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November 12, 2020
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Iptacopan may provide safe treatment option for patients with C3 glomerulopathy

Iptacopan was shown to safely reduce proteinuria in patients with C3 glomerulopathy after 12 weeks of treatment, according to study results presented at the virtual ASN Kidney Week.

“LNP023 [Iptacopan] is a highly selective oral low-molecular-weight inhibitor of complement factor B, a key alternative pathway (AP) protease,” Edwin K. Wong, MRCP, PhD, of Newcastle upon Tyne NHS Foundation Trust, Newcastle University in the United Kingdom, and colleagues wrote in a poster. “The aim of the preliminary interim analysis of this phase 2 study was to determine whether LNP023 safely and effectively reduces proteinuria in patients with C3 glomerulopathy.”

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For the analysis, patients received open-label LNP023, with researchers assessing the change in urinary protein to creatinine ratio from baseline (mean age, 25 years; median eGFR, 80 mL/min/1.73m2). All patients also received ongoing LNP023 in a long-term extension study.

After 12 weeks, researchers found iptacopan reduced proteinuria by 53% compared with baseline values. Further findings indicated the drug improved plasma C3 levels and stabilized kidney function as measured by eGFR. In addition, data from the long-term extension study show an ongoing reduction in proteinuria.

Wong commented on the results in a related press release.

“This data also highlights iptacopan’s ability to strongly and specifically inhibit the alternative complement pathway, targeting the underlying cause of this disease and potentially providing a much needed treatment option for [C3 glomerulopathy] C3G patients who have significant unmet needs,” he said.