Q3 2022 Results
Company overview
Financial review
2022 priorities
Appendix
References
Iptacopan has the potential to be a first line, oral complement
inhibitor mono-therapy in patients with PNH
3 INNOVATION
Unmet need in PNH1-5
Iptacopan PNH value proposition
10-20
cases/million; US 4-6k
☑
Addresses both intra- and extravascular
hemolysis, resulting in improvement of Hb levels
Potential for lower transfusion requirements
~40%
remain anemic (Hb <10g/dl) despite
anti-C5 treatments (eculizumab /
ravulizumab)
-50%
of these receive transfusions
Potential for improved quality of life
Potentially first oral administration,
offering significant convenience to patients
Potential for broad first line label
PNH - Paroxysmal nocturnal hemoglobinuria Hb Hemoglobin 1. Cançado RD, 2021.
2. Jalbert JJ, 2019. 3. Mon Pere N, 2018. 4. Debureaux PE 2021
5. Petropoulou AD 2010.
18 Investor Relations | Q3 2022 Results
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