Novartis' iptacopan meets Phase II goals in rare kidney disease

Swiss pharma giant Novartis (NOVN: VX) says that a Phase II study of its investigational compound iptacopan (LNP023) – a first-in-class, oral, selective factor B inhibitor – in patients with C3 glomerulopathy (C3G) met primary endpoints in both patient cohorts. The data were presented at the American Society of Nephrology (ASN) 2021 Annual Meeting.

In the final analysis from the open-label, two-cohort Phase II study (NCT03832114), patients were treated with 200mg of iptacopan twice daily for 12 weeks, in addition to background therapy. Patients in cohort A (16 with C3G, but who have not had a kidney transplant [native C3G]) showed a significant 45% reduction in proteinuria (protein in urine) compared to baseline, as measured by 24-hour urinary protein to creatine ratio (UPCR 24h; P=0.0003). Patients in cohort B (7 whose C3G had returned following a kidney transplant) showed significantly reduced C3 protein deposits compared to baseline, as measured by C3 deposit score (based on immunofluorescence microscopy) from kidney biopsy (P=0.0313).

Additionally, both cohorts of this Phase II study showed strong and sustained inhibition of alternative complement pathway activity and normalization of serum C3 levels over 12 weeks. In combined data from both cohorts, kidney function remained stable after 12 weeks, as assessed by estimated glomerular filtration rate (eGFR, average increase of 1.04 mL/min compared to baseline).