Novartis announces new interim analysis Phase II data for iptacopan in rare kidney disease C3 glomerulopathy (C3G)

  • Data shows investigational iptacopan improved estimated glomerular filtration rate (eGFR) slope and stabilized kidney function in patients with C3G1; Phase III clinical trial to start in 2021
  • Results add to body of information on iptacopan, with previous interim analysis data from same trial showing significant reduction in proteinuria, improved plasma C3 levels, and favorable safety and tolerability profile over 12 weeks2
  • There are no currently approved treatments for C3G - a rare and often progressive disease that mainly affects adolescents and young adults and can progress to kidney failure3-6
  • Iptacopan is in development for several complement-driven renal diseases (CDRDs), including C3Gand IgA nephropathy (IgAN), and the blood disorder paroxysmal nocturnal hemoglobinuria (PNH), targeting a key driver of these diseases

Basel, June 07, 2021 - Novartis today announced positive new interim Phase II data showing investigational iptacopan (LNP023) - a first-in-class, oral, targeted factor B inhibitor - improved estimated glomerular filtration rate (eGFR) slope and stabilized kidney function in patients with C3 glomerulopathy (C3G) treated with iptacopan1. The data were presented at the 58th ERA-EDTA Congress held virtually from June 5-8, 2021.

The new interim analysis data from the open-label Phase II study (NCT03832114) in patients with C3G showed twice-daily 200mg iptacopan stabilized kidney function, as measured by change in eGFR slope1: a key measure of kidney clearance function that estimates the rate of blood passing through and being filtered by the kidneys7. The kidney function of 12 patients treated with iptacopan for 12 weeks was compared with their historical kidney function data for the two-year period before they entered the study (or since diagnosis was made where this was less than two years)1. Furthermore, seven C3G patients who received iptacopan for up to 25 weeks in a long-term extension study (NCT03955445) showed ongoing eGFR stabilization, suggesting extended iptacopan treatment may prolong the time to, or even potentially prevent, the development of kidney failure1.

About the study
NCT03832114 is a Phase II, open-label, two cohort, non-randomized study evaluating the efficacy, safety and pharmacokinetics of iptacopan in patients with C3 glomerulopathy (C3G) (Cohort A) and patients who have undergone kidney transplantation and have subsequent C3G recurrence in the transplanted organ (Cohort B)1,2,8. The aim of the analysis presented at the 2021 ERA-EDTA Congress was to determine whether iptacopan treatment altered eGFR slope in Cohort A1. On completion of the study, all patients had the option to receive ongoing iptacopan in a long-term extension study (NCT03955445)1.

About iptacopan
Iptacopan is an investigational, first-in-class, orally administered factor B inhibitor of the alternative complement pathway, targeting one of the key drivers of these diseases9-11. It has the potential to become the first targeted therapy to delay progression to dialysis in C3G. Discovered at the Novartis Institutes for BioMedical Research, iptacopan is currently in development for a number of CDRDs where significant unmet needs exist, including C3G, IgA nephropathy (IgAN), atypical hemolytic uremic syndrome (aHUS), and membranous nephropathy (MN), as well as the blood disorder PNH.

While Novartis has a 35-year history in kidney transplantation treatments, iptacopan is the first treatment in the nephrology pipeline addressing CDRDs. Our aim is to transform treatment by targeting one of the key drivers of these rare and often progressive diseases10 and, in doing so, potentially extend dialysis-free life for people with CDRDs.

About complement-driven renal diseases (CDRDs)
CDRDs, which include C3G, are thought to be partly caused by an overactivation of the alternative complement pathway - part of the immune system - creating an inflammatory response, which can lead to kidney damage10,12-15. CDRDs mainly affect adolescents and young adults, and can often lead to kidney failure which requires dialysis or transplantation and can lead to premature death3-6.

Approximately 50% of C3G patients progress to kidney failure within 10 years of diagnosis4,5,16. Among patients who have undergone kidney transplantation, disease recurrence is not uncommon, with one study seeing an estimated 30% and 70% risk of transplant loss at 5 and 10 years, respectively5,16.

There is a need for effective and well-tolerated, targeted therapies C3G that can delay disease progression.

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