An orally available small molecule that targets soluble TNF to deliver anti-TNF biologic-like efficacy in rheumatoid arthritis

Tumor necrosis factor (TNF) is a pleiotropic cytokine belonging to a family of trimeric proteins with both proinflammatory and immunoregulatory functions. TNF is a key mediator in autoimmune diseases and during the last couple of decades several biologic drugs have delivered new therapeutic options...

Full description

Saved in:
Bibliographic Details
Main Authors: Alexander Vugler, James O’Connell, Mai Anh Nguyen, Dietmar Weitz, Thomas Leeuw, Elizabeth Hickford, Alexander Verbitsky, Xiaoyou Ying, Markus Rehberg, Bruce Carrington, Mark Merriman, Andrew Moss, Jean-Marie Nicholas, Phil Stanley, Sara Wright, Tim Bourne, Yann Foricher, Zhaoning Zhu, Daniel Brookings, Helen Horsley, Jag Heer, Laurent Schio, Matthias Herrmann, Srinivas Rao, Markus Kohlmann, Peter Florian
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-11-01
Series:Frontiers in Pharmacology
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fphar.2022.1037983/full
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1850185148428976128
author Alexander Vugler
James O’Connell
Mai Anh Nguyen
Dietmar Weitz
Thomas Leeuw
Elizabeth Hickford
Alexander Verbitsky
Xiaoyou Ying
Markus Rehberg
Bruce Carrington
Mark Merriman
Andrew Moss
Jean-Marie Nicholas
Phil Stanley
Sara Wright
Tim Bourne
Yann Foricher
Zhaoning Zhu
Daniel Brookings
Helen Horsley
Jag Heer
Laurent Schio
Matthias Herrmann
Srinivas Rao
Markus Kohlmann
Peter Florian
author_facet Alexander Vugler
James O’Connell
Mai Anh Nguyen
Dietmar Weitz
Thomas Leeuw
Elizabeth Hickford
Alexander Verbitsky
Xiaoyou Ying
Markus Rehberg
Bruce Carrington
Mark Merriman
Andrew Moss
Jean-Marie Nicholas
Phil Stanley
Sara Wright
Tim Bourne
Yann Foricher
Zhaoning Zhu
Daniel Brookings
Helen Horsley
Jag Heer
Laurent Schio
Matthias Herrmann
Srinivas Rao
Markus Kohlmann
Peter Florian
author_sort Alexander Vugler
collection DOAJ
description Tumor necrosis factor (TNF) is a pleiotropic cytokine belonging to a family of trimeric proteins with both proinflammatory and immunoregulatory functions. TNF is a key mediator in autoimmune diseases and during the last couple of decades several biologic drugs have delivered new therapeutic options for patients suffering from chronic autoimmune diseases such as rheumatoid arthritis and chronic inflammatory bowel disease. Attempts to design small molecule therapies directed to this cytokine have not led to approved products yet. Here we report the discovery and development of a potent small molecule inhibitor of TNF that was recently moved into phase 1 clinical trials. The molecule, SAR441566, stabilizes an asymmetrical form of the soluble TNF trimer, compromises downstream signaling and inhibits the functions of TNF in vitro and in vivo. With SAR441566 being studied in healthy volunteers we hope to deliver a more convenient orally bioavailable and effective treatment option for patients suffering with chronic autoimmune diseases compared to established biologic drugs targeting TNF.
format Article
id doaj-art-1ee03f08f53b498dba01c240aad2ca37
institution OA Journals
issn 1663-9812
language English
publishDate 2022-11-01
publisher Frontiers Media S.A.
record_format Article
series Frontiers in Pharmacology
spelling doaj-art-1ee03f08f53b498dba01c240aad2ca372025-08-20T02:16:49ZengFrontiers Media S.A.Frontiers in Pharmacology1663-98122022-11-011310.3389/fphar.2022.10379831037983An orally available small molecule that targets soluble TNF to deliver anti-TNF biologic-like efficacy in rheumatoid arthritisAlexander Vugler0James O’Connell1Mai Anh Nguyen2Dietmar Weitz3Thomas Leeuw4Elizabeth Hickford5Alexander Verbitsky6Xiaoyou Ying7Markus Rehberg8Bruce Carrington9Mark Merriman10Andrew Moss11Jean-Marie Nicholas12Phil Stanley13Sara Wright14Tim Bourne15Yann Foricher16Zhaoning Zhu17Daniel Brookings18Helen Horsley19Jag Heer20Laurent Schio21Matthias Herrmann22Srinivas Rao23Markus Kohlmann24Peter Florian25Immunology Therapeutic Area, PV Early Solutions, UCB Pharma, Slough, United KingdomDiscovery Sciences, PV Early Solutions, UCB Pharma, Slough, United KingdomSanofi R&D, TMED Pharmacokinetics Dynamics and Metabolism, Frankfurt am Main, GermanySanofi R&D, Drug Metabolism and Pharmacokinetics, Frankfurt am Main, GermanySanofi R&D, Type 1/17 Immunology, Immunology & Inflammation Research TA, Frankfurt, GermanyDevelopment Science, PV Early Solutions, UCB Pharma, Slough, United KingdomSanofi R&D, Translation In vivo Models, Cambridge, MA, United StatesSanofi R&D, Translation In vivo Models, Cambridge, MA, United StatesSanofi R&D, Translational Disease Modelling, Frankfurt am Main, GermanyDiscovery Sciences, PV Early Solutions, UCB Pharma, Slough, United KingdomImmunology Therapeutic Area, PV Early Solutions, UCB Pharma, Slough, United KingdomTranslational Medicine Immunology, PV Early Solutions, UCB Pharma, Slough, United Kingdom0Development Science, Drug Metabolism and Pharmacokinetics, UCB Pharma, Braine-I’Alleud, BelgiumImmunology Therapeutic Area, PV Early Solutions, UCB Pharma, Slough, United Kingdom1Early PV Missions, PV Early Solutions, UCB Pharma, Slough, United Kingdom2Milvuswood Consultancy, Penn, United Kingdom3Sanofi R&D, Integrated Drug Discovery, Vitry-sur-Seine, France4Global Chemistry, Discovery Sciences, PV Early Solutions, UCB Pharma, Slough, United Kingdom4Global Chemistry, Discovery Sciences, PV Early Solutions, UCB Pharma, Slough, United Kingdom4Global Chemistry, Discovery Sciences, PV Early Solutions, UCB Pharma, Slough, United Kingdom4Global Chemistry, Discovery Sciences, PV Early Solutions, UCB Pharma, Slough, United Kingdom3Sanofi R&D, Integrated Drug Discovery, Vitry-sur-Seine, FranceSanofi R&D, Type 1/17 Immunology, Immunology & Inflammation Research TA, Frankfurt, GermanySanofi R&D, Translation In vivo Models, Cambridge, MA, United States5Sanofi R&D, Early Clinical Development, Therapeutic Area Immunology and Inflammation, Frankfurt am Main, GermanySanofi R&D, Type 1/17 Immunology, Immunology & Inflammation Research TA, Frankfurt, GermanyTumor necrosis factor (TNF) is a pleiotropic cytokine belonging to a family of trimeric proteins with both proinflammatory and immunoregulatory functions. TNF is a key mediator in autoimmune diseases and during the last couple of decades several biologic drugs have delivered new therapeutic options for patients suffering from chronic autoimmune diseases such as rheumatoid arthritis and chronic inflammatory bowel disease. Attempts to design small molecule therapies directed to this cytokine have not led to approved products yet. Here we report the discovery and development of a potent small molecule inhibitor of TNF that was recently moved into phase 1 clinical trials. The molecule, SAR441566, stabilizes an asymmetrical form of the soluble TNF trimer, compromises downstream signaling and inhibits the functions of TNF in vitro and in vivo. With SAR441566 being studied in healthy volunteers we hope to deliver a more convenient orally bioavailable and effective treatment option for patients suffering with chronic autoimmune diseases compared to established biologic drugs targeting TNF.https://www.frontiersin.org/articles/10.3389/fphar.2022.1037983/fullTNFrheumatoid arthritissmall moleculeTNF trimerimmunogenicity
spellingShingle Alexander Vugler
James O’Connell
Mai Anh Nguyen
Dietmar Weitz
Thomas Leeuw
Elizabeth Hickford
Alexander Verbitsky
Xiaoyou Ying
Markus Rehberg
Bruce Carrington
Mark Merriman
Andrew Moss
Jean-Marie Nicholas
Phil Stanley
Sara Wright
Tim Bourne
Yann Foricher
Zhaoning Zhu
Daniel Brookings
Helen Horsley
Jag Heer
Laurent Schio
Matthias Herrmann
Srinivas Rao
Markus Kohlmann
Peter Florian
An orally available small molecule that targets soluble TNF to deliver anti-TNF biologic-like efficacy in rheumatoid arthritis
Frontiers in Pharmacology
TNF
rheumatoid arthritis
small molecule
TNF trimer
immunogenicity
title An orally available small molecule that targets soluble TNF to deliver anti-TNF biologic-like efficacy in rheumatoid arthritis
title_full An orally available small molecule that targets soluble TNF to deliver anti-TNF biologic-like efficacy in rheumatoid arthritis
title_fullStr An orally available small molecule that targets soluble TNF to deliver anti-TNF biologic-like efficacy in rheumatoid arthritis
title_full_unstemmed An orally available small molecule that targets soluble TNF to deliver anti-TNF biologic-like efficacy in rheumatoid arthritis
title_short An orally available small molecule that targets soluble TNF to deliver anti-TNF biologic-like efficacy in rheumatoid arthritis
title_sort orally available small molecule that targets soluble tnf to deliver anti tnf biologic like efficacy in rheumatoid arthritis
topic TNF
rheumatoid arthritis
small molecule
TNF trimer
immunogenicity
url https://www.frontiersin.org/articles/10.3389/fphar.2022.1037983/full
work_keys_str_mv AT alexandervugler anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT jamesoconnell anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT maianhnguyen anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT dietmarweitz anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT thomasleeuw anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT elizabethhickford anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT alexanderverbitsky anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT xiaoyouying anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT markusrehberg anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT brucecarrington anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT markmerriman anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT andrewmoss anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT jeanmarienicholas anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT philstanley anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT sarawright anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT timbourne anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT yannforicher anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT zhaoningzhu anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT danielbrookings anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT helenhorsley anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT jagheer anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT laurentschio anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT matthiasherrmann anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT srinivasrao anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT markuskohlmann anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT peterflorian anorallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT alexandervugler orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT jamesoconnell orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT maianhnguyen orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT dietmarweitz orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT thomasleeuw orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT elizabethhickford orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT alexanderverbitsky orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT xiaoyouying orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT markusrehberg orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT brucecarrington orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT markmerriman orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT andrewmoss orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT jeanmarienicholas orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT philstanley orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT sarawright orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT timbourne orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT yannforicher orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT zhaoningzhu orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT danielbrookings orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT helenhorsley orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT jagheer orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT laurentschio orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT matthiasherrmann orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT srinivasrao orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT markuskohlmann orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis
AT peterflorian orallyavailablesmallmoleculethattargetssolubletnftodeliverantitnfbiologiclikeefficacyinrheumatoidarthritis