Biological impact of iberdomide in patients with active systemic lupus erythematosus


Por: Lipsky P.E., Vollenhoven R.V., Dörner T., Werth V.P., Merrill J.T., Furie R., Petronijevic M., Velasco Zamora B., Majdan M., Irazoque-Palazuelos F., Terbrueggen R., Delev N., Weiswasser M., Korish S., Stern M., Hersey S., Ye Y., Gaudy A., Liu Z., Gagnon R., Tang S., Schafer P.H.

Publicada: 1 ene 2022
Resumen:
Objectives Iberdomide is a high-Affinity cereblon ligand that promotes proteasomal degradation of transcription factors Ikaros (IKZF1) and Aiolos (IKZF3). Pharmacodynamics and pharmacokinetics of oral iberdomide were evaluated in a phase 2b study of patients with active systemic lupus erythematosus (SLE). Methods Adults with autoantibody-positive SLE were randomised to placebo (n=83) or once daily iberdomide 0.15 mg (n=42), 0.3 mg (n=82) or 0.45 mg (n=81). Pharmacodynamic changes in whole blood leucocytes were measured by flow cytometry, regulatory T cells (Tregs) by epigenetic assay, plasma cytokines by ultrasensitive cytokine assay and gene expression by Modular Immune Profiling. Results Iberdomide exhibited linear pharmacokinetics and dose-dependently modulated leucocytes and cytokines. Compared with placebo at week 24, iberdomide 0.45 mg significantly (p<0.001) reduced B cells, including those expressing CD268 (TNFRSF13C) (-58.3%), and plasmacytoid dendritic cells (-73.9%), and increased Tregs (+104.9%) and interleukin 2 (IL-2) (+144.1%). Clinical efficacy was previously reported in patients with high IKZF3 expression and high type I interferon (IFN) signature at baseline and confirmed here in those with an especially high IFN signature. Iberdomide decreased the type I IFN gene signature only in patients with high expression at baseline (-81.5%; p<0.001) but decreased other gene signatures in all patients. Conclusion Iberdomide significantly reduced activity of type I IFN and B cell pathways, and increased IL-2 and Tregs, suggesting a selective rebalancing of immune abnormalities in SLE. Clinical efficacy corresponded to reduction of the type I IFN gene signature. Trial registration number NCT03161483. © 2022 BMJ Publishing Group. All rights reserved.

Filiaciones:
Lipsky P.E.:
 RILITE Foundation and AMPEL BioSolutions, Charlottesville, VA, United States

Vollenhoven R.V.:
 Amsterdam University Medical Centers, Amsterdam, Netherlands

Dörner T.:
 German Rheumatism Research Center, Charité University Hospital, Berlin, Germany

Werth V.P.:
 University of Pennsylvania and the Michael J. Crescenz VA Medical Center, Philadelphia, PA, United States

Merrill J.T.:
 Oklahoma Medical Research Foundation, Oklahoma City, OK, United States

Furie R.:
 Department of Rheumatology, Northwell Health, Great Neck, NY, United States

Petronijevic M.:
 Military Medical Academy, Belgrade, Serbia

Velasco Zamora B.:
 Instituto CER S.A, Buenos Aires, Argentina

Majdan M.:
 Samodzielny Publiczny Szpital Kliniczny Nr 4 W Lublinie, Medical University of Lublin, Lublin, Poland

Irazoque-Palazuelos F.:
 Centro de Investigación y Tratamiento Reumatológico SC, Mexico City, Mexico

Terbrueggen R.:
 DxTerity Diagnostics, Rancho DominguezCA, United States

Delev N.:
 Bristol Myers Squibb, Princeton, NJ, United States

Weiswasser M.:
 Bristol Myers Squibb, Princeton, NJ, United States

Korish S.:
 Bristol Myers Squibb, Princeton, NJ, United States

Stern M.:
 Bristol Myers Squibb, Princeton, NJ, United States

Hersey S.:
 Bristol Myers Squibb, Princeton, NJ, United States

Ye Y.:
 Bristol Myers Squibb, Princeton, NJ, United States

Gaudy A.:
 Bristol Myers Squibb, Princeton, NJ, United States

Liu Z.:
 Bristol Myers Squibb, Princeton, NJ, United States

Gagnon R.:
 Bristol Myers Squibb, Princeton, NJ, United States

Tang S.:
 Bristol Myers Squibb, Princeton, NJ, United States

Schafer P.H.:
 Bristol Myers Squibb, Princeton, NJ, United States
ISSN: 00034967
Editorial
BMJ PUBLISHING GROUP, BRITISH MED ASSOC HOUSE, TAVISTOCK SQUARE, LONDON WC1H 9JR, ENGLAND, Reino Unido
Tipo de documento: Article
Volumen: 81 Número: 8
Páginas: 1136-1142
WOS Id: 000788577900001
ID de PubMed: 35477518
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