Kidney Week

Abstract: INFO09-TH

Randomized, Controlled Phase 2 Study to Evaluate Iptacopan Treatment in Patients with Active ANCA-Associated Vasculitis

Session Information

• Informational Posters - 1
  November 06, 2025 | Location: Exhibit Hall, Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Glomerular Diseases

• No subcategory defined

Authors

• Terrier, Benjamin, Department of Internal Medicine, Hôpital Cochin, Assistance Publique - Hôpitaux de Paris, Université Paris-Cité, Paris, France

Benjamin Terrier

• Pugnet, Grégory, Department of Internal Medicine and Clinical Immunology, Toulouse University Hospital, Paris, France

Grégory Pugnet, MD, PhD

• Alibaz-Oner, Fatma, Division of Rheumatology, Department of Internal Medicine, School of Medicine, Marmara University, Istanbul, Turkey

Fatma Alibaz-Oner, MD

• Brigante, Jorge Alejandro, Rheumatology Department, Internal Medicine Service, Sanatorio Güemes, Buenos Aires, Argentina

Jorge Alejandro Brigante, MD, MSc

• Löffler, Christian, Department of Internal Medicine, Rheumatology, Pulmonology, Nephrology and Diabetology, Vasculitis Reference Center of the European Union, ERN-RITA, Medius Klinik, Kirchheim, Germany

Christian Löffler, DrMed

• Schreiber, Adrian, Department of Nephrology and Medical Intensive Care, Charité-Universitätsmedizin Berlin, Berlin, Germany

Adrian Schreiber, DrMed

• Bai, Lili, China Novartis Institutes for BioMedical Research Co., Ltd., Shanghai, China

Lili Bai

• Hanser, Malika, Novartis Pharma AG, Basel, Basel-Stadt, Switzerland

Malika Hanser, PharmD, PhD

• Rysz, Michal, Novartis Pharma AG, Basel, Basel-Stadt, Switzerland

Michal Rysz, MSc

• Seroutou, Abdelkader, Novartis Pharma AG, Basel, Basel-Stadt, Switzerland

Abdelkader Seroutou

• Junge, Guido, Novartis Pharma AG, Basel, Basel-Stadt, Switzerland

Guido Junge, MD, PhD

• Chen, Min, Renal Division, Department of Medicine, Peking University First Hospital, Bejing, China

Min Chen, MD, PhD

Description

ANCA-associated vasculitis (AAV) is a group of rare multi-system autoimmune diseases characterized by inflammation and damage to mainly small blood vessels. The alternative pathway (AP) of the complement system plays a central role in the pathogenesis of AAV. There are limited approved therapies, and an unmet need remains for a therapy to induce persistent remission in patients with AAV while reducing glucocorticoid (GC) treatment and avoiding long-term B cell depletion.
Iptacopan is an oral, proximal complement inhibitor that targets Factor B to selectively inhibit the AP. It is approved for the treatment of several complement-mediated diseases (PNH, IgAN, C3G) and may have the potential to improve outcomes in patients with AAV.
This Phase 2 study randomizes patients in a placebo-controlled 24-week (W) induction period to evaluate the efficacy and safety of iptacopan (200mg twice daily) in combination with rituximab induction therapy and rapid defined GC tapering, for treatment of newly diagnosed or relapsed adult patients with active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA). The induction period is followed by an open label 24W maintenance period (Figure 1).
The primary endpoint: sustained remission through W48 defined as complete remission (Birmingham Vasculitis Activity Score [BVAS]=0 and GC ≤5mg/day) at W24 without major relapse up to W48.
This study will provide evidence towards the safety and efficacy of oral iptacopan for the treatment of patients with AAV (ClinicalTrials.gov: NCT06388941).

Figure 1. Iptacopan in patients with ANCA-associated vasculitis – Phase 2 trial design

Funding

• This study is funded by Novartis Pharma AG. Professional medical writing assistance was provided Carol Crawford (Novartis Ireland Ltd.) and Andrew Jobson (Novartis Pharmaceuticals UK Limited) and funded by Novartis Pharma AG.