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Efficacy and safety of iscalimab, a novel anti-CD40 monoclonal antibody, in moderate-to-severe myasthenia gravis: A phase 2 randomized study

Background - Increased morbidity in many patients with myasthenia gravis (MG) on long-term immunosuppression highlights the need for improved treatments. The aim of this study is to investigate the safety and efficacy of iscalimab (CFZ533), a fully human anti-CD40 monoclonal antibody, in patients wi...

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Main Authors: Gomez-Mancilla, Baltazar (Author) , Meriggioli, Matthew N. (Author) , Genge, Angela (Author) , Roubenoff, Ronenn (Author) , Espié, Pascal (Author) , Dupuy, Cyrielle (Author) , Hartmann, Nicole (Author) , Pezous, Nicole (Author) , Kinhikar, Arvind (Author) , Tichy, Mia (Author) , Dionne, Annie (Author) , Vissing, John (Author) , Andersen, Henning (Author) , Schoser, Benedikt (Author) , Meisel, Andreas (Author) , Devlikamova, Farida (Author) , Poverennova, Irina (Author) , Stuchevskaya, Fatima (Author) , Lin, Thy-Sheng (Author) , Rush, James S. (Author) , Gergely, Peter (Author)
Other Authors: Jordan, Berit (Other)
Format: Article (Journal)
Language:English
Published: 20 November 2023
In: Journal of clinical neuroscience
Year: 2024, Volume: 119, Pages: 76-84
ISSN:1532-2653
DOI:10.1016/j.jocn.2023.11.013
Online Access:Resolving-System, lizenzpflichtig, Volltext: https://doi.org/10.1016/j.jocn.2023.11.013
Verlag, lizenzpflichtig, Volltext: https://www.sciencedirect.com/science/article/pii/S0967586823003430
Resolving-System, lizenzpflichtig: https://doi.org/10.1016/j.jocn.2023.11.013
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Author Notes:Baltazar GomezMancilla, Matthew N. Meriggioli, Angela Genge, Ronenn Roubenoff, Pascal Espié, Cyrielle Dupuy, Nicole Hartmann, Nicole Pezous, Arvind Kinhikar, Mia Tichy, Annie Dionne, John Vissing, Henning Andersen, Benedikt Schoser, Andreas Meisel, Berit Jordan, Farida Devlikamova, Irina Poverennova, Fatima Stuchevskaya, Thy-Sheng Lin, James S. Rush, Peter Gergely
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Summary:Background - Increased morbidity in many patients with myasthenia gravis (MG) on long-term immunosuppression highlights the need for improved treatments. The aim of this study is to investigate the safety and efficacy of iscalimab (CFZ533), a fully human anti-CD40 monoclonal antibody, in patients with moderate-to-severe MG receiving standard-of-care (SoC) therapies. - Methods - In this double-blind, placebo-controlled phase 2 study, symptomatic patients (n = 44) despite SoC were randomized 1:1 to receive intravenous iscalimab (10 mg/kg; n = 22) or placebo (n = 22) every 4 weeks for 6 doses in total. Patients were followed up for 6 months after the last dose. The total duration of the study was 52 weeks. - Results - In total, 34 of 44 patients (77.3 %) completed the study. The primary endpoint, Quantitative MG score, did not change significantly between baseline and week 25 for iscalimab (median [90 % CI], −4.07 [−5.67, −2.47]) versus placebo (−2.93 [−4.53, −1.33]); however, non-thymectomized patients (n = 29) showed more favorable results (iscalimab, −4.35 [−6.07, −2.64] vs placebo, −2.26 [−4.16, −0.36]). A statistically significant difference between iscalimab and placebo groups was observed in MG Composite score (adjusted mean change: −4.19 [−6.67, −1.72]; p = 0.007) at week 13, and MG-Activities of Daily Living score (−1.93 [−3.24, −0.62]; p = 0.018) at week 21. Adverse events were comparable between the iscalimab (91 %) and placebo (96 %) groups. - Conclusion - Iscalimab showed favorable safety and improvements compared with placebo in non-thymectomized patients with moderate-to-severe MG. It did not show any protective effect in patients with moderate-to-severe MG.
Item Description:Gesehen am 08.07.2024
Physical Description:Online Resource
ISSN:1532-2653
DOI:10.1016/j.jocn.2023.11.013

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