An experimental medicine from Novartis has succeeded in a late-stage clinical trial testing it against an uncommon kidney disease.
Novartis expects the high-level results released Monday will further support the company’s plan to submit a second approval application to the Food and Drug Administration sometime next year. Regulators in the U.S. and Europe are already reviewing the medicine, called iptacopan, as a potential treatment for another rare disorder known as PNH, or paroxysmal nocturnal hemoglobinuria.
Iptacopan is meant to help control the immune system, specifically the “complement” component that acts as the initial line of defense against pathogens and unwanted cells. Though the complement system is essential to human health, when it works in overdrive it can lead to a variety of illnesses. Novartis has been testing iptacopan across at least five diseases, including IgA nephropathy, which is marked by inflammation and damage to the kidneys that, eventually, may require patients to go on dialysis.
Novartis’ trial, codenamed APPLAUSE-IgAN, enrolled close to 500 participants who, on top of supportive care, either received iptacopan or a placebo. Novartis said an interim analysis of the study results so far found drug-treated patients had “clinically meaningful and highly statistically significant” reductions in the amount of protein in their urine — an important measure of kidney health.
Novartis also said the safety of its drug was “consistent with previously reported data.” Earlier, much smaller trials had described iptacopan as well-tolerated and “without any major drug-related safety findings.”
A final readout of the larger study should come in 2025, according to Novartis.
“These positive data ... reinforce the potential of iptacopan to provide clinically meaningful benefit to patients with IgAN, a debilitating disease that affects mostly young adults,” said Shreeram Aradhye, the company’s chief medical officer, in a statement.
Novartis also believes iptacopan could be meaningful to the company’s bottom line, having estimated peak annual sales of over $3 billion. If approved in PNH, the drug would compete against the blockbuster treatments Soliris and Ultomiris, which have been sold by AstraZeneca since its $39 billion acquisition of Alexion Pharmaceuticals.
An approval in IgA nephropathy, meanwhile, would help affirm a recent push by Novartis to invest more in kidney drugs. In June, the Swiss pharmaceutical giant announced plans to purchase Chinook Therapeutics for as much as $3.5 billion. Chinook has a pair of experimental medicines designed to treat IgA nephropathy in different ways.
As with other kidney diseases, treatment of IgA nephropathy commonly involves the use of so-called ACE or ARB drugs. But in recent years, the FDA has approved two other therapies: Tarpeyo from Calliditas Therapeutics and Filspari from Travere Therapeutics.