Trial results for the Phase 2 study (NCT05086744) investigating Iptacopan in autoimmune benign hematological disorders were posted on ClinicalTrials.gov on 2025-03-19. The study reported that for participants with Cold Agglutinin Disease (CAD), 5 participants achieved a clinically meaningful response, while for Immune Thrombocytopenia (ITP), 0 participants achieved this outcome.
Background
The study, titled "Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders," aimed to evaluate the efficacy and safety of Iptacopan in participants with autoimmune benign hematological disorders, including primary Immune Thrombocytopenia (ITP) and primary Cold Agglutinin Disease (CAD).
Trial design
The study (NCT05086744) was a Phase 2 basket study that enrolled 19 participants. The trial was designed to assess Iptacopan in individuals diagnosed with Immune Thrombocytopenia (ITP) and Cold Agglutinin Disease (CAD). The overall status of the study is TERMINATED.
Key results
The trial results provided outcome measurements for both cohorts:
- For "Cohort 1 (ITP): Number of Participants With a Clinically Meaningful Response": 0 Participants in the overall ITP cohort (sC5b-9 High, sC5b-9 Low, and combined) achieved this response.
- For "Cohort 1 (ITP): Magnitude of Platelet Count Increase From Baseline": 3 Participants in the overall ITP cohort showed a platelet count increase from baseline (1 Participant in sC5b-9 High, 2 Participants in sC5b-9 Low).
- For "Cohort 2 (CAD): Number of Participants With a Clinically Meaningful Response": 5 Participants achieved a clinically meaningful response.
- For "Cohort 2 (CAD): Time to First Hemoglobin Level ≥1.5 g/dL Above Baseline": The median time was 29.0 days.
- For "Cohort 2 (CAD): Duration of Time During Which Hemoglobin Level Remains ≥1.5 g/dL Above Baseline Without the Use of Rescue Therapy": The median duration was 56.0 days.
What this means
The results from this Phase 2 basket study suggest that Iptacopan may have a differential effect across the studied autoimmune hematological disorders. While no participants with Immune Thrombocytopenia (ITP) achieved a clinically meaningful response, a subset of participants with Cold Agglutinin Disease (CAD) did show a response, with 5 participants achieving a clinically meaningful response. The median time to hemoglobin increase and duration of response in the CAD cohort provide initial insights into the potential activity of Iptacopan in this specific condition. The termination of the study indicates that further development for ITP in this context may not proceed, while the CAD data might warrant further investigation.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT05086744, titled "Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders," were posted on 2025-03-19 on clinicaltrials.gov.