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APL-2
APL-2 is a complement C3 inhibitor that blocks the central component of the complement cascade to reduce inflammation and tissue damage.
APL-2 is a complement C3 inhibitor that blocks the central component of the complement cascade to reduce inflammation and tissue damage. Used for Geographic atrophy (age-related macular degeneration), C3 glomerulopathy, Post-transplant thrombotic microangiopathy.
At a glance
| Generic name | APL-2 |
|---|---|
| Also known as | Pegcetacoplan |
| Sponsor | Apellis Pharmaceuticals, Inc. |
| Drug class | Complement C3 inhibitor |
| Target | Complement C3 |
| Modality | Small molecule |
| Therapeutic area | Immunology |
| Phase | Phase 3 |
Mechanism of action
APL-2 inhibits complement factor C3, a central hub protein in the complement cascade that is activated in all three pathways (classical, alternative, and lectin). By blocking C3 activation and cleavage, the drug prevents downstream complement-mediated inflammation, cell lysis, and tissue injury. This mechanism is intended to treat diseases driven by excessive or dysregulated complement activation.
Approved indications
- Geographic atrophy (age-related macular degeneration)
- C3 glomerulopathy
- Post-transplant thrombotic microangiopathy
Common side effects
- Meningococcal infection risk
- Nasopharyngitis
- Headache
Key clinical trials
- Safely Quenching Complement in Stroke Survivors (PHASE1)
- 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 (PHASE2)
- 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 (PHASE2)
- Pegcetacoplan in Combination With Modified FOLFIRINOX for the Treatment of Metastatic Pancreatic Ductal Adenocarcinoma (PHASE1, PHASE2)
- Study to Evaluate a Pegcetacoplan (Syfovre®) Prefilled Syringe (PHASE3)
- An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic Atrophy Secondary to AMD (PHASE3)
- A Sequential Phase 2/3 Study of APL2 in Patients With Focal Segmental Glomerulosclerosis (PHASE2, PHASE3)
- A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (PHASE2)
Primary sources
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |