The following article features coverage from the European Hematology Association (EHA) 2021 Virtual Congress. Click here to read more of Hematology Advisor‘s conference coverage.

Among patients with paroxysmal nocturnal hemoglobinuria (PNH), iptacopan shows promising efficacy and safety, and may help to improve treatment in the first line, according to research presented at the European Hematology Association (EHA) 2021 Virtual Congress.

PNH is a severe blood disease linked with PIGA mutation in hematopoietic stem cells. While anti–complement component 5 antibodies control PNH-linked intravascular hemolysis and may reduce thromboembolic events, as many as 50% of patients with PNH remain transfusion-dependent, and as many as 40% of patients may have residual anemia.

Iptacopan (LNP023), a novel, orally administered selective B factor inhibitor, has been shown in preclinical study to inhibit intravascular and extravascular hemolysis. For this randomized multi-center phase 2 study ( Identifier: NCT03896152), researchers evaluated the safety, efficacy, and pharmacokinetics of iptacopan among patients with PNH-associated hemolysis. All included patients were naïve to treatment.

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Overall, 13 patients were included in this study; the age range was 20 to 62 years, and 7 patients were female. Of enrolled patients, 7 were randomly assigned to receive iptacopan 25 mg, increasing to 100 mg through week 4 (cohort 1), and 6 were assigned to receive iptacopan 50 mg, increasing to 200 mg (cohort 2).

Lactate dehydrogenase levels decreased by at least 60% in all patients, meeting the study’s primary endpoint. In cohorts 1 and 2, the mean lactate dehydrogenase levels decreased by 79.7% and 89.7% at week 4, respectively, and by 86.2% and 85.9% at week 12.

All patients were transfusion-free week through 12, except for 1 patient who received a red blood cell transfusion on day 3. At week 12, mean hemoglobin levels had increased by 23.4 g/L and 37.1 g/L in cohorts 1 and 2, respectively.

One patient discontinued treatment because of headache on day 2; no serious adverse events were recorded over the 12-week period.

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Jang JH, Wong L, Bor Ko BS, et al. First-line treatment of PNH patients with iptacopan leads to rapid and durable hemoglobin increase by controlling both intra- and extravascular hemolysis. Paper presented at: European Hematology Association 2021 Virtual Congress; June 2021. Abstract S173.