Preclinical pharmacokinetics, pharmacodynamics, and toxicity of novel small-molecule GPR119 agonists to treat type-2 diabetes and obesity

The escalating global prevalence of type-2 diabetes (T2D) and obesity necessitates the development of novel oral medications. Agonism at G-protein coupled receptor-119 (GPR119) has been recognized for modulation of metabolic homeostasis in T2D, obesity, and fatty liver disease. However, off-target e...

Full description

Bibliographic Details
Main Authors: Patil, Mohan, Casari, Ilaria, Thapa, Dinesh, Warne, Leon, Dallerba, Elena, Massi, Max, Carlessi, Rodrigo, Falasca, M.
Format: Journal Article
Language:English
Published: 2024
Subjects:
Online Access:http://hdl.handle.net/20.500.11937/96028
_version_ 1848766076808069120
author Patil, Mohan
Casari, Ilaria
Thapa, Dinesh
Warne, Leon
Dallerba, Elena
Massi, Max
Carlessi, Rodrigo
Falasca, M.
author_facet Patil, Mohan
Casari, Ilaria
Thapa, Dinesh
Warne, Leon
Dallerba, Elena
Massi, Max
Carlessi, Rodrigo
Falasca, M.
author_sort Patil, Mohan
building Curtin Institutional Repository
collection Online Access
description The escalating global prevalence of type-2 diabetes (T2D) and obesity necessitates the development of novel oral medications. Agonism at G-protein coupled receptor-119 (GPR119) has been recognized for modulation of metabolic homeostasis in T2D, obesity, and fatty liver disease. However, off-target effects have impeded the advancement of synthetic GPR119 agonist drug candidates. Non-systemic, gut-restricted GPR119 agonism is suggested as an alternative strategy that may locally stimulate intestinal enteroendocrine cells (EEC) for incretin secretion, without the need for systemic drug availability, consequently alleviating conventional class-related side effects. Herein, we report the preclinical acute safety, efficacy, and pharmacokinetics (PK) of novel GPR119 agonist compounds ps297 and ps318 that potentially target gut EEC for incretin secretion. In a proof-of-efficacy study, both compounds demonstrated glucagon-like peptide-1 (GLP-1) secretion capability during glucose and mixed-meal tolerance tests in healthy mice. Furthermore, co-administration of sitagliptin with investigational compounds in diabetic db/db mice resulted in synergism, with GLP-1 concentrations rising by three-fold. Both ps297 and ps318 exhibited low gut permeability assessed in the in-vitro Caco-2 cell model. A single oral dose PK study conducted on healthy mice demonstrated poor systemic bioavailability of both agents. PK measures (mean ± SD) for compound ps297 (Cmax 23 ± 19 ng/mL, Tmax range 0.5 – 1 h, AUC0–24 h 19.6 ± 21 h*ng/mL) and ps318 (Cmax 75 ± 22 ng/mL, Tmax range 0.25 – 0.5 h, AUC0–24 h 35 ± 23 h*ng/mL) suggest poor oral absorption. Additionally, examinations of drug excretion patterns in mice revealed that around 25 % (ps297) and 4 % (ps318) of the drugs were excreted through faeces as an unchanged form, while negligible drug concentrations (<0.005 %) were excreted in the urine. These acute PK/PD assessments suggest the gut is a primary site of action for both agents. Toxicity assessments conducted in the zebrafish and healthy mice models confirmed the safety and tolerability of both compounds. Future chronic in-vivo studies in relevant disease models will be essential to confirm the long-term safety and efficacy of these novel compounds.
first_indexed 2025-11-14T11:45:24Z
format Journal Article
id curtin-20.500.11937-96028
institution Curtin University Malaysia
institution_category Local University
language eng
last_indexed 2025-11-14T11:45:24Z
publishDate 2024
recordtype eprints
repository_type Digital Repository
spelling curtin-20.500.11937-960282024-11-07T00:42:38Z Preclinical pharmacokinetics, pharmacodynamics, and toxicity of novel small-molecule GPR119 agonists to treat type-2 diabetes and obesity Patil, Mohan Casari, Ilaria Thapa, Dinesh Warne, Leon Dallerba, Elena Massi, Max Carlessi, Rodrigo Falasca, M. GLP-1 Synthetic GPR119 agonists obesity pharmacodynamics pharmacokinetics toxicity type-2 diabetes Animals Humans Male Mice Caco-2 Cells Diabetes Mellitus, Type 2 Enteroendocrine Cells Glucagon-Like Peptide 1 Hypoglycemic Agents Mice, Inbred C57BL Obesity Receptors, G-Protein-Coupled Caco-2 Cells Animals Mice, Inbred C57BL Humans Mice Diabetes Mellitus, Type 2 Obesity Receptors, G-Protein-Coupled Hypoglycemic Agents Male Enteroendocrine Cells Glucagon-Like Peptide 1 The escalating global prevalence of type-2 diabetes (T2D) and obesity necessitates the development of novel oral medications. Agonism at G-protein coupled receptor-119 (GPR119) has been recognized for modulation of metabolic homeostasis in T2D, obesity, and fatty liver disease. However, off-target effects have impeded the advancement of synthetic GPR119 agonist drug candidates. Non-systemic, gut-restricted GPR119 agonism is suggested as an alternative strategy that may locally stimulate intestinal enteroendocrine cells (EEC) for incretin secretion, without the need for systemic drug availability, consequently alleviating conventional class-related side effects. Herein, we report the preclinical acute safety, efficacy, and pharmacokinetics (PK) of novel GPR119 agonist compounds ps297 and ps318 that potentially target gut EEC for incretin secretion. In a proof-of-efficacy study, both compounds demonstrated glucagon-like peptide-1 (GLP-1) secretion capability during glucose and mixed-meal tolerance tests in healthy mice. Furthermore, co-administration of sitagliptin with investigational compounds in diabetic db/db mice resulted in synergism, with GLP-1 concentrations rising by three-fold. Both ps297 and ps318 exhibited low gut permeability assessed in the in-vitro Caco-2 cell model. A single oral dose PK study conducted on healthy mice demonstrated poor systemic bioavailability of both agents. PK measures (mean ± SD) for compound ps297 (Cmax 23 ± 19 ng/mL, Tmax range 0.5 – 1 h, AUC0–24 h 19.6 ± 21 h*ng/mL) and ps318 (Cmax 75 ± 22 ng/mL, Tmax range 0.25 – 0.5 h, AUC0–24 h 35 ± 23 h*ng/mL) suggest poor oral absorption. Additionally, examinations of drug excretion patterns in mice revealed that around 25 % (ps297) and 4 % (ps318) of the drugs were excreted through faeces as an unchanged form, while negligible drug concentrations (<0.005 %) were excreted in the urine. These acute PK/PD assessments suggest the gut is a primary site of action for both agents. Toxicity assessments conducted in the zebrafish and healthy mice models confirmed the safety and tolerability of both compounds. Future chronic in-vivo studies in relevant disease models will be essential to confirm the long-term safety and efficacy of these novel compounds. 2024 Journal Article http://hdl.handle.net/20.500.11937/96028 10.1016/j.biopha.2024.117077 eng https://creativecommons.org/licenses/by-nc/4.0/ fulltext
spellingShingle GLP-1
Synthetic GPR119 agonists
obesity
pharmacodynamics
pharmacokinetics
toxicity
type-2 diabetes
Animals
Humans
Male
Mice
Caco-2 Cells
Diabetes Mellitus, Type 2
Enteroendocrine Cells
Glucagon-Like Peptide 1
Hypoglycemic Agents
Mice, Inbred C57BL
Obesity
Receptors, G-Protein-Coupled
Caco-2 Cells
Animals
Mice, Inbred C57BL
Humans
Mice
Diabetes Mellitus, Type 2
Obesity
Receptors, G-Protein-Coupled
Hypoglycemic Agents
Male
Enteroendocrine Cells
Glucagon-Like Peptide 1
Patil, Mohan
Casari, Ilaria
Thapa, Dinesh
Warne, Leon
Dallerba, Elena
Massi, Max
Carlessi, Rodrigo
Falasca, M.
Preclinical pharmacokinetics, pharmacodynamics, and toxicity of novel small-molecule GPR119 agonists to treat type-2 diabetes and obesity
title Preclinical pharmacokinetics, pharmacodynamics, and toxicity of novel small-molecule GPR119 agonists to treat type-2 diabetes and obesity
title_full Preclinical pharmacokinetics, pharmacodynamics, and toxicity of novel small-molecule GPR119 agonists to treat type-2 diabetes and obesity
title_fullStr Preclinical pharmacokinetics, pharmacodynamics, and toxicity of novel small-molecule GPR119 agonists to treat type-2 diabetes and obesity
title_full_unstemmed Preclinical pharmacokinetics, pharmacodynamics, and toxicity of novel small-molecule GPR119 agonists to treat type-2 diabetes and obesity
title_short Preclinical pharmacokinetics, pharmacodynamics, and toxicity of novel small-molecule GPR119 agonists to treat type-2 diabetes and obesity
title_sort preclinical pharmacokinetics, pharmacodynamics, and toxicity of novel small-molecule gpr119 agonists to treat type-2 diabetes and obesity
topic GLP-1
Synthetic GPR119 agonists
obesity
pharmacodynamics
pharmacokinetics
toxicity
type-2 diabetes
Animals
Humans
Male
Mice
Caco-2 Cells
Diabetes Mellitus, Type 2
Enteroendocrine Cells
Glucagon-Like Peptide 1
Hypoglycemic Agents
Mice, Inbred C57BL
Obesity
Receptors, G-Protein-Coupled
Caco-2 Cells
Animals
Mice, Inbred C57BL
Humans
Mice
Diabetes Mellitus, Type 2
Obesity
Receptors, G-Protein-Coupled
Hypoglycemic Agents
Male
Enteroendocrine Cells
Glucagon-Like Peptide 1
url http://hdl.handle.net/20.500.11937/96028