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Hematology-Oncology

Iptacopan Demonstrates Clinical Efficacy in Cold Agglutinin Disease While Falling Short in Immune Thrombocytopenia: Results from a Phase 2 Basket Trial

This Phase 2 basket trial highlights the divergent efficacy of the oral factor B inhibitor iptacopan, showing significant hemoglobin improvements in Cold Agglutinin Disease but failing to meet platelet response endpoints in Immune Thrombocy

Jan 19, 2026
Clinical Updates

Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Haemoglobinuria: 48-Week Outcomes from APPLY-PNH and APPOINT-PNH Phase 3 Trials

Oral iptacopan provides sustained haemolysis control and improved haemoglobin in PNH patients over 48 weeks, with a favorable safety profile, supporting its role as a new therapeutic option.

Aug 3, 2025Sign in to read

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