Q4 2021 Results
Participants
Company overview
Pharmaceuticals
Oncology
Financial review
Conclusion
Appendix
Financial performance
Innovation: Pipeline overview
Innovation: Clinical trials
CRM
IHD
Neuroscience
Ophthalmology
Respiratory & Allergy
Oncology: Solid Tumors
Hematology
Biosimilars
Global Health
Abbreviations
iptacopan - CFB inhibitor
Study
Indication
Phase
Patients
Primary Outcome
Measures
Arms Intervention
NCT04889430 APPELHUS (CLNP023F12301)
Atypical haemolytic uraemic syndrome
Phase 3
50
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)
Target Patients
Read-out Milestone(s)
2024
Publication
TBD
137 Investor Relations | Q4 2021 Results
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