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IPTACOPAN

Excessive local activation and dysregulation of the alternative complement pathway in the glomeruli can cause excessive deposition of multiple complement components in the glomerulus.  Iptacopan (LNP023) is a potent oral highly selective inhibitor of factor B of alternative complement pathway. 

Wavy Abstract Background

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Novartis Pharmaceuticals

1-888-669-6682

+41-613-241-111

novartis.email@novartis.com

How is Iptacopan being studied in C3GN?

In a phase 2 study, treatment with Iptacopan was associated with a statistically significant reduction in proteinuria and stabilization of eGFR in patients with C3G.

 

APPEAR-C3G is a randomized, double-blind, placebo-controlled pivotal Phase 3 study to evaluate the efficacy and safety of iptacopan in patients with native kidney C3G. 83 patients will be randomized 1:1 to receive either iptacopan 200 mg bid or placebo for 6 months, followed by open-label treatment with iptacopan 200 mg bid for all patients for 6 months. The primary objective is to demonstrate the superiority of iptacopan versus placebo on proteinuria reduction at 6 and 12 months. 

Image from Smith et al, Kidney week 2021

Eligibility Criteria

  1. Male and female participants age ≥ 12 and ≤ 60 years at screening.

  2. Diagnosis of C3G as confirmed by renal biopsy within 12 months prior to enrollment in adults and within 3 years in adolescents.

  3. Maximally recommended or tolerated dose of ACEI or ARB for at least 90 days. Stable doses of anti-proteinuric medications like MMF and Cortocosteroids.

  4. ​Reduced serum C3  at screening.

  5. UPCR ≥ 1.0 g/g sampled from the first morning void urine sample at Day -75 and Day -15.

  6. Estimate GFR or measured GFR ≥ 30 ml/min/1.73m2 

  7. Mandatory vaccination against Neisseria meningitidis and Streptococcus pneumoniae prior to the start of study treatment.

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