ORAL IPTACOPAN MONOTHERAPY DEMONSTRATES CLINICALLY MEANINGFUL HEMOGLOBIN INCREASES IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WITH BASELINE HEMOGLOBIN LEVELS 10 TO <12 G/DL ON ANTI-C5 THERAPY: SUBGROUP ANALYSIS OF THE APPULSE-PNH PHASE 3B TRIAL (SIE 2026) - Oct 6, 2026 - Abstract #C016; Pres time: Sep 21, 2026; 11:50 AM - 01:20 PM; Location: HALL 4; P3 | "Overall, 93.8% switched from ravulizumab and 6.3% from eculizumab. Re- sults in pts with BL Hb ≥10 to <12 g/dL were consistent with the overall study findings. Iptacopan was well tolerat- ed, representing a potentially practice-changing, outpatient treatment option for pts with PNH 3."
Clinical • Monotherapy • P3 data • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases
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E. Di Bona1; A. Kulasekararaj2,3,4; F. Sicre-De-Fontbrune4,5; A. Gaya6; I. Weitz7; D. Kuter8,9; D. Araten10,11; B. Patel12; A. Singh13; R. Kelly14; M. Loschi15; V. Pullarkat16; J. Schubert17; A. M. Vannucchi18; M. N. Yenerel19; R. Peffault De Latour4,5,20; N. Mahajan21; L. Monaco21; T. Ding22; T. Lawniczek21; P. Ferber21; M. Dahlke21; A. Risitano4,23,24
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