Reimagining Medicine
Company overview
Innovation: Pipeline overview
Cardiovascular
iptacopan - CFB inhibitor
Financial review
Immunology
2023 priorities
Innovation: Clinical trials
Neuroscience
NCT04889430 APPELHUS (CLNP023F12301)
Atypical haemolytic uraemic syndrome
Phase 3
Indication
Phase
Patients
50
Primary
Outcome
Measures
Arms
Intervention
Target
Patients
Percentage of participants with complete TMA response without the use of
PE/PI and anti-C5 antibody
Single arm open-label with 50 adult patients receiving 200mg oral twice daily
doses of iptacopan
Adult patients with aHUS who are treatment naive to complement inhibitor
therapy (including anti-C5 antibody)
Readout
Milestone(s)
Publication
2025
TBD
Oncology
Appendix
Abbreviations
Other
84 Investor Relations | Q4 2022 Results
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