Basel, December 16, 2020 — Novartis today announced that the U.S. Food and Drug Administration (FDA) granted iptacopan (LNP023) Breakthrough Therapy Designation (BTD) in paroxysmal nocturnal hemoglobinuria (PNH) and Rare Pediatric Disease (RPD) Designation in C3 glomerulopathy (C3G).

LNP023: oral low molecular weight Factor B inhibitor Potent and selective FB inhibitor that inhibits ex vivo stimulated Membrane Attack Complex (MAC) formation in serum and whole blood

Nov 5, 2021 · Iptacopan (LNP023) is a new, oral, selective and potent first-in-class inhibitor of factor B, a key component of the AP. Recent phase 2 data showed that iptacopan effectively controls both IVH and EVH and leads to rapid, transfusion-free improvements in Hb levels in the majority of PNH patients.

Aug 2, 2022 · Single-agent oral factor B inhibition led to rapid improvements of hemolytic markers, Hb levels, and transfusion requirements in PNH. Iptacopan (LNP023) is a novel, oral selective inhibitor of complement factor B under clinical development for paroxysmal nocturnal hemoglobinuria (PNH).

Inhibition of AP activity was demonstrated in both blood and urine complement markers upon treatment with LNP023, with maximal effects observed at 100 to 200 mg bid

Mar 25, 2025 · The study is designed as a multicenter, single-arm, open label study to demonstrate the efficacy and safety of LNP023 (iptacopan) at a dose of 200 mg b.i.d. in adult patients with aHUS who are treatment naive to complement …

Basel, December 16, 2020 — Novartis today announced that the U.S. Food and Drug Administration (FDA) granted iptacopan (LNP023) Breakthrough Therapy Designation (BTD) in paroxysmal...

LNP023 is a trial drug designed to block a protein in the complement system. Researchers think it may prevent or reduce hemolysis caused by PNH. • How many participants had a sign of hemolysis go down after receiving LNP023 for 12 weeks? • What medical problems did the participants have during this trial?

The purpose of this study is to assess the clinical efficacy, pharmacodynamics, safety, tolerability and pharmacokinetics of LNP023 in patients with C3 glomerulopathy (C3G) and to evaluate the effect of different doses of LNP023 on complement biomarkers CCI .

An open-label, non-randomized study on efficacy, pharmacokinetics, pharmacodynamics, safety, and tolerability of LNP023 in two patient populations with C3 glomerulopathy [NCT03832114]

Basel, June 11, 2021 — Novartis today announced new Phase II data for iptacopan (LNP023), an investigational oral treatment for paroxysmal nocturnal hemoglobinuria (PNH), presented at the 26th Annual Congress of the European Hematology Association (EHA).

Iptacopan (LNP023) is a first-in-class, orally bioavailable, highly potent and highly selective factor B inhibitor with an IC50 value of 10 nM. Iptacopan shows direct, reversible, and high-affinity binding to human factor B with a KD of 7.9 nM.

May 29, 2021 · Iptacopan (LNP023) is a highly selective oral low molecular weight inhibitor of Factor B, a key complement alternative pathway protease.

Basel, June 11, 2021 — Novartis today announced new Phase II data for iptacopan (LNP023), an investigational oral treatment for paroxysmal nocturnal hemoglobinuria (PNH), presented at the...

Dec 5, 2023 · This clinical trial examines LNP023's effectiveness and safety in lupus nephritis treatment alongside standard care in two-part study.

Basel, October 26, 2020 — Novartis today announced positive Phase II interim analysis results for iptacopan (LNP023), an investigational oral treatment for C3 glomerulopathy (C3G), presented at the virtually held American Society of Nephrology (ASN) 2020 Annual Meeting.

Dec 2, 2023 · Here, we report kidney function and outcomes over 110 weeks from the double-blind final analysis. Methods: PROTECT, a double-blind, randomised, active-controlled, phase …

Discovery of 4- ( (2S,4S)-4-Ethoxy-1- ( (5-methoxy-7-methyl-1H-indol-4-yl)methyl)piperidin-2-yl)benzoic acid (LNP023), a factor B inhibitor specifically designed to be applicable to treating a diverse array of complement mediated diseases

We aimed to evaluate the safety, clinical efficacy, and pharmacodynamic effects of obexelimab in patients with active IgG4-related disease. We conducted an open-label, single-arm, single centre, phase 2 pilot trial at the Massachusetts General Hospital in Boston, MA, USA.

Feb 28, 2025 · A Multicenter, Randomized, Double-Blind, Parallel Group, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Iptacopan (LNP023) in Idiopathic Immune Complex Mediated Membranoproliferative Glomerulonephritis (IC-MPGN) (APPARENT).