IPTACOPAN ASH UPDATE slide image

IPTACOPAN ASH UPDATE

Opportunity to redefine PNH treatment paradigm ~6k Prevalent¹ PNH patients in US 30% ~400 Incident² PNH patients/year in US Treated with complement inhibitor³ Untreated 70% • ■ ■ Current market -USD 2bn WW (USD 1bn US)4 ■ Of C5-treated patients, -80% have Hb < 12g/dL5 Still experiencing symptoms - - Managing life around infusion schedule Some still receiving transfusions Varying views of when treatment should be started Heterogeneous presentation of symptoms Some unwilling to commit to regular infusions Some still receiving transfusions Displace Anti-C5 Potentially increase treatment rate Start appropriate patients on iptacopan 1. Prevalence: 12-18 per million individuals in the US (Jalbert JJ, 2019, Mon Pere N, 2018). 2. Incidence: 1.0-1.5 per million individuals (Hill A, 2017). 3. Treated with anti-C5 or anti-C3 4. Based on C5i therapies only 5. Debureaux PE et al. Bone Marrow Transplant 2021;56:2600-2 Source: Patient journey market research 2022 27 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine
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