Inhibition of Mast Cell Degranulation by Novel Small Molecule MRGPRX2 Antagonists.

IgE independent Inflammation degranulation mast cells neuropeptides sensory neurons skin urticaria

Journal

The Journal of allergy and clinical immunology
ISSN: 1097-6825
Titre abrégé: J Allergy Clin Immunol
Pays: United States
ID NLM: 1275002

Informations de publication

Date de publication:
04 Jul 2024
Historique:
received: 21 02 2024
revised: 22 05 2024
accepted: 02 07 2024
medline: 7 7 2024
pubmed: 7 7 2024
entrez: 6 7 2024
Statut: aheadofprint

Résumé

Mas-related G-protein coupled receptor X2 (MRGPRX2) is a promiscuous receptor on mast cells that mediates IgE-independent degranulation and has been implicated in multiple mast cell-mediated disorders, including chronic urticaria, atopic dermatitis, and pain disorders. Although it is a promising therapeutic target, few potent, selective, small molecule antagonists have been identified, and functional effects of human MRGPRX2 inhibition have not been evaluated in vivo. We identified and characterized novel, potent, and selective orally active small molecule MRGPRX2 antagonists for potential treatment of mast cell-mediated disease. Antagonists were identified using multiple functional assays in cell lines overexpressing human MRGPRX2, LAD2 mast cells, human peripheral stem cell-derived mast cells, and isolated skin mast cells. Skin mast cell degranulation was evaluated in Mrgprb2em(-/-) knockout (KO) and Mrgprb2em(MRGPRX2) transgenic human MRGPRX2 knock-in (KI) mice by assessment of agonist-induced skin vascular permeability. Ex vivo skin mast cell degranulation and associated histamine release was evaluated by microdialysis of human skin tissue samples. MRGPRX2 antagonists potently inhibited agonist-induced MRGPRX2 activation and mast cell degranulation in all mast cell types tested, in an IgE-independent manner. Orally administered MRGPRX2 antagonists also inhibited agonist-induced degranulation and resulting vascular permeability in MRGPRX2 KI mice. In addition, antagonist treatment dose dependently inhibited agonist-induced degranulation in ex vivo human skin. MRGPRX2 small molecule antagonists potently inhibited agonist-induced mast cell degranulation in vitro and in vivo as well as ex vivo in human skin, supporting potential therapeutic utility as a novel treatment for multiple human diseases involving clinically relevant mast cell activation.

Sections du résumé

BACKGROUND BACKGROUND
Mas-related G-protein coupled receptor X2 (MRGPRX2) is a promiscuous receptor on mast cells that mediates IgE-independent degranulation and has been implicated in multiple mast cell-mediated disorders, including chronic urticaria, atopic dermatitis, and pain disorders. Although it is a promising therapeutic target, few potent, selective, small molecule antagonists have been identified, and functional effects of human MRGPRX2 inhibition have not been evaluated in vivo.
OBJECTIVE OBJECTIVE
We identified and characterized novel, potent, and selective orally active small molecule MRGPRX2 antagonists for potential treatment of mast cell-mediated disease.
METHODS METHODS
Antagonists were identified using multiple functional assays in cell lines overexpressing human MRGPRX2, LAD2 mast cells, human peripheral stem cell-derived mast cells, and isolated skin mast cells. Skin mast cell degranulation was evaluated in Mrgprb2em(-/-) knockout (KO) and Mrgprb2em(MRGPRX2) transgenic human MRGPRX2 knock-in (KI) mice by assessment of agonist-induced skin vascular permeability. Ex vivo skin mast cell degranulation and associated histamine release was evaluated by microdialysis of human skin tissue samples.
RESULTS RESULTS
MRGPRX2 antagonists potently inhibited agonist-induced MRGPRX2 activation and mast cell degranulation in all mast cell types tested, in an IgE-independent manner. Orally administered MRGPRX2 antagonists also inhibited agonist-induced degranulation and resulting vascular permeability in MRGPRX2 KI mice. In addition, antagonist treatment dose dependently inhibited agonist-induced degranulation in ex vivo human skin.
CONCLUSION CONCLUSIONS
MRGPRX2 small molecule antagonists potently inhibited agonist-induced mast cell degranulation in vitro and in vivo as well as ex vivo in human skin, supporting potential therapeutic utility as a novel treatment for multiple human diseases involving clinically relevant mast cell activation.

Identifiants

pubmed: 38971540
pii: S0091-6749(24)00675-4
doi: 10.1016/j.jaci.2024.07.002
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Informations de copyright

Copyright © 2024. Published by Elsevier Inc.

Auteurs

Joshua Wollam (J)

Escient Pharmaceuticals, San Diego, California, USA.

Michelle Solomon (M)

Escient Pharmaceuticals, San Diego, California, USA.

Christiane Villescaz (C)

Escient Pharmaceuticals, San Diego, California, USA.

Marion Lanier (M)

Escient Pharmaceuticals, San Diego, California, USA.

Samantha Evans (S)

Escient Pharmaceuticals, San Diego, California, USA.

Corinne Bacon (C)

Escient Pharmaceuticals, San Diego, California, USA.

David Freeman (D)

Escient Pharmaceuticals, San Diego, California, USA.

Alexis Vasquez (A)

Escient Pharmaceuticals, San Diego, California, USA.

Alan Vest (A)

Escient Pharmaceuticals, San Diego, California, USA.

Jim Napora (J)

Escient Pharmaceuticals, San Diego, California, USA.

Brittney Charlot (B)

Escient Pharmaceuticals, San Diego, California, USA.

Christine Cavarlez (C)

Escient Pharmaceuticals, San Diego, California, USA.

Andrew Kim (A)

Escient Pharmaceuticals, San Diego, California, USA.

Lisa Dvorak (L)

Escient Pharmaceuticals, San Diego, California, USA.

Brandon Selfridge (B)

Escient Pharmaceuticals, San Diego, California, USA.

Liming Huang (L)

Escient Pharmaceuticals, San Diego, California, USA.

Andres Nevarez (A)

Escient Pharmaceuticals, San Diego, California, USA.

Harry Dedman (H)

Escient Pharmaceuticals, San Diego, California, USA.

Jennifer Brooks (J)

Escient Pharmaceuticals, San Diego, California, USA.

Stefan Frischbutter (S)

Institute of Allergology, Charité - Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany; Fraunhofer Institute for Translational Medicine and Pharmacology (ITMP), Immunology and Allergology, Berlin, Germany.

Martin Metz (M)

Institute of Allergology, Charité - Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany; Fraunhofer Institute for Translational Medicine and Pharmacology (ITMP), Immunology and Allergology, Berlin, Germany.

Nadine Serhan (N)

Toulouse Institute for Infectious and Inflammatory Diseases (Infinity) - University Toulouse III, Toulouse, France.

Nicolas Gaudenzio (N)

Toulouse Institute for Infectious and Inflammatory Diseases (Infinity) - University Toulouse III, Toulouse, France; Genoskin SAS, Toulouse, France.

Gregg Timony (G)

Escient Pharmaceuticals, San Diego, California, USA.

Esther Martinborough (E)

Escient Pharmaceuticals, San Diego, California, USA.

Marcus F Boehm (MF)

Escient Pharmaceuticals, San Diego, California, USA.

Veena Viswanath (V)

Escient Pharmaceuticals, San Diego, California, USA. Electronic address: vviswanath@escientpharma.com.

Classifications MeSH