Pegcetacoplan is a Synthetic Peptide owned by Apellis Pharmaceuticals, and is involved in 35 clinical trials, of which 21 were completed, 11 are ongoing, and 3 are planned.

Pegcetacoplan (APL-2) acts as complement factor C3 (C3) inhibitor. APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer that binds specifically to C3 and C3b. Complement 3 is a protein of the immune system that plays a key role in the complement system and contributes to innate immunity. The drug candidate acts as disease-modifying agent for PNH and AMD. Complement activation leads to formation of a structure called the membrane attack complex, which disrupts the membranes of target cells, leading to cell dysfunction and death. The therapeutic candidate binds tightly to complement component C3, preventing its participation in the complement activation cascade. Inhibition of complement C3, C3a anaphylatoxin, a mediator of local inflammatory process checks the disease progression.

The revenue for Pegcetacoplan is expected to reach a total of $6bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Pegcetacoplan NPV Report.

Pegcetacoplan was originated by University of Pennsylvania and is currently owned by Apellis Pharmaceuticals. Swedish Orphan Biovitrum is the other company associated in development or marketing of Pegcetacoplan.

Pegcetacoplan Overview

Pegcetacoplan (Empaveli, Aspaveli) is a synthetic peptide acts as a complement inhibitor. It is formulated as injectable solution for subcutaneous route of administration. Empaveli is indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), Aspaveli is indicated in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least 3 months.

Pegcetacoplan (APL-2) is under development for the treatment of post-transplant recurrence of C3G or IC-MPGN, transplant-associated thrombotic microangiopathy (TA-TMA), IgA nephropathy (Berger's disease), lupus nephritis, primary membranous nephropathy, or C3 glomerulopathy (Kidney Disease (Nephropathy)) includes C3 glomerulonephritis, paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (advanced form of dry age-related macular degeneration) and autoimmune hemolytic anemia or cold agglutinin disease(wAIHA) and amyotrophic lateral sclerosis. It is a  synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer. It is administered subcutaneously as an infusion for PNH and intravitreally for AMD. It is a compstatin derivative and a long-acting small cyclic peptide that targets complement factor C3. It was also under development for the treatment of kidney transplant rejection, refractory myasthenia gravis and neuromyelitis optica, neovascular age-related macular degeneration (AMD).

Swedish Orphan Biovitrum Overview

Swedish Orphan Biovitrum (Sobi) is an integrated biopharmaceutical company. It focuses on the development of products for the treatment of a few rare diseases. The company specializes in biotechnology with prime capabilities in protein biochemistry and biologics manufacturing. Its product portfolio focuses on hemophilia, immunology, specialty care, inflammation and genetic and metabolic diseases. The company also provides innovative treatment for conditions such as amyotrophic lateral sclerosis (ALS) and Hemophilia A. Sobi also manufactures and markets specialty products for rare diseases for partner companies. The company has operational presence in Europe, the Middle East, North America, Russia, and Asia. Sobi is headquartered in Stockholm, Sweden.

The company reported revenues of (Swedish Krona) SEK15,529 million for the fiscal year ended December 2021 (FY2021), an increase of 1.8% over FY2020. In FY2021, the company’s operating margin was 24%, compared to an operating margin of 31.6% in FY2020. In FY2021, the company recorded a net margin of 17.3%, compared to a net margin of 21.3% in FY2020. The company reported revenues of SEK3,999 million for the third quarter ended September 2022, an increase of 3.2% over the previous quarter.

Quick View – Pegcetacoplan

Report Segments
  • Innovator (NME)
Drug Name
  • Pegcetacoplan
Administration Pathway
  • Intravitreal
  • Subcutaneous
Therapeutic Areas
  • Cardiovascular
  • Central Nervous System
  • Genito Urinary System And Sex Hormones
  • Hematological Disorders
  • Immunology
  • Ophthalmology
Key Companies
Highest Development Stage
  • Marketed

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.