Apellis and Sobi Announce EU Approval of Aspaveli® (pegcetacoplan) for Treatment of PNH

Rhea-AI Summary

Apellis Pharmaceuticals announced that the European Commission has approved Aspaveli (pegcetacoplan), the first targeted C3 therapy for paroxysmal nocturnal hemoglobinuria (PNH), aimed at adults who remain anemic after C5 inhibitor treatment. This approval is based on the PEGASUS Phase 3 study showing Aspaveli's effectiveness over eculizumab in improving hemoglobin levels. Aspaveli will enjoy market exclusivity in the EU due to its orphan drug designation, supporting its potential to enhance patient care for PNH.

Positive

• Aspaveli approved as the first targeted C3 therapy in the EU.
• Demonstrated superiority to eculizumab in improving hemoglobin levels.
• Will have market exclusivity for PNH due to orphan drug designation.

Negative

• None.

• The first targeted C3 therapy approved in the EU

• Approval based on results from head-to-head PEGASUS Phase 3 study where Aspaveli demonstrated superiority to eculizumab in improving hemoglobin levels¹

• Aspaveli will have market exclusivity for paroxysmal nocturnal hemoglobinuria (PNH) based on orphan drug designation
  

WALTHAM, Mass. and STOCKHOLM, Sweden, Dec. 15, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) announced today that the European Commission (EC) has approved Aspaveli^® (pegcetacoplan), the first and only targeted C3 therapy, for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who are anemic after treatment with a C5 inhibitor for at least three months. Based on the recommendation from the European Medicines Agency’s Committee for Orphan Medicinal Products, the EC determined that pegcetacoplan continues to meet the criteria for the orphan drug designation status granted in 2017 for the treatment of PNH.

PNH is a rare, chronic and life-threatening blood disorder where uncontrolled complement activation leads to the destruction of oxygen-carrying red blood cells through intravascular hemolysis and extravascular hemolysis. Characterized by persistently low hemoglobin, PNH can result in frequent transfusions and debilitating symptoms such as severe fatigue caused by anemia. Despite improvements in hemolytic activity with C5 inhibitor treatment, approximately 72% of people with PNH treated with C5 inhibitors remain anemic, according to a retrospective and a cross-sectional study.^2,3

“The European Commission’s approval of Aspaveli is a milestone for people living with PNH across Europe,” said Guido Oelkers, chief executive officer and president at Sobi. “The symptoms of PNH can significantly impact quality of life. In addition, despite current therapy, many people still require frequent blood transfusions. We are now working with EU member states to provide access to this important medicine as quickly as possible.”

“As the first and only targeted C3 therapy in Europe, Aspaveli has the potential to elevate the standard of care for patients living with PNH,” said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. “Today's approval represents the first new class of complement medicines in Europe in over a decade, building on the launch of this important treatment in the United States.”

The approval is based on the results from the head-to-head PEGASUS Phase 3 study, which evaluated the efficacy and safety of Aspaveli compared to eculizumab at 16 weeks in adults with PNH who had persistent anaemia despite treatment with eculizumab. The full safety and efficacy results were published in The New England Journal of Medicine in March 2021.¹

Orphan drug designation is granted to therapies that treat a serious disease that affects fewer than five in 10,000 people in the EU and provide a significant benefit over existing treatments. Aspaveli will have market exclusivity based on orphan drug designation for PNH.

About Aspaveli^®/Empaveli™
Aspaveli^®/Empaveli™ (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. Aspaveli is approved in the EU as an orphan drug for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who are anemic after treatment with a C5 inhibitor for at least three months and in the United States as EMPAVELI for the treatment of adults with PNH. The therapy is also under investigation for several other rare diseases across hematology, nephrology, and neurology.

About the Sobi and Apellis Collaboration
Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-US commercialization rights for systemic pegcetacoplan, and Apellis has exclusive US commercialization rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy (GA).

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 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.

About Sobi™
Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across Europe, North America, Middle East, and Asia. In 2020, Sobi's revenues amounted to SEK 15.3 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube.

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 implications of clinical data. 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 and other factors discussed in the “Risk Factors” section of Apellis’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 8, 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.

Contacts:

Apellis

Media:
Lissa Pavluk 
media@apellis.com 
617.977.6764

Investors: 
Meredith Kaya
meredith.kaya@apellis.com
617.599.8178

Sobi

To contact the Sobi Investor Relations Team, click here. For Sobi Media contacts, click here.

^References

1. ^{Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. DOI: 10.1056/NEJMoa2029073.}
2. ^{McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.}
3. ^{Dingli ASH 2020 Abstract/ p.1/ Methods/ ln.1-2; p.2/ Results/ln.7-9; ln.14-1} 

FAQ

What is Aspaveli and its significance for PNH patients?

Aspaveli (pegcetacoplan) is the first targeted C3 therapy approved in the EU for PNH, aimed at patients who are anemic despite C5 inhibitor treatments.

What study supported the approval of Aspaveli?

The European Commission's approval was based on the results from the PEGASUS Phase 3 study, which showed Aspaveli's superiority over eculizumab.

How does Aspaveli compare to eculizumab?

Aspaveli demonstrated improved hemoglobin levels compared to eculizumab in adults with PNH in the PEGASUS Phase 3 study.

What market exclusivity does Aspaveli have?

Aspaveli will have market exclusivity in the EU for treating paroxysmal nocturnal hemoglobinuria based on its orphan drug designation.

What is the potential patient impact of Aspaveli's approval?

Aspaveli's approval is expected to significantly enhance the quality of life for PNH patients, many of whom require frequent blood transfusions.