Novartis 'excited' about top line results for chronic kidney disease treatment

02 Oct 2023
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Clinical ResultPhase 3Breakthrough TherapyOrphan DrugAcquisition
The results were from a pre-specified interim analysis of a phase 3 study called Applause. Small molecule iptacopan is an investigational factor B inhibitor targeting the alternative complement pathway and demonstrated superiority versus placebo in proteinuria (protein in urine) reduction.
It has provided a clinically meaningful and highly statistically significant reduction on top of supportive care in patients with IgAN.
Kidney Research UK describes the conditions as a common chronic kidney disease mainly affecting young adults. In IgAN immunoglobulin A (IgA) becomes trapped in the very fine filters of the kidney (glomerul), causing damage and scarring to the whole kidney.
IgA is normally present in the bloodstream and its main role is to fight infections throughout the body. But in IgAN the body’s immune cells produce abnormally formed IgA. It is not yet known why this happens.
Slow IgAN progression
In the study, the safety profile of iptacopan, 200mg given twice daily was consistent with previously reported data. The study continues in a double-blind fashion to evaluate iptacopan’s ability to slow IgAN progression by measuring estimated glomerular filtration rate (eGFR) slope over 24 months – the primary endpoint at the study end with topline results expected in 2025.
Shreeram Aradhye, president, development and chief medical officer, Novartis​, said: “These positive data from the phase 3 Applause study reinforce the potential of iptacopan to provide clinically meaningful benefit to patients with IgAN, a debilitating disease that affects mostly young adults.
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“We are excited about this milestone in the development of our factor B inhibitor of the alternative complement pathway and remain focused on further advancing our portfolio of renal programs through pivotal trials.”
In its research, Novartis found that is estimated that approximately 25 people per million worldwide are newly diagnosed with IgAN each year. Up to 30% of people who have IgAN with persistent higher levels of proteinuria 1g or more per day, may progress to kidney failure within 10 years.
Novartis said there is nothing that addresses a key pathogenic step in the progression of IgAN – activation of the complement system. There is therefore a need for effective, targeted therapies that slow or prevent progression to kidney failure.
Novartis has discovered and developed iptacopan with its aim to address IgAN​ and other complement-mediated diseases by inhibiting the factor B – a protease essential to the alternative complement pathway.
The treatment us under review by regulators following the positive phase 3 results in paroxysmal nocturnal hemoglobinuria. It is also being investigated in phase 3 studies for C3 glomerulopathy, atypical hemolytic uremic syndrome and immune complex membranoproliferative glomerulonephritis.
With its recent acquisition of Chinook Therapeutics, the Novartis renal portfolio expands with two additional late-stage medicines in development for IgAN, complementing the existing pipeline.
Novartis intends to submit for possible accelerated approval with the FDA in 2024.
Based on disease prevalence, unmet needs and data from phase 2 studies, iptacopan has received US Food and Drug Administration (FDA) breakthrough therapy designation in paroxysmal nocturnal hemoglobinuria (PNH) - a rare blood disorder named for a single symptom - red/brown/dark urine noticed during late night or early morning trips to the bathroom - FDA breakthrough therapy designation in rare kidney disease C3G, orphan drug designations from the FDA and European Medicines Agancy (EMA) in PNH and C3G, EMA Prime designation for C3G, and EMA orphan drug designation in IgAN23.
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