Target inhibition of galectin-3 by inhaled TD139 in patients with idiopathic pulmonary fibrosis

Galectin (Gal)-3 is a profibrotic β-galactoside-binding lectin that plays a key role in the pathogenesis of idiopathic pulmonary fibrosis (IPF) and IPF exacerbations. TD139 is a novel and potent small-molecule inhibitor of Gal-3.A randomised, double-blind, multicentre, placebo-controlled, phase 1/2a...

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Veröffentlicht in:The European respiratory journal Jg. 57; H. 5
Hauptverfasser: Hirani, Nikhil, MacKinnon, Alison C, Nicol, Lisa, Ford, Paul, Schambye, Hans, Pedersen, Anders, Nilsson, Ulf J, Leffler, Hakon, Sethi, Tariq, Tantawi, Susan, Gravelle, Lise, Slack, Robert J, Mills, Ross, Karmakar, Utsa, Humphries, Duncan, Zetterberg, Fredrik, Keeling, Lucy, Paul, Lyn, Molyneaux, Philip L, Li, Feng, Funston, Wendy, Forrest, Ian A, Simpson, A John, Gibbons, Michael A, Maher, Toby M
Format: Journal Article
Sprache:Englisch
Veröffentlicht: England 01.05.2021
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ISSN:1399-3003, 1399-3003
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Abstract Galectin (Gal)-3 is a profibrotic β-galactoside-binding lectin that plays a key role in the pathogenesis of idiopathic pulmonary fibrosis (IPF) and IPF exacerbations. TD139 is a novel and potent small-molecule inhibitor of Gal-3.A randomised, double-blind, multicentre, placebo-controlled, phase 1/2a study was conducted to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of inhaled TD139 in 36 healthy subjects and 24 patients with IPF. Six dose cohorts of six healthy subjects were evaluated (4:2 TD139:placebo ratio) with single doses of TD139 (0.15-50 mg) and three dose cohorts of eight patients with IPF (5:3 TD139:placebo ratio) with once-daily doses of TD139 (0.3-10 mg) for 14 days.Inhaled TD139 was well tolerated with no significant treatment-related side-effects. TD139 was rapidly absorbed, with mean time taken to reach maximum plasma concentration ( ) values ranging from 0.6 to 3 h and a plasma half-life ( ) of 8 h. The concentration of TD139 in the lung was >567-fold higher than in the blood, with systemic exposure predicting exposure in the target compartment. Gal-3 expression on alveolar macrophages was reduced in the 3 and 10 mg dose groups compared with placebo, with a concentration-dependent inhibition demonstrated. Inhibition of Gal-3 expression in the lung was associated with reductions in plasma biomarkers centrally relevant to IPF pathobiology (platelet-derived growth factor-BB, plasminogen activator inhibitor-1, Gal-3, CCL18 and YKL-40).TD139 is safe and well tolerated in healthy subjects and IPF patients. It was shown to suppress Gal-3 expression on bronchoalveolar lavage macrophages and, in a concerted fashion, decrease plasma biomarkers associated with IPF progression.
AbstractList Galectin (Gal)-3 is a profibrotic β-galactoside-binding lectin that plays a key role in the pathogenesis of idiopathic pulmonary fibrosis (IPF) and IPF exacerbations. TD139 is a novel and potent small-molecule inhibitor of Gal-3.A randomised, double-blind, multicentre, placebo-controlled, phase 1/2a study was conducted to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of inhaled TD139 in 36 healthy subjects and 24 patients with IPF. Six dose cohorts of six healthy subjects were evaluated (4:2 TD139:placebo ratio) with single doses of TD139 (0.15-50 mg) and three dose cohorts of eight patients with IPF (5:3 TD139:placebo ratio) with once-daily doses of TD139 (0.3-10 mg) for 14 days.Inhaled TD139 was well tolerated with no significant treatment-related side-effects. TD139 was rapidly absorbed, with mean time taken to reach maximum plasma concentration (C max) values ranging from 0.6 to 3 h and a plasma half-life (T 1/2) of 8 h. The concentration of TD139 in the lung was >567-fold higher than in the blood, with systemic exposure predicting exposure in the target compartment. Gal-3 expression on alveolar macrophages was reduced in the 3 and 10 mg dose groups compared with placebo, with a concentration-dependent inhibition demonstrated. Inhibition of Gal-3 expression in the lung was associated with reductions in plasma biomarkers centrally relevant to IPF pathobiology (platelet-derived growth factor-BB, plasminogen activator inhibitor-1, Gal-3, CCL18 and YKL-40).TD139 is safe and well tolerated in healthy subjects and IPF patients. It was shown to suppress Gal-3 expression on bronchoalveolar lavage macrophages and, in a concerted fashion, decrease plasma biomarkers associated with IPF progression.Galectin (Gal)-3 is a profibrotic β-galactoside-binding lectin that plays a key role in the pathogenesis of idiopathic pulmonary fibrosis (IPF) and IPF exacerbations. TD139 is a novel and potent small-molecule inhibitor of Gal-3.A randomised, double-blind, multicentre, placebo-controlled, phase 1/2a study was conducted to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of inhaled TD139 in 36 healthy subjects and 24 patients with IPF. Six dose cohorts of six healthy subjects were evaluated (4:2 TD139:placebo ratio) with single doses of TD139 (0.15-50 mg) and three dose cohorts of eight patients with IPF (5:3 TD139:placebo ratio) with once-daily doses of TD139 (0.3-10 mg) for 14 days.Inhaled TD139 was well tolerated with no significant treatment-related side-effects. TD139 was rapidly absorbed, with mean time taken to reach maximum plasma concentration (C max) values ranging from 0.6 to 3 h and a plasma half-life (T 1/2) of 8 h. The concentration of TD139 in the lung was >567-fold higher than in the blood, with systemic exposure predicting exposure in the target compartment. Gal-3 expression on alveolar macrophages was reduced in the 3 and 10 mg dose groups compared with placebo, with a concentration-dependent inhibition demonstrated. Inhibition of Gal-3 expression in the lung was associated with reductions in plasma biomarkers centrally relevant to IPF pathobiology (platelet-derived growth factor-BB, plasminogen activator inhibitor-1, Gal-3, CCL18 and YKL-40).TD139 is safe and well tolerated in healthy subjects and IPF patients. It was shown to suppress Gal-3 expression on bronchoalveolar lavage macrophages and, in a concerted fashion, decrease plasma biomarkers associated with IPF progression.
Galectin (Gal)-3 is a profibrotic β-galactoside-binding lectin that plays a key role in the pathogenesis of idiopathic pulmonary fibrosis (IPF) and IPF exacerbations. TD139 is a novel and potent small-molecule inhibitor of Gal-3.A randomised, double-blind, multicentre, placebo-controlled, phase 1/2a study was conducted to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of inhaled TD139 in 36 healthy subjects and 24 patients with IPF. Six dose cohorts of six healthy subjects were evaluated (4:2 TD139:placebo ratio) with single doses of TD139 (0.15-50 mg) and three dose cohorts of eight patients with IPF (5:3 TD139:placebo ratio) with once-daily doses of TD139 (0.3-10 mg) for 14 days.Inhaled TD139 was well tolerated with no significant treatment-related side-effects. TD139 was rapidly absorbed, with mean time taken to reach maximum plasma concentration ( ) values ranging from 0.6 to 3 h and a plasma half-life ( ) of 8 h. The concentration of TD139 in the lung was >567-fold higher than in the blood, with systemic exposure predicting exposure in the target compartment. Gal-3 expression on alveolar macrophages was reduced in the 3 and 10 mg dose groups compared with placebo, with a concentration-dependent inhibition demonstrated. Inhibition of Gal-3 expression in the lung was associated with reductions in plasma biomarkers centrally relevant to IPF pathobiology (platelet-derived growth factor-BB, plasminogen activator inhibitor-1, Gal-3, CCL18 and YKL-40).TD139 is safe and well tolerated in healthy subjects and IPF patients. It was shown to suppress Gal-3 expression on bronchoalveolar lavage macrophages and, in a concerted fashion, decrease plasma biomarkers associated with IPF progression.
Author Schambye, Hans
Forrest, Ian A
Ford, Paul
Maher, Toby M
Hirani, Nikhil
Molyneaux, Philip L
Funston, Wendy
Simpson, A John
Pedersen, Anders
Keeling, Lucy
Mills, Ross
Nicol, Lisa
Zetterberg, Fredrik
Li, Feng
Sethi, Tariq
Nilsson, Ulf J
Humphries, Duncan
Karmakar, Utsa
MacKinnon, Alison C
Paul, Lyn
Gibbons, Michael A
Slack, Robert J
Tantawi, Susan
Gravelle, Lise
Leffler, Hakon
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  surname: Hirani
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  email: n.hirani@ed.ac.uk
  organization: MRC Centre for Inflammation Research, The Queen's Medical Research Institute, University of Edinburgh, Edinburgh, UK n.hirani@ed.ac.uk
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  givenname: Alison C
  orcidid: 0000-0002-7967-4141
  surname: MacKinnon
  fullname: MacKinnon, Alison C
  organization: Galecto, Copenhagen, Denmark
– sequence: 3
  givenname: Lisa
  surname: Nicol
  fullname: Nicol, Lisa
  organization: MRC Centre for Inflammation Research, The Queen's Medical Research Institute, University of Edinburgh, Edinburgh, UK
– sequence: 4
  givenname: Paul
  surname: Ford
  fullname: Ford, Paul
  organization: Galecto, Copenhagen, Denmark
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  organization: Galecto, Copenhagen, Denmark
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  organization: Galecto, Copenhagen, Denmark
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  organization: Dept of Chemistry, Lund University, Lund, Sweden
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  organization: Dept of Laboratory Medicine, Lund University, Lund, Sweden
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  surname: Sethi
  fullname: Sethi, Tariq
  organization: Galecto, Copenhagen, Denmark
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  surname: Tantawi
  fullname: Tantawi, Susan
  organization: Galecto, Copenhagen, Denmark
– sequence: 11
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  orcidid: 0000-0003-4472-090X
  surname: Gravelle
  fullname: Gravelle, Lise
  organization: Galecto, Copenhagen, Denmark
– sequence: 12
  givenname: Robert J
  surname: Slack
  fullname: Slack, Robert J
  organization: Galecto, Copenhagen, Denmark
– sequence: 13
  givenname: Ross
  surname: Mills
  fullname: Mills, Ross
  organization: MRC Centre for Inflammation Research, The Queen's Medical Research Institute, University of Edinburgh, Edinburgh, UK
– sequence: 14
  givenname: Utsa
  surname: Karmakar
  fullname: Karmakar, Utsa
  organization: MRC Centre for Inflammation Research, The Queen's Medical Research Institute, University of Edinburgh, Edinburgh, UK
– sequence: 15
  givenname: Duncan
  orcidid: 0000-0002-5551-6536
  surname: Humphries
  fullname: Humphries, Duncan
  organization: MRC Centre for Inflammation Research, The Queen's Medical Research Institute, University of Edinburgh, Edinburgh, UK
– sequence: 16
  givenname: Fredrik
  orcidid: 0000-0002-7789-8782
  surname: Zetterberg
  fullname: Zetterberg, Fredrik
  organization: Galecto, Copenhagen, Denmark
– sequence: 17
  givenname: Lucy
  surname: Keeling
  fullname: Keeling, Lucy
  organization: Exploristics, Belfast, UK
– sequence: 18
  givenname: Lyn
  surname: Paul
  fullname: Paul, Lyn
  organization: National Institute for Health Research Respiratory Clinical Research Facility, Royal Brompton and Harefield NHS Foundation Trust, and Fibrosis Research Group, National Heart and Lung Institute, Imperial College London, London, UK
– sequence: 19
  givenname: Philip L
  orcidid: 0000-0003-1301-8800
  surname: Molyneaux
  fullname: Molyneaux, Philip L
  organization: National Institute for Health Research Respiratory Clinical Research Facility, Royal Brompton and Harefield NHS Foundation Trust, and Fibrosis Research Group, National Heart and Lung Institute, Imperial College London, London, UK
– sequence: 20
  givenname: Feng
  orcidid: 0000-0002-7658-6138
  surname: Li
  fullname: Li, Feng
  organization: MRC Centre for Inflammation Research, The Queen's Medical Research Institute, University of Edinburgh, Edinburgh, UK
– sequence: 21
  givenname: Wendy
  surname: Funston
  fullname: Funston, Wendy
  organization: Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK
– sequence: 22
  givenname: Ian A
  surname: Forrest
  fullname: Forrest, Ian A
  organization: Respiratory Medicine Unit, Royal Victoria Infirmary, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK
– sequence: 23
  givenname: A John
  orcidid: 0000-0003-4731-7294
  surname: Simpson
  fullname: Simpson, A John
  organization: Respiratory Medicine Unit, Royal Victoria Infirmary, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK
– sequence: 24
  givenname: Michael A
  surname: Gibbons
  fullname: Gibbons, Michael A
  organization: Respiratory Dept, Institute of Biomedical and Clinical Science, Royal Devon and Exeter NHS Foundation Trust, Medical School, University of Exeter, Exeter, UK
– sequence: 25
  givenname: Toby M
  surname: Maher
  fullname: Maher, Toby M
  organization: Keck School of Medicine, University of Southern California, Los Angeles, CA, USA
BackLink https://www.ncbi.nlm.nih.gov/pubmed/33214209$$D View this record in MEDLINE/PubMed
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PublicationDateYYYYMMDD 2021-05-01
PublicationDate_xml – month: 05
  year: 2021
  text: 2021-05-01
  day: 01
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PublicationTitle The European respiratory journal
PublicationTitleAlternate Eur Respir J
PublicationYear 2021
References 34045285 - Eur Respir J. 2021 May 27;57(5):2100691. doi: 10.1183/13993003.00691-2021.
36735934 - Am J Respir Crit Care Med. 2023 Apr 15;207(8):1089-1091. doi: 10.1164/rccm.202206-1035RR.
35422427 - Eur Respir J. 2022 Apr 14;59(4):2052559. doi: 10.1183/13993003.52559-2020.
References_xml – reference: 36735934 - Am J Respir Crit Care Med. 2023 Apr 15;207(8):1089-1091. doi: 10.1164/rccm.202206-1035RR.
– reference: 34045285 - Eur Respir J. 2021 May 27;57(5):2100691. doi: 10.1183/13993003.00691-2021.
– reference: 35422427 - Eur Respir J. 2022 Apr 14;59(4):2052559. doi: 10.1183/13993003.52559-2020.
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Snippet Galectin (Gal)-3 is a profibrotic β-galactoside-binding lectin that plays a key role in the pathogenesis of idiopathic pulmonary fibrosis (IPF) and IPF...
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SubjectTerms Double-Blind Method
Galectin 3
Humans
Idiopathic Pulmonary Fibrosis
Lung
Title Target inhibition of galectin-3 by inhaled TD139 in patients with idiopathic pulmonary fibrosis
URI https://www.ncbi.nlm.nih.gov/pubmed/33214209
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