Iptacopan Demonstrates Reductions in Proteinuria for Patients With C3 Glomerulopathy


Investigational iptacopan (LNP023) demonstrated statistically significant and clinically important reductions in proteinuria for patients with C3 glomerulopathy (C3G), according to the final analysis of a phase 2 study. Further, statistically significant reduction in C3 protein deposits were achieved in the same study in a cohort of patients whose C3G recurred following kidney transplantation. These data were presented at the American Society of Nephrology (ASN) 2021 Annual Meeting.

“The data presented at ASN provide a detailed picture of the potential of iptacopan for the treatment of patients with C3G and, for the first time, in patients whose C3G had returned following kidney transplantation,” said Edwin Wong, PhD, consultant nephrologist at the National Renal Complement Therapeutics Centre, Newcastle upon Tyne NHS Foundation Trust, Newcastle University, UK, in a press release. “These results are important for patients with C3G because proteinuria is a key risk predictor for kidney disease progression, and deposits of C3 protein ultimately cause inflammation and kidney damage.”

In the open-label, 2-cohort phase 2 study, patients were treated with 200 mg of iptacopan twice daily for 12 weeks, in addition to background therapy. Patients in cohort A—those who had C3G but who have not had a kidney transplant—showed a 45% reduction in proteinuria (protein in urine) compared to baseline, as measured by 24-hour urinary protein to creatine ratio. Patients in cohort B—those whose C3G had returned following a kidney transplant—had significantly reduced C3 protein deposits compared to baseline, as measured by C3 deposit score from kidney biopsy.

Both cohorts demonstrated strong and sustained inhibition of alternative complement pathway activity and normalization of serum C3 levels over 12 weeks. Further, data from both cohorts showed stable kidney function after 12 weeks, as assessed by estimated glomerular filtration rate.

“C3G is a devastating disease where people can end up facing life-altering and often exhausting kidney dialysis or transplantation at a time when they might otherwise be focused on building their lives, careers, and families,” said John Tsai, head of Global Drug Development and chief medical officer at Novartis, in a press release. “With currently no approved treatments, there is a major unmet need for therapies that can delay progression to kidney failure. These data demonstrate the ability of iptacopan to strongly and specifically inhibit the key driver for C3G – the alternative complement pathway. The results also show the potential for iptacopan to provide the first targeted treatment for people living with C3G, and we are actively recruiting for our pivotal phase 3 APPEAR-C3G study.”

Iptacopan showed a favorable safety and tolerability profile in the phase 2 final analysis. No serious adverse events were suspected to be related to iptacopan.


Novartis iptacopan meets primary endpoints in Phase II study in rare kidney disease C3 glomerulopathy (C3G) [news release]. Novartis; November 4, 2021. Accessed November 5, 2021. https://www.novartis.com/news/media-releases/novartis-iptacopan-meets-primary-endpoints-phase-ii-study-rare-kidney-disease-c3-glomerulopathy-c3g

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