Novartis Announces Positive Phase III Results for First-in-Class Oral Complement Inhibitor Iptacopan in C3 Glomerulopathy

On December 11, Novartis announced that its oral complement B factor inhibitor iptacopan achieved the primary endpoint in the Phase III APPEAR-C3G study for the treatment of C3 glomerulopathy. Based on six months of background treatment, iptacopan (200mg, twice daily) demonstrated superiority over placebo in reducing proteinuria, with the reduction being both clinically and statistically significant. The safety profile of iptacopan was consistent with previously reported data.

C3 Glomerulopathy (C3G) is an extremely rare progressive complement-mediated kidney disease, initially more common in children and young adults. Each year, approximately 1-2 people per million globally are newly diagnosed with C3G.

Novartis stated that it will discuss these data with global regulatory agencies and anticipates potentially submitting a regulatory application in 2024. The APPEAR-C3G study will continue into a 6-month open-label period, during which all patients will receive iptacopan treatment, including those previously on placebo. Additionally, another cohort study targeting adolescent patients with C3G is currently underway.

Iptacopan is a first-in-class, orally administered inhibitor targeting Factor B of the alternative complement pathway. The product acts upstream of the terminal C5 pathway, simultaneously controlling both intravascular and extravascular hemolysis, addressing the limitations of anti-C5 antibodies while providing patients with an oral monotherapy option.

Iptacopan is also being studied in other complement-mediated diseases. Recently, iptacopan has been approved by the FDA for marketing as the first oral monotherapy for the treatment of adult paroxysmal nocturnal hemoglobinuria (PNH). In October this year, the Phase III APPLAUSE-IgAN study of the drug for IgA nephropathy (IgAN) also achieved positive key results.

Shreeram Aradhye, MD, Chief Medical Officer and President of Global Drug Development at Novartis, said: "There are currently no approved treatments for C3 glomerulopathy, a progressive disease, which presents significant challenges and uncertainties for the predominantly young patient population. These positive results indicate that iptacopan has the potential to deliver clinically meaningful benefits to patients with C3G and will support the further development of iptacopan across multiple complement-mediated diseases."

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