Novartis presents promising interim Phase II data of potential first-in-class oral therapy iptacopan (LNP023) in rare renal disease C3 glomerulopathy (C3G)

• C3 glomerulopathy (C3G) is a rare renal disease, affecting young patients with a poor prognosis and significant unmet need.^1–3 

• Iptacopan (LNP023) is a potential first-in-class, oral, potent and selective factor B inhibitor of the complement system’s alternative pathway, targeting the underlying cause of C3G.^4–6

• Data presented at the American Society of Nephrology (ASN) 2020 Annual Meeting shows that investigational iptacopan effectively and safely reduced proteinuria in patients with C3G⁷.

• Iptacopan is in parallel development for a number of renal conditions, including C3 glomerulopathy (C3G), IgA nephropathy (IgAN), atypical hemolytic uremic syndrome (aHUS), and membranous nephropathy (iMN) as well as in paroxysmal nocturnal hemoglobinuria (PNH), a hematological disease. Positive Phase II results in PNH were presented at EBMT in August⁸.

• The European Medicines Agency has granted iptacopan a priority medicines (PRIME) designation in C3G and an orphan drug designation in IgA nephropathy (IgAN).

Basel, October 26, 2020 — Novartis today announced positive Phase II interim analysis results for iptacopan (LNP023), an investigational oral treatment for C3 glomerulopathy (C3G), presented at the virtually held American Society of Nephrology (ASN) 2020 Annual Meeting.

Data from the open-label Phase II study (NCT03832114), showed that after 12 weeks, iptacopan significantly reduced proteinuria by 49% compared to baseline values, as measured by 24-hour urine protein/creatinine ratio (UPCR) assessment, in twelve patients with C3G (P=0.0005). Iptacopan strongly inhibited alternative complement pathway activity and improved plasma C3 levels. In addition, iptacopan stabilized renal function as assessed by eGFR (estimated glomerular filtration rate) at week 12 and this effect was maintained in the seven patients that were treated for a total of six months after rolling over into the long-term extension study (NCT03955445)⁷.

“Proteinuria indicates the presence of inflammation in the kidney. Results from this study demonstrate that iptacopan significantly reduces proteinuria in patients with C3G,” said the lead study investigator, Dr Edwin Wong, Consultant Nephrologist at the National Renal Complement Therapeutics Centre, Newcastle upon Tyne NHS Foundation Trust, Newcastle University. “This data also highlights iptacopan’s ability to strongly and specifically inhibit the alternative complement pathway, targeting the underlying cause of this disease and potentially providing a much needed treatment option for C3G patients who have significant unmet needs.”