Study on Efficacy and Safety of LNP023 in C3 Glomerulopathy Patients Transplanted and Not Transplanted

Phase 2 Completed 27 enrolled Novartis Pharmaceuticals

Non-randomizedSingle-groupOpen-labelTreatment

Summary

The study is an open-label, two cohort non-randomized study evaluating the efficacy, safety, and pharmacokinetics of LNP023 in patients with C3G (Cohort A) and patients who have undergone kidney transplant and have C3G recurrence (Cohort B).

Timeline

Start: 2019-02-20
Primary completion: 2021-04-23
Completion: 2021-04-23

Outcome

Met primary endpoint

paper In the native cohort, UPCR levels decreased by 45% from baseline to week 12 (P = 0.0003). PMID 38106570 ↗

paper In the recurrent KT cohort, the median C3 deposit score decreased by 2.50 (scale: 0-12) on day 84 versus baseline (P = 0.03). PMID 38106570 ↗

release “iptacopan (LNP023) – a first-in-class, oral, selective factor B inhibitor – in patients with C3 glomerulopathy (C3G) met primary endpoints in both patient cohorts” novartis.com ↗

Publications

Wong E, Nester C, Cavero T, Karras A, Le Quintrec M, Lightstone L, Eisenberger U, Soler MJ, Kavanagh D, Daina E, Praga M, Medjeral-Thomas NR, Gackler A, Garcia-Carro C, Biondani A, Chaperon F, Kulmatycki K, Milojevic J, Webb NJA, Nidamarthy PK, Junge G, Remuzzi G. Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy. Kidney Int Rep. 2023 Sep 22;8(12):2754-2764. doi: 10.1016/j.ekir.2023.09.017. eCollection 2023 Dec.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Iptacopan	Small molecule	200 mg	—