Among complement inhibitor­-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH), oral iptacopan appears effective at controlling hemolysis, according to research presented at the SOHO 2023 Annual Meeting. Furthermore, the drug was associated with transfusion avoidance.

Iptacopan is a novel, orally administered complement inhibitor targeting factor B, with consequent effects on the complement cascade. In a previous phase 3 trial, iptacopan appeared superior to both eculizumab and ravulizumab among patients with PNH and anemia.

For the single-arm phase 3 APPOINT-PNH trial ( Identifier: NCT04820530), researchers evaluated the safety and efficacy of iptacopan among complement inhibitor–naïve patients with PNH. At the SOHO 2023 Annual Meeting, researchers presented safety and efficacy data from the 24-week core treatment period from APPOINT-PNH.

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Overall, 40 patients were enrolled and treated with iptacopan. In the cohort, at baseline, the mean age was 42.1 years, 57.5% of patients were male sex, 70% had received red blood cell transfusions, and the mean base hemoglobin levels were 8.2 g/dL.

Analysis showed that the study met its primary endpoint: 92.2% (95% CI, 82.5%-100%) of patients had an increase of hemoglobin levels of at least 2 g/dL from baseline without blood transfusions. Furthermore, more than 50% of patients had a hemoglobin level increase of at least 12 g/dL without a transfusion.

No cases of hemolysis were reported during the study period. Treatment-related events included headache (27.5%), COVID-19 (15.0%), upper respiratory tract infections (12.5%), and diarrhea (7.5%); no deaths were reported.

“Iptacopan monotherapy may become a potentially practice-changing, oral, outpatient treatment that could become a preferred option for complement inhibitor-naïve patients with hemolytic PNH,” the authors noted in their poster.

Disclosures: This research was supported by Novartis Pharmaceuticals Corporation. Please see the original reference for a full list of disclosures.


de Latour RP, Han B, Maciejewski JP, et al. Substantial increases in paroxysmal nocturnal hemoglobinuria (PNH) red blood cell clone size with oral iptacopan monotherapy confirms control of hemolysis in complement inhibitor–naive PNH patients. Presented at: the Eleventh Annual Meeting of the Society of Hematologic Oncology (SOHO); September 6-9, 2023. Abstract MDS-278.