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APL-2
APL-2 is a Complement C3 inhibitor Small molecule drug developed by Apellis Pharmaceuticals, Inc.. It is currently in Phase 3 development for Geographic atrophy (age-related macular degeneration), C3 glomerulopathy, Post-transplant thrombotic microangiopathy. Also known as: Pegcetacoplan.
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.
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Immunology slight uplift
+1.0pp
Mature endpoint landscape (ACR, DAS28, PASI) makes immunology approvals slightly more predictable.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
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
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:
- APL-2 CI brief — competitive landscape report
- APL-2 updates RSS · CI watch RSS
- Apellis Pharmaceuticals, Inc. portfolio CI
Frequently asked questions about APL-2
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Is APL-2 also known as anything else?
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Related
- Drug class: All Complement C3 inhibitor drugs
- Target: All drugs targeting Complement C3
- Manufacturer: Apellis Pharmaceuticals, Inc. — full pipeline
- Therapeutic area: All drugs in Immunology
- Indication: Drugs for Geographic atrophy (age-related macular degeneration)
- Indication: Drugs for C3 glomerulopathy
- Indication: Drugs for Post-transplant thrombotic microangiopathy
- Also known as: Pegcetacoplan