Updates in PNH

Current Treatment Paradigms

Updates on Current Therapies (From ASH
2020)
Limitations of Current PNH Therapies:
Unmet Needs
New Agents With Different MOAs
Emerging Treatments for PNH
Emerging Treatments for PNH (cont)
Novel C5 Inhibitor: Crovalimab
COMPOSER Parts 2 and 3: Efficacy Markers
Over Time
COMPOSER: Adverse Events
Phase 3 Studies of Crovalimab
Novel C5 Inhibitor: Pozelimab
Novel C5 Inhibitor: Pozelimab (cont)
Perspectives on Novel C5 Inhibitors
Emerging Treatments for PNH (cont)
C3 Inhibitor: Pegcetacoplan
PEGASUS: Pivotal Phase 3 Trial
PEGASUS: Change in Hgb Level
PEGASUS: Secondary Endpoints
PEGASUS: Adverse Events
PEGASUS: Additional Analyses Reported at
ASH 2020
Perspectives on Pegcetacoplan
Emerging Treatments for PNH (cont)
Factor B Inhibitor: Iptacopan
Iptacopan Phase 2: Baseline Characteristics
Iptacopan Phase 2: LDH and Hgb Time
Profiles
Iptacopan Phase 2: Transfusion Independence
Iptacopan Phase 2: Safety Outcomes
Phase 3 Studies of Iptacopan
Perspectives on Iptacopan
Emerging Treatments for PNH (cont)
Factor D Inhibitor: Danicopan
Danicopan Phase 2: Change in LDH and Hgb
Danicopan Phase 2: Transfusion
Requirements
Perspectives on Danicopan
Phase 3 Study of Danicopan
Factor D Inhibitor: ALXN2050
Emerging Treatments for PNH
Key Takeaways
Abbreviations