Novartis (NVS) said that an investigational iptacopan or LNP023- an oral, targeted factor B inhibitor- met Phase II study primary endpoint in rare kidney disease IgA nephropathy or IgAN.
The phase II data showed that iptacopan reduced protein in the urine or proteinuria, an increasingly recognized surrogate marker correlating with progression to kidney failure, and showed promise in stabilizing kidney function in patients with IgA nephropathy.
In the phase II study, dubbed as NCT03373461, patients with IgAN were randomized to placebo or different doses of iptacopan. The primary endpoint was met with a statistically significant dose response effect on reduction in proteinuria with iptacopan vs. placebo, at 90 days. At the highest dose of 200mg twice daily a 23% reduction in proteinuria was predicted, compared with placebo, at 90 days.
Iptacopan also demonstrated a trend towards stabilizing kidney function, as assessed by estimated glomerular filtration rate, a key measure of kidney clearance function that estimates the rate of blood passing through and being filtered by the kidneys. Additionally, iptacopan showed a favorable safety and tolerability profile.
Iptacopan is the most advanced asset in the company’s nephrology pipeline and targets the alternative complement pathway, a key driver of complement-driven renal diseases (CDRDs). Novartis has plans to initiate additional Phase III studies in other renal indications.
The company noted that Iptacopan is also in development for a life-threatening blood disorder, paroxysmal nocturnal hemoglobinuria (PNH). Based on disease prevalence and positive data from Phase II studies, iptacopan has received EMA orphan drug designation in IgAN10, orphan drug designations from the FDA and EMA in C3G and PNH11, FDA Breakthrough Therapy Designation in PNH12, and EMA PRIME designation for C3G.
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