Novartis presented results from a pre-specified interim analysis of the Phase III APPLAUSE-IgAN study of Fabhalta (iptacopan), an investigational Factor B inhibitor of the alternative complement pathway, in patients with IgA nephropathy (IgAN).

In the analysis, patients treated with Fabhalta achieved a 38.3% (p<0.0001) proteinuria reduction (as measured by 24-hour urine protein to creatinine ratio [UPCR]) at 9 months when compared to placebo on top of supportive care.

Proteinuria reduction is an increasingly recognized surrogate marker correlating with progression to kidney failure and has been used as an endpoint in IgAN clinical trials to support accelerated approvals. The study also showed that Fabhalta was well tolerated with a favorable safety profile consistent with previously reported data1,8. Results were presented today during a late-breaking clinical trials session at the World Congress of Nephrology (WCN) in Buenos Aires, Argentina.

“In IgAN, part of the immune system called the alternative complement pathway can become overly activated in the kidneys, which causes an inflammatory response, leading to progressive kidney damage and gradual loss of kidney function. The loss of kidney function, together with potential side effects of IgAN treatments available until recently, significantly impact patients’ lives,” said Professor Dana Rizk, Investigator and APPLAUSE-IgAN Steering Committee Member and professor in the UAB Division of Nephrology. “Fabhalta is the first potential treatment for IgAN that specifically targets the alternative complement pathway.”

This pre-specified interim analysis included 250 patients for the efficacy analysis and 443 for the safety analysis1. The APPLAUSE-IgAN study continues in a double-blind fashion, and therefore only limited interim analysis results can be presented.

“IgAN progresses over many years, and patients’ needs may evolve such that different therapies may be best used at different times,” said David Soergel, M.D., Global Head, Cardiovascular, Renal and Metabolism Development Unit, Novartis. “Our renal pipeline includes medicines with a variety of mechanisms which may allow them to be targeted to patients based on their clinical characteristics.”

Other data presented at WCN include IgAN and C3 glomerulopathy (C3G) real-world studies. Novartis will be presenting further data from the renal portfolio at future medical meetings.