Novartis AG (NYSE:NVS) has disclosed encouraging top-line findings from a pivotal late-stage trial involving its developmental drug, iptacopan, aimed at treating patients with IgA nephropathy (IgAN).
Iptacopan represents an oral, proximal complement inhibitor that targets factor B and effectively hinders the alternative complement pathway.
The clinical trial in question, named APPLAUSE-IgAN, is a phase III multicenter investigation characterized by its randomized, double-blind, placebo-controlled, parallel-group design. This study intends to assess the effectiveness and safety of twice-daily oral iptacopan (200 mg) in a cohort of 470 adults afflicted with primary IgAN.
IgAN is a complement-mediated kidney ailment, primarily impacting young adults, and constitutes a substantial contributor to chronic kidney disease and kidney failure cases worldwide.
Iptacopan exhibited clear superiority over the placebo group by notably reducing proteinuria (the presence of excess protein in urine). This reduction is considered clinically substantial and holds significant statistical relevance when combined with supportive care for patients suffering from IgAN.
Importantly, the safety profile of iptacopan aligns with previously reported data.
The ongoing APPLAUSE study is set to gauge iptacopan’s ability to slow down the progression of IgAN by assessing the estimated glomerular filtration rate slope over a 24-month period, which stands as the primary endpoint of the study. The presentation of top-line results is anticipated in 2025.
Additionally, iptacopan is currently undergoing regulatory review for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
The candidate is also the subject of phase III investigations for conditions such as C3 glomerulopathy (C3G), immune complex membranoproliferative glomerulonephritis, and atypical hemolytic uremic syndrome. Notably, it has garnered Breakthrough Therapy Designation from the FDA for both PNH and C3G. Further, orphan drug designations have been granted by both the FDA and European Medicines Agency for PNH and C3G.
Novartis recently bolstered its renal pipeline through the acquisition of Chinook Therapeutics for $3.5 billion, which included two late-stage candidates, atrasentan and zigakibart (BION-1301), tailored for IgAN.
Novartis shares have witnessed a 9.8% year-to-date increase, surpassing the industry’s 4.2% growth.
Novartis’ strategic focus is also reflected in its decision to streamline its pharmaceutical portfolio. It recently concluded the divestment of its ophthalmology assets related to the “front of eye” category to Bausch + Lomb (BLCO), a global eye-health company, for $2.5 billion. This transaction comprises Xiidra, a treatment for dry eye disease, investigational medicine SAF312 (libvatrep) designed for chronic ocular surface pain, as well as the AcuStream delivery device in dry eye indications and OJL332, currently in pre-clinical development.
In alignment with these developments, BLCO initiated an offering of $1.4 billion in aggregate principal amount of new senior secured notes due in 2028 to finance the Xiidra acquisition. BLCO is also considering entering into an incremental term loan facility to facilitate the transaction.
Novartis is actively pursuing its goal of becoming a pure-play pharmaceutical enterprise. Despite facing generic competition for some of its older drugs, the company’s strong performance in key products like Entresto, Pluvicto, Kesimpta, and Kisqali continues to fuel growth and sustain momentum.
With a robust portfolio, strategic acquisitions, and a sharpened focus, Novartis is poised for strong growth in the forthcoming quarters.
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