PNH
4
Pipeline Programs
1
Companies
2
Clinical Trials
2
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
1
0
1
2
Early DiscoveryClinical DevelopmentMarket
On Market (2)
Approved therapies currently available
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Apellis PharmaceuticalsAustralia - Hawthorn East
2 programs1
1
1
PegcetacoplanPhase 31 trial
PegcetacoplanPhase 21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Apellis PharmaceuticalsPegcetacoplan
Apellis PharmaceuticalsPegcetacoplan
Clinical Trials (2)
Total enrollment: 164 patients across 2 trials
Pegcetacoplan Long Term Safety and Efficacy Extension Study
Start: Aug 2018Est. completion: Jun 2025160 patients
Phase 3Active Not Recruiting
A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH
Start: Aug 2018Est. completion: Oct 20194 patients
Phase 2Completed
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
1 late-stage (Phase 3) programs — potential near-term approvals
1 companies competing in this space