Results 41 to 50 of about 898 (115)

Complement Inhibition in the Clinic: Are We Doing Enough to Protect Patients From Infection?

European Journal of Immunology, Volume 56, Issue 7, July 2026.
Excessive complement activation is implicated in a broad range of diseases. Therapeutic approaches targeting the complement cascade, from pathway‐selective inhibition to terminal blockade, can effectively control disease activity. However, increasing degrees of complement inhibition are associated with a heightened susceptibility to bacterial, viral ...
Serena Bettoni, Ebba Qviberg, Edward Chaloner, Hayley Lavender, Maisem Laabei   +4 more
wiley   +1 more source

Complement Factor H‐Based Therapeutics: A Comprehensive Overview

European Journal of Immunology, Volume 56, Issue 7, July 2026.
Complement factor H is a central regulator of the complement system and thus a target for therapeutic intervention. In this review, we provide an overview of past and current factor H‐based therapeutic concepts and modalities to treat complement‐mediated diseases.
Sebastiaan M. W. R. Hamers, Emmy J. Buter, Richard B. Pouw   +2 more
wiley   +1 more source

Shifting From Systemic to Precision‐Targeted Complement Therapies: Opportunities and Hurdles

European Journal of Immunology, Volume 56, Issue 6, June 2026.
Complement therapeutics have expanded considerably, but systemic inhibitors remain limited by infection risks, breakthrough events, and loss of physiological functions. Emerging targeted approaches aim for organ‐, tissue‐, or cell‐specific modulation of complement activity, potentially offering greater precision while reducing treatment burden and ...
Marco Mannes, Wioleta M. Zelek, Leendert A. Trouw   +2 more
wiley   +1 more source

Patient-reported improvements in paroxysmal nocturnal hemoglobinuria treated with iptacopan from 2 phase 3 studies

Blood Advances
: Iptacopan, a first-in-class, oral, selective complement factor B inhibitor, demonstrated efficacy and safety as monotherapy in C5 inhibitor (C5i)–experienced (APPLY-PNH; NCT04558918) and C5i-naive (APPOINT-PNH; NCT04820530) patients with paroxysmal ...
Antonio M. Risitano, Carlos de Castro, Bing Han, Austin Kulasekararaj, Jaroslaw P. Maciejewski, Phillip Scheinberg, Yasutaka Ueda, Susan Vallow, Georgina Bermann, Marion Dahlke, Rakesh Kumar, Régis Peffault de Latour   +11 more
doaj   +1 more source

Iptacopan: First Approval

   Declarations Funding The preparation of this review was not supported by any external funding. Authorship and Conflict of interest Yahiya Y. Syed is a salaried employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of ...
Yahiya Y. Syed (6594444)
core   +1 more source

Randomised, phase 1 evaluation of the safety, tolerability, pharmacokinetics and pharmacodynamics of iptacopan in healthy volunteers

British Journal of Pharmacology, Volume 183, Issue 11, Page 2874-2889, June 2026.
Abstract Background and Purpose Overactivation of the alternative pathway (AP) underlies several diseases. Iptacopan is an oral, first‐in‐class, highly potent specific inhibitor of factor B, a key AP protease. Experimental Approach The analysis included data from two phase 1 randomised, volunteer‐blinded, placebo‐controlled studies: Study 1, a single ...
Irina Baltcheva, Guido Junge, Kenneth Kulmatycki, Robert Schmouder, Anna Schubart, Marie‐Anne Valentin   +5 more
wiley   +1 more source

Novel drugs approved by the EMA, the FDA and the MHRA in 2025: A year in review

British Journal of Pharmacology, Volume 183, Issue 9, Page 1779-1813, May 2026.
Abstract In the 2025 novel drug mini‐review, one can take a full measure of the ingenuity that underlies current drug design and development, despite the year's smaller harvest (46 novel drugs) compared to 2024 (53) and 2023 (70). 54% of the novel drugs are first‐in‐class (FIC).
Andreas Papapetropoulos, Stavros Topouzis, Steve P. H. Alexander, Miriam M. Cortese‐Krott, Zsuzsanna Helyes, Kirill Martemyanov, Claudio Mauro, Nithyanandan Nagercoil, Reynold A. Panettieri Jr, Hemal H. Patel, Rainer Schulz, Barbara Stefanska, Gary J. Stephens, Nathalie Vergnolle, Xin Wang, Stephen Ward, Péter Ferdinandy   +16 more
wiley   +1 more source

Adventitial Niches, Complement and Inflammation in Pulmonary Vascular Disease: Current Status and Future Directions

Comprehensive Physiology, Volume 16, Issue 2, April 2026.
Local complement production by pulmonary artery adventitial fibroblasts, activated intracellularly by CFD and CFB and extracellularly by GZMK+ CD8 T cells, and its secretion in soluble form and within EVs promotes macrophage and T cell chemotaxis and activation.
Hui Zhang, Ram Raj Prasad, Sushil Kumar, Min Li, Dallas Jones, Cheng‐Jun Hu, Claudia Mickael, Yen‐Rei Yu, Rubin M. Tuder, Kurt R. Stenmark   +9 more
wiley   +1 more source

Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor‐Naive Patients With Paroxysmal Nocturnal Haemoglobinuria

eJHaem
Background Paroxysmal nocturnal haemoglobinuria (PNH) is characterised by haemolytic anaemia, bone marrow failure and thrombosis. The single‐arm phase 3 APPOINT‐PNH trial (NCT04820530) investigating iptacopan monotherapy in complement inhibitor‐naive ...
Matthew Holt, Richard J. Kelly, Jilles M. Fermont, Soudeh Ansari, Marion Dahlke, Isabelle Brindel, Lynda Maafa, Flore Sicre deFontbrune, Régis Peffault deLatour   +8 more
doaj   +1 more source

Results of a randomized double-blind placebo-controlled Phase 2 study propose iptacopan as an alternative complement pathway inhibitor for IgA nephropathy

, 2023
Targeting the alternative complement pathway is an attractive therapeutic strategy given its role in the pathogenesis of immunoglobulin A nephropathy (IgAN). Iptacopan (LNP023) is an oral, proximal alternative complement inhibitor that specifically binds
Vlado Perkovic (141532), Hong Zhang (25820), Dana V Rizk (11138403), Brad Rovin (8412759), Jonathan Barratt (3387179), Julie Milojevic (17381014), Bart Maes (17381011), Olympia Papachristofi (3521138), Hernán Trimarchi (7803695), Ben Sprangers (4403833), Matthias Meier (658232), Prasanna Kumar Nidamarthy (17381020), Dmitrij Kollins (156218), Alan Charney (17381023), Naoki Kashihara (3161976), Guido Junge (17381017)   +15 more
core   +1 more source