According to the results, 200mg twice daily iptacopan showed superiority against placebo in delivering clinically meaningful and statistically significant proteinuria reduction on top of background therapy at six months
Novartis announces positive results for iptacopan in Phase 3 APPEAR-C3G trial. (Credit: Novartis AG)
Novartis said that the Phase 3 APPEAR-C3G trial of iptacopan in patients with C3 glomerulopathy (C3G) has met its primary endpoint.
Iptacopan is an oral, Factor B inhibitor of the alternative complement pathway.
APPEAR-C3G randomised patients in a 1:1 ratio in a six-month double-blind period to receive iptacopan or a placebo on top of background therapy.
This was followed by a six-month open-label period where all patients were administered with the Factor B inhibitor in addition to background therapy.
According to the results, 200mg twice daily iptacopan showed superiority against placebo in delivering clinically meaningful and statistically significant reduction of proteinuria on top of background therapy at six months.
In addition, the safety profile of the investigational therapy was consistent with previously reported data.
Novartis chief medical officer and development president Shreeram Aradhye said: “People living with C3 glomerulopathy have no approved treatment options indicated for this progressive disease, posing many challenges and uncertainty for these mostly young patients.
“These positive results demonstrate the potential of Iptacopan to provide clinically meaningful benefit in C3G and add to our growing body of evidence that supports its use across multiple complement-mediated diseases.”
APPEAR-C3G is a multicentre, randomised, double-blind, parallel group, placebo-controlled study.
The primary endpoint for the double-blind period was proteinuria reduction at six months. For an open-label period, it was defined as the proteinuria reduction at 12 months and a comparison between proteinuria reduction at six and 12 months.
The results will be shared with international health authorities in anticipation of possible regulatory submissions in 2024, Novartis said.
Additionally, the Swiss pharmaceutical firm is recruiting a separate cohort of adolescent patients with C3G.
Iptacopan recently showed positive interim results in IgA nephropathy (IgAN). It is also being studied in other complement-mediated disorders.
Last week, the US Food and Drug Administration (FDA) granted approval to the Factor B inhibitor, under the brand name Fabhalta, for treating adults having paroxysmal nocturnal hemoglobinuria (PNH).
Additionally, the European Medicines Agency (EMA) is reviewing the oral monotherapy for the same indication.