplan is an investigational C3 inhibitor designed for PNH treatment, showing promising clinical efficacy in the PEGASUS study.

Has pegcetacoplan received any FDA designations?

Yes, pegcetacoplan has been granted Fast Track designation by the FDA for the treatment of paroxysmal nocturnal hemoglobinuria.

What is the significance of the PEGASUS study for APLS?

The PEGASUS study is pivotal as it validates years of research and aims to elevate the standard of care for patients with PNH.