Novartis AG (NYSE:NVS) has unveiled encouraging top-line findings from the pre-planned interim analysis of its advanced-stage clinical study involving the investigative drug iptacopan for patients diagnosed with IgA nephropathy (IgAN).
Iptacopan, an oral proximal complement inhibitor targeting factor B and the alternative complement pathway, showed remarkable promise in the APPLAUSE-IgAN trial. This phase III multicenter study, characterized by its randomized, double-blind, placebo-controlled design, evaluated the safety and effectiveness of twice-daily oral iptacopan (200 mg) in 470 adults afflicted with primary IgAN.
IgAN, recognized as a complement-mediated kidney ailment predominantly affecting young adults, represents a substantial contributor to chronic kidney disease and kidney failure worldwide. Iptacopan exhibited superior performance when compared to a placebo, notably in the reduction of proteinuria (protein in the urine). The observed reduction in proteinuria, both clinically meaningful and highly statistically significant, occurred in conjunction with standard supportive care for IgAN patients.
The safety profile of iptacopan aligned with previously documented data. The APPLAUSE study will persist in assessing iptacopan’s potential to slow the progression of IgAN, employing the estimated glomerular filtration rate slope over a 24-month period as the primary endpoint. Top-line results from this study are anticipated in 2025.
Furthermore, iptacopan is presently undergoing regulatory review for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The candidate is also undergoing phase III trials for C3 glomerulopathy (C3G), immune complex membranoproliferative glomerulonephritis, and atypical hemolytic uremic syndrome. It has been granted Breakthrough Therapy Designation for PNH and C3G by the FDA, in addition to orphan drug designations for PNH and C3G from both the FDA and the European Medicines Agency.
Novartis recently acquired Chinook Therapeutics for $3.5 billion, bolstering its renal drug pipeline with two late-stage candidates, atrasentan and zigakibart (BION-1301), focused on IgAN. Novartis’ stock has seen a 9.8% increase year-to-date, surpassing the industry’s 4.2% growth rate.
Novartis is currently streamlining its pharmaceutical portfolio, having recently completed the divestment of its “front of eye” ophthalmology assets to Bausch + Lomb (BLCO) for $2.5 billion. This deal encompassed Xiidra, a treatment for dry eye disease symptoms, and investigational medication SAF312 (libvatrep), intended as a pioneering therapy for chronic ocular surface pain. It also includes the AcuStream delivery device for dry eye indications and OJL332, in the pre-clinical development stage.
In alignment with its objective of becoming a pure-play pharmaceutical company, Novartis remains focused on maximizing the performance of its key drugs, such as Entresto, Pluvicto, Kesimpta, and Kisqali, which continue to drive growth and maintain positive momentum. With these strong performers, strategic acquisitions, and a streamlined focus, Novartis anticipates robust growth in the quarters ahead.
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