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APL2
APL2 is a complement C3 inhibitor that blocks the central component of the complement cascade to reduce inflammation and tissue damage.
APL2 is a complement C3 inhibitor that blocks the central component of the complement cascade to reduce inflammation and tissue damage. Used for Paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy, Post-transplant thrombotic microangiopathy.
At a glance
| Generic name | APL2 |
|---|---|
| Sponsor | Apellis Pharmaceuticals, Inc. |
| Drug class | Complement C3 inhibitor |
| Target | Complement component C3 |
| Modality | Small molecule |
| Therapeutic area | Immunology |
| Phase | Phase 2 |
Mechanism of action
APL2 inhibits complement component C3, a key node in the complement pathway that amplifies both the classical and alternative pathways. By blocking C3 activation and cleavage, the drug prevents downstream complement-mediated inflammation, cell lysis, and tissue injury. This mechanism is intended to treat complement-driven diseases including paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy, and other complement-mediated disorders.
Approved indications
- Paroxysmal nocturnal hemoglobinuria (PNH)
- C3 glomerulopathy
- Post-transplant thrombotic microangiopathy
Common side effects
- Meningococcal infection
- Headache
- Nasopharyngitis
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
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- APL2 CI brief — competitive landscape report
- APL2 updates RSS · CI watch RSS
- Apellis Pharmaceuticals, Inc. portfolio CI