Novartis Announces First-in-Class Oral Factor B Inhibitor Iptacopan Meets Primary Endpoint in Phase II Trial for C3 Glomerulopathy

Today, Novartis announced that its potential first-in-class oral factor B inhibitor, iptacopan (LNP023), met the primary endpoint in a Phase 2 clinical trial in patients with C3 glomerulopathy (C3G), significantly reducing proteinuria levels. Novartis has already actively initiated patient enrollment for a pivotal Phase 3 clinical trial. The press release noted that iptacopan has the potential to become the first targeted therapy for patients with C3G.

C3G is a rare and severe form of primary glomerulonephritis characterized by complement dysregulation. Approximately 50% of patients progress to end-stage renal disease (ESRD) within 10 years, and 50–70% experience disease recurrence following kidney transplantation.

Iptacopan is an oral, specific inhibitor of factor B in the alternative complement pathway. It may treat various diseases caused by alternative complement pathway dysfunction without affecting immune responses against microbial invasion mediated by other complement pathways, thereby reducing the risk of infection in patients.

▲ Factor B inhibitors can be used to treat various complement-driven kidney diseases (Image source: Novartis official website)

In this Phase 2 clinical trial comprising two patient cohorts, C3G patients who had not yet undergone kidney transplantation exhibited a 45% reduction in proteinuria levels from baseline after 12 weeks of iptacopan treatment (p=0.0003). In the cohort of patients who had undergone kidney transplantation but experienced C3G recurrence, iptacopan significantly reduced renal deposition of complement C3 protein (p=0.0313).

Additionally, both patient cohorts demonstrated sustained and potent inhibition of alternative complement pathway activity. Pooled data from both patient cohorts indicated that renal function remained stable after 12 weeks.

References:

[1] Novartis iptacopan meets primary endpoints in Phase II study in rare kidney disease C3 glomerulopathy (C3G). Retrieved November 4, 2021, from https://www.prnewswire.com/news-releases/novartis-iptacopan-meets-primary-endpoints-in-phase-ii-study-in-rare-kidney-disease-c3-glomerulopathy-c3g-301416937.html

(Text abridged)

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