Apellis Pharmaceuticals is focused on advancing complement science to develop transformative therapies for a variety of debilitating diseases. Their lead product, pegcetacoplan (APL-2), is designed to control the complement system, which plays a crucial role in immune response and inflammation. The company has made significant strides in clinical research, with multiple ongoing and completed studies targeting conditions such as paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA) secondary to age-related macular degeneration, and various forms of glomerulonephritis. These studies aim to evaluate the safety, efficacy, and pharmacokinetics of pegcetacoplan, demonstrating Apellis's commitment to innovative treatment solutions.

The clinical trials conducted by Apellis are structured to assess the therapeutic potential of pegcetacoplan across different patient populations and disease states. For instance, the PIONEER study focuses on pediatric patients with PNH, while the DERBY and OAKS studies evaluate its effects on adults with GA. The company emphasizes patient involvement and community engagement in its research efforts, ensuring that the voices of those affected by these diseases are considered in the development of new therapies. Through these initiatives, Apellis Pharmaceuticals aims to challenge conventional treatment paradigms and provide hope for patients facing serious health challenges.

United States

Apellis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on the development of therapeutic compounds through the inhibition of the complement system for autoimmune and inflammatory diseases. Its lead product candidate is pegcetacoplan that is in Phase III clinical trials for the treatment of geographic atrophy in age-related macular degeneration and paroxysmal nocturnal hemoglobinuria diseases; and in Phase II clinical trials for the treatment of cold agglutinin disease, C3 glomerulopathy, IgA nephropathy, primary membranous nephropathy, and lupus nephritis. The company is also developing APL-9, which is in single ascending dose Phase I randomized, double-blind, and placebo-controlled clinical trials for the prevention of immune system activation coincident with adeno-associated virus for intravenous administration. Apellis Pharmaceuticals, Inc. has a strategic collaboration with Swedish Orphan Biovitrum AB (publ) for the advancement of pegcetacoplan, a C3 therapy for the treatment of multiple rare diseases, including paroxysmal nocturnal hemoglobinuria. The company was founded in 2009 and is based in Waltham, Massachusetts.

Central Nervous System (CNS)

Eye Diseases

Hematologic Diseases

Rare diseases

Various

Apellis Pharmaceuticals

Paroxysmal nocturnal hemoglobinuria

Pegcetacoplan is an investigational, targeted C3 therapy designed to &nbsp;regulate excessive activation of the complement cascade, part of the &nbsp;body’s immune system, which can lead to the onset and progression of &nbsp;many serious diseases. Pegcetacoplan&nbsp;is a synthetic cyclic peptide &nbsp;conjugated to a polyethylene glycol polymer that binds specifically to &nbsp;C3 and C3b. Apellis is evaluating&nbsp;pegcetacoplan&nbsp;in several clinical &nbsp;studies across hematology, ophthalmology, nephrology, and &nbsp;neurology.&nbsp;Pegcetacoplan&nbsp;was granted Fast Track designation by the U.S. &nbsp;Food and Drug Administration (FDA) for&nbsp;the treatment of paroxysmal &nbsp;nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, &nbsp;and received orphan drug designation for the treatment of C3G by the FDA &nbsp;and European Medicines Agency.&nbsp;
<a href="https://investors.apellis.com/news-releases/news-release-details/apellis-announces-fda-acceptance-and-priority-review-new-drug">Source</a>

Empaveli (systemic pegcetacoplan) (APL-2)

Age-related macular degeneration

Geographic atrophy

Pegcetacoplan is an investigational, targeted C3 therapy designed to &nbsp;regulate excessive activation of the complement cascade, part of the &nbsp;body’s immune system, which can lead to the onset and progression of &nbsp;many serious diseases. Pegcetacoplan is a synthetic cyclic peptide &nbsp;conjugated to a polyethylene glycol polymer that binds specifically to &nbsp;C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical &nbsp;studies across hematology, ophthalmology, nephrology, and &nbsp;neurology. Pegcetacoplan was granted Fast Track designation by the U.S. &nbsp;Food and Drug Administration (FDA) for the treatment of paroxysmal &nbsp;nocturnal hemoglobinuria (PNH) and the treatment of geographic&nbsp;atrophy, &nbsp;and&nbsp;received orphan drug designation for the treatment of C3 &nbsp;glomerulopathy (C3G) by the FDA and European Medicines Agency.
<a href="https://investors.apellis.com/news-releases/news-release-details/apellis-announces-late-breaking-presentation-largest">Source</a>

SYFOVRE™ (Intravitreal pegcetacoplan)

C3 Glomerulopathy

Immune complex membranoproliferative glomerulonephritis

Pegcetacoplan is an investigational, targeted C3 therapy designed to &nbsp;regulate excessive activation of the complement cascade, part of the &nbsp;body’s immune system, which can lead to the onset and progression of &nbsp;many serious diseases. Pegcetacoplan&nbsp;is a synthetic cyclic peptide &nbsp;conjugated to a polyethylene glycol polymer that binds specifically to &nbsp;C3 and C3b. Apellis is evaluating&nbsp;pegcetacoplan&nbsp;in several clinical &nbsp;studies across hematology, ophthalmology, nephrology, and &nbsp;neurology.&nbsp;Pegcetacoplan&nbsp;was granted Fast Track designation by the U.S. &nbsp;Food and Drug Administration (FDA) for&nbsp;the treatment of paroxysmal &nbsp;nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, &nbsp;and received orphan drug designation for the treatment of C3G by the FDA &nbsp;and European Medicines Agency.&nbsp;
<a href="https://investors.apellis.com/news-releases/news-release-details/apellis-announces-fda-acceptance-and-priority-review-new-drug">Source</a>

Systemic pegcetacoplan (APL-2)

Hematopoietic stem cell transplantation thrombotic microangiopathies

Autoimmune disease

Inflammatory disease

 APL-3007 + SYFOVRE

Acute respiratory distress syndrome

APL-9 is an investigational, targeted C3 therapy designed to control the &nbsp;complement cascade centrally and may have the potential to treat a &nbsp;range of diseases caused by excessive activation of complement. APL-9 &nbsp;leverages the same mechanism of action as Apellis’ lead compound, &nbsp;pegcetacoplan, but has a lower molecular weight and shorter half-life. &nbsp;APL-9 is designed to be intravenously administered for acute use.
<a href="https://investors.apellis.com/news-releases/news-release-details/apellis-pharmaceuticals-reports-third-quarter-2020-financial">Source</a>

Apellis Pharmaceuticals

Company Details