Novartis' Iptacopan Meets Primary Endpoint Goal In Rare Kidney Disease Trial - Novartis (NYSE:NVS)

Novartis AG NVS has announced Phase 2 trial data of iptacopan, a selective factor B inhibitor, in patients with C3 glomerulopathy (C3G).
In C3G, an overly-active alternative complement pathway causes deposits of C3 protein to build up in blood vessels that filter waste and remove extra fluids from the blood.
The data were presented at the American Society of Nephrology (ASN) 2021 Annual Meeting.
The trial met primary endpoints in both patient cohorts.
Patients were treated with 200mg of iptacopan twice daily for 12 weeks, in addition to background therapy.
Patients in cohort A (n=16) who have not had a kidney transplant [native C3G]) showed a significant 45% reduction in proteinuria (protein in urine) compared to baseline.
Patients in cohort B (n=7) whose C3G had returned following a kidney transplant) showed significantly reduced C3 protein deposits compared to baseline.
Additionally, both cohorts showed sustained and robust inhibition of alternative complement pathway activity and normalization of serum C3 levels over 12 weeks.
Iptacopan showed a favorable safety and tolerability profile, with no serious adverse events suspected to be related to iptacopan.
Related Link: Novartis's Iptacopan Aces Mid-Stage Kidney Disease Trial.
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