Jun 07, 2021
  • 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.

This media update contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this media update, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this media update will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this media update as of this date and does not undertake any obligation to update any forward-looking statements contained in this media update as a result of new information, future events or otherwise.

About Novartis
Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.

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  1. Wong E, Praga M, Nester C, et al. Iptacopan (LNP023): a novel oral complement alternative pathway factor B inhibitor safely and effectively stabilises eGFR in C3 glomerulopathy. Presented at the ERA-EDTA congress.
  2. Wong E, Praga M, Nester CM, et al. LNP023: a novel oral complement alternative pathway factor B inhibitor safely and effectively reduces proteinuria in C3 glomerulopathy. Abstract presented at the 2020 American Society of Nephrology Congress in October 22–25, 2020.
  3. McGrogan A, Franssen CFM, de Vries CS. The incidence of primary glomerulonephritis worldwide: a systematic review of the literature. Nephrol Dial Transplant. 2011;26(2):414–430.
  4. Nester CM, Smith RJ. Treatment options for C3 glomerulopathy. Curr Opin Nephrol Hypertens. 2013;22(2):231–237.
  5. Smith RJH, Appel GB, Blom AM, et al. C3 glomerulopathy - understanding a rare complement-driven renal disease. Nat Rev Nephrol. 2019;15(3):129–143.
  6. Abbasi MA, Chertow GM, Hall YN. End-stage renal disease. BMJ Clin Evid. 2010;2010.
  7. Lopez-Giacoman S, Madero M. Biomarkers in chronic kidney disease, from kidney function to kidney damage. World J Nephrol. 2015;4(1):57–73.
  8. Clinicaltrials.gov. Study on Efficacy and Safety of LNP023 in C3 Glomerulopathy Patients Transplanted and Not Transplanted. Available at: https://clinicaltrials.gov/ct2/show/NCT03832114. Accessed May 2021.
  9. Merle NS, Church SE, Fremeaux-Bacchi V, Roumenina LT. Complement system part I – molecular mechanisms of activation and regulation. Front Immunol. 2015;6:262.
  10. Schubart A, Anderson K, Mainolfi N, et al. Small-molecule factor B inhibitor for the treatment of complement-mediated diseases. Proc Natl Acad Sci U S A. 2019;116(16):7926–7931.
  11. Sarma JV, Ward PA. The complement system. Cell Tissue Res. 2011;343(1):227–235.
  12. Willows J, Brown M, Sheerin NS. The role of complement in kidney disease. Clin Med. 2020;20(2):156–160.
  13. Łukawska E, Polcyn-Adamczak M, Niemir ZI. The role of the alternative pathway of complement activation in glomerular diseases. Clin Exp Med. 2018;18(3):297–318.
  14. Koscielska-Kasprzak K, Bartoszek D, Myszka M, Zabinska M, Klinger M. The complement cascade and renal disease. Arch Immunol Ther Exp (Warsz). 2014;62(1):47–57.
  15. De Vriese AS, Sethi S, Van Praet J, Nath KA, Fervenza FC. Kidney disease caused by dysregulation of the complement alternative pathway: An etiologic approach. J Am Soc Nephrol. 2015;26(12):2917–2929.
  16. Medjeral-Thomas NR, O’Shaughnessy MM, O’Regan JA, et al. C3 glomerulopathy: clinicopathologic features and predictors of outcome. Clin J Am Soc Nephrol. 2014;9(1):46–53.

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