Pegcetacoplan is under clinical development by Apellis Pharmaceuticals and currently in Phase III for Acquired (Autoimmune) Hemolytic Anemia. According to GlobalData, Phase III drugs for Acquired (Autoimmune) Hemolytic Anemia have a 62% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData’s report assesses how Pegcetacoplan’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

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).

Apellis Pharmaceuticals overview

Apellis Pharmaceuticals (Apellis), is a clinical-stage biopharmaceutical company that focuses on the discovery and development of therapeutic compounds for autoimmune and inflammatory diseases through inhibition of the complement system at the level of C3 (central protein). Its pipeline product portfolio includes APL-2, a C3 inhibitor administered by intravitreal injections and subcutaneous injections for the treatment of geographic atrophy (GA), paroxysmal nocturnal hemoglobinuria (PNH), wet age-related macular degeneration (AMD), autoimmune hemolytic anemia (AIHA) and complement-dependent nephropathies. Apellis complement system activates C3 protein which enables immune responses such as opsonization, inflammation and formation of the membrane attack complex. It also conducts research on chronic therapy with a C3 inhibitor into clinical trials. The company operates with offices in the US, Ireland, Switzerland and Australia. Apellis is headquartered in Waltham, Massachusetts, the US.

For a complete picture of Pegcetacoplan’s drug-specific PTSR and LoA scores, buy the report here.


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

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.