Trial results for the Phase 3 study (NCT05630001) investigating iptacopan in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who switched from anti-C5 treatment were posted on ClinicalTrials.gov on 2025-12-15. The study showed that patients treated with iptacopan experienced a mean increase in hemoglobin levels of 2.01 g/dL and 100.0% remained free from transfusions.
Background
The study aimed to evaluate the efficacy and safety of iptacopan in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). Specifically, it focused on patients who transitioned from existing standard of care anti-C5 treatments, such as eculizumab or ravulizumab, to iptacopan.
Trial design
The study (NCT05630001) was a Phase 3, single-arm, open-label trial. It enrolled 52 adult participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) who were on a stable regimen of anti-C5 treatment and switched to iptacopan. The treatment duration with iptacopan was 24 weeks.
Key results
The trial results for iptacopan 200mg b.i.d. demonstrated several key findings:
- The mean change in hemoglobin (Hb) levels as a mean of visits between Day 126 and Day 168 compared to baseline was an increase of 2.01 g/dL. This outcome was tested for both non-inferiority and superiority.
- The proportion of hematological responders to iptacopan treatment was 92.7%.
- 100.0% of participants remained free from transfusions.
- The mean change from baseline in Absolute Reticulocytes Count (ARC) levels was -89.19 10^9 cells/L.
- The mean percentage change from baseline in Lactate Dehydrogenase (LDH) levels was -1.30%.
- Changes from baseline in Treatment Satisfaction Score using the TSQM-9 Questionnaire showed mean improvements of 15.08, 12.54, 20.34, 23.86, 14.26, and 18.53 on a scale across different aspects.
Statistical analyses, including Mixed Models Analysis, yielded a p-value of 0.0001 for relevant outcomes.
What this means
The results of this Phase 3 trial suggest that iptacopan can effectively improve hematological parameters in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who switch from anti-C5 therapies. The observed increase in hemoglobin levels, high proportion of hematological responders, and complete absence of transfusions among participants indicate a positive impact on disease management. These findings support the potential of iptacopan as an alternative treatment option for PNH patients previously on anti-C5 inhibitors.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT05630001, titled "Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan," were posted on 2025-12-15 on clinicaltrials.gov.