Q3 2021 Investor Relations Results
Participants
Company overview
Pharmaceuticals
Oncology
Financial review
Conclusion
Appendix
References
Financial performance
Innovation: Pipeline overview
Innovation: Clinical trials
Neuroscience
Ophthalmology
Respiratory & Allergy
Oncology: Solid Tumors
Hematology
Biosimilars
Global Health
Abbreviations
CRM
IHD
iptacopan - CFB inhibitor
Study
Indication
Phase
Patients
Primary Outcome
Measures
Arms Intervention
NCT04817618 APPEAR-C3G (CLNP023B12301)
C3 glomerulopathy
Phase 3
68
Log-transformed ratio to baseline in UPCR (sampled from a 24 hour urine
collection)
Experimental: iptacopan 200mg b.i.d.
Placebo Comparator: Placebo to iptacopan 200mg b.i.d.
Patients with native C3G
Target Patients
Read-out Milesstone(s)
2023
Publication
TBD
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)
2024
TBD
69 Investor Relations | Q3 2021 Results
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