Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today announced that results from the long-term extension study of EMPAVELI (pegcetacoplan) in adults with paroxysmal nocturnal hemoglobinuria (PNH) will be presented at the American Society of Hematology (ASH) Annual Meeting being held Dec. 10-13 in New Orleans, Louisiana.

The data show that EMPAVELI, the first and only targeted C3 therapy, demonstrated robust and sustained improvements in key markers of disease across a broad population of PNH patients for up to two years.

'These results highlight the meaningful, long-term improvements that treatment with EMPAVELI can make in the lives of PNH patients, regardless of their severity of disease or prior treatment,' said Peter Hillmen, M.B. Ch.B., Ph.D., head of hematology engagement at Apellis. 'EMPAVELI continues to demonstrate its potential to become a new standard of care for adults living with PNH, including the ability to achieve normalization across key markers of disease.'

The new results reinforce the positive efficacy and safety of EMPAVELI in both treatment-naive patients and patients who had previously been treated with eculizumab, a C5 inhibitor, across all five prior PNH clinical studies. After being treated with EMPAVELI for up to two years, mean hemoglobin was sustained at near-normal levels at 11.6 g/dl, 83% of patients were transfusion free, and 73% of patients had normalized levels of lactate dehydrogenase (LDH). Long-term safety data were consistent with previous clinical study results, with no thrombotic events or meningitis infections reported. The most common adverse events (AEs) were hemolysis (17%) and injection site reactions (11%).

Presentation details include:

Long-Term Safety and Efficacy of Pegcetacoplan Treatment in Adults with Paroxysmal Nocturnal Hemoglobinuria- 1248 - Dec. 10, 5:30 p.m. - 7:30 p.m. ET

About the Long-Term Efficacy and Safety Extension (APL2-307) Study

The APL2-307 study was a nonrandomized, open-label, multicenter 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 naive to complement inhibitors. During the 48-week trial period, 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 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, Australia, and Saudi Arabia as EMPAVELI and in the European Union and the United Kingdom as Aspaveli. The therapy is also under investigation for several other rare diseases across hematology, nephrology, and neurology.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)

PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria and difficulty breathing (dyspnea).

About the Apellis and Sobi Collaboration

Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.

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. Leaders in complement, we ushered in the first new class of complement medicine in 15 years with the approval of the first and only targeted C3 therapy. We are advancing this science to continually develop transformative medicines for people living with rare, retinal, and neurological diseases.

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. 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 discussed in the 'Risk Factors' section of Apellis' Annual Report on Form 10-K with the Securities and Exchange Commission on February 28, 2022 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.

Contact:

Jennifer Henesey

Tel: 339.223.0962

Email: media@apellis.com

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