Pegcetacoplan

Summary

Pegcetacoplan (CHEMBL4298211) is an approved protein (ATC L04AJ03); indicated across 9 conditions including eye disorder and autoimmune hemolytic anemia.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Protein
• ATC class: L04AJ03 (+1 more)
• Indications: 9 conditions
• Clinical trials: 32

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4298211
Name	Pegcetacoplan
Type	Protein
Max phase	4
ATC	L04AJ03, S01XA31

Also known as: APL-2, Aspaveli, Empaveli, Pegcetacoplan, Syfovre, PEGCETACOPLAN

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

9 indications (3 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
eye disorder	4	MONDO:0005328	EFO:0003966
autoimmune hemolytic anemia	3	MONDO:0020108	MONDO:0018922
amyotrophic lateral sclerosis	2	MONDO:0004976	MONDO:0004976
membranoproliferative glomerulonephritis	2	MONDO:0002461	MONDO:0019736
thrombotic microangiopathy	2	MONDO:0019737	MONDO:0019737
age-related macular degeneration	1	MONDO:0005150	EFO:0001365

3 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 32.

Phase distribution

Phase	Trials
PHASE3	11
PHASE2	11
PHASE1	4
PHASE1/PHASE2	3
Not specified	3

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03531255	PHASE3	ACTIVE_NOT_RECRUITING	Pegcetacoplan Long Term Safety and Efficacy Extension Study
NCT05809531	PHASE3	ACTIVE_NOT_RECRUITING	An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Participants With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
NCT07020832	PHASE3	RECRUITING	A Phase 3 Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults at High Risk of Delayed Graft Function (DGF) Following Kidney Allograft Transplantation
NCT03500549	PHASE3	COMPLETED	Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT03525600	PHASE3	COMPLETED	Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration
NCT03525613	PHASE3	COMPLETED	A Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration
NCT04085601	PHASE3	COMPLETED	A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
NCT04770545	PHASE3	COMPLETED	An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic Atrophy Secondary to AMD
NCT05067127	PHASE3	COMPLETED	Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
NCT05096403	PHASE3	COMPLETED	A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)
NCT07214740	PHASE3	COMPLETED	Study to Evaluate a Pegcetacoplan (Syfovre®) Prefilled Syringe
NCT04901936	PHASE2	RECRUITING	A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT04919629	PHASE2	RECRUITING	APL-2 and Pembrolizumab Versus APL-2, Pembrolizumab and Bevacizumab Versus Bevacizumab Alone for the Treatment of Recurrent Ovarian, Fallopian Tube, or Primary Peritoneal Cancer and Malignant Effusion
NCT06722157	PHASE2	ACTIVE_NOT_RECRUITING	A Study to Test Whether BI 771716 Helps People With an Advanced Form of Age-related Macular Degeneration (AMD) Called Geographic Atrophy
NCT07160179	PHASE1/PHASE2	RECRUITING	Study to Assess the Adverse Events and How Intravitreal ABBV-6628 Moves Through the Body of Adult Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration
NCT07214298	PHASE1/PHASE2	RECRUITING	Pegcetacoplan in Combination With Modified FOLFIRINOX for the Treatment of Metastatic Pancreatic Ductal Adenocarcinoma
NCT07215390	PHASE2	RECRUITING	A Phase 2, Randomized, Placebo Controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Multidose APL 3007 in Combination With Syfovre/Pegcetacoplan (APL-2) in Patients Diagnosed With Geographic Atrophy Secondary to Age Related Macular Degeneratio
NCT02503332	PHASE2	COMPLETED	Study of Pegcetacoplan (APL-2) Therapy in Patients With Geographic Atrophy
NCT03226678	PHASE2	COMPLETED	Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)
NCT03453619	PHASE2	COMPLETED	Phase II Study Assessing Safety and Efficacy of APL-2 in Glomerulopathies
NCT03465709	PHASE1/PHASE2	TERMINATED	Pegcetacoplan (APL-2) in Neovascular AMD
NCT03593200	PHASE2	COMPLETED	A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH
NCT04572854	PHASE2	COMPLETED	Study Assessing the Safety and Efficacy of Pegcetacoplan in Post-Transplant Recurrence of C3G or IC-MPGN
NCT04579666	PHASE2	TERMINATED	MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)
NCT05148299	PHASE2	COMPLETED	A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation
NCT02264639	PHASE1	COMPLETED	A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH
NCT02461771	PHASE1	COMPLETED	Assessment of Safety, Tolerability and Pharmacokinetics of Intravitreal Pegcetacoplan (APL-2) for Patients With Wet AMD
NCT02588833	PHASE1	COMPLETED	Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.
NCT03777332	PHASE1	COMPLETED	Study to Evaluate the Safety of Intravitreal APL-2 in Patients Diagnosed With Geographic Atrophy
NCT05776472	Not specified	RECRUITING	A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT06161584	Not specified	ACTIVE_NOT_RECRUITING	A Prospective, Multicenter, Open-Label, Observational Phase 4 Study to Evaluate Real-World Safety, Tolerability, and Treatment Patterns of Pegcetacoplan (Syfovre) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration
NCT04729062	Not specified	APPROVED_FOR_MARKETING	C3G/Primary IC-MPGN EAP

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).