Details
Stereochemistry | ACHIRAL |
Molecular Formula | C13H18O2 |
Molecular Weight | 206.2808 |
Optical Activity | NONE |
Defined Stereocenters | 0 / 0 |
E/Z Centers | 0 |
Charge | 0 |
SHOW SMILES / InChI
SMILES
CCCCCC1=CC=CC(CC(O)=O)=C1
InChI
InChIKey=PEGQOIGYZLJMIB-UHFFFAOYSA-N
InChI=1S/C13H18O2/c1-2-3-4-6-11-7-5-8-12(9-11)10-13(14)15/h5,7-9H,2-4,6,10H2,1H3,(H,14,15)
Molecular Formula | C13H18O2 |
Molecular Weight | 206.2808 |
Charge | 0 |
Count |
|
Stereochemistry | ACHIRAL |
Additional Stereochemistry | No |
Defined Stereocenters | 0 / 0 |
E/Z Centers | 0 |
Optical Activity | NONE |
PBI 4050, a synthetic analog of a medium-chain fatty acid that displays agonist and antagonist ligand affinity toward GPR40 and GPR84, that was developed for managing inflammatory and fibrosis-related diseases. In addition, PBI-4050 may exert antifibrotic activity in the liver through a novel mechanism of action involving modulation of intracellular ATP levels and the LKB1/AMPK/mTOR pathway in stellate cells. This drug participated in clinical trials for the treatment of acute lung injury, cystic fibrosis, diabetic nephropathies; idiopathic pulmonary fibrosis; metabolic syndrome; scleroderma; type 2 diabetes mellitus. Besides, this drug has granted a Rare Pediatric Disease Designation for the treatment of Alström syndrome (AS). PBI-4050 was also previously granted Orphan Drug Designation by the FDA and the EMA for the treatments of AS and idiopathic pulmonary fibrosis (IPF) as well as PIM (Promising Innovative Medicine) designation by the Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of IPF and AS. The FDA grants Rare Pediatric Disease Designations for serious or life-threatening diseases wherein the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups, often called neonates, infants, children, and adolescents. Now Prometic Life Sciences plans to file investigational new drug application for pivotal phase III trial for Alstrom's syndrome in the second half of 2019.
Originator
Approval Year
PubMed
Title | Date | PubMed |
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A Newly Discovered Antifibrotic Pathway Regulated by Two Fatty Acid Receptors: GPR40 and GPR84. | 2018 May |
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Treatment with PBI-4050 in patients with Alström syndrome: study protocol for a phase 2, single-Centre, single-arm, open-label trial. | 2018 Nov 26 |
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Phase 2 clinical trial of PBI-4050 in patients with idiopathic pulmonary fibrosis. | 2019 Mar |
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PBI-4050 reduces pulmonary hypertension, lung fibrosis, and right ventricular dysfunction in heart failure. | 2020 Jan 1 |
Sample Use Guides
In Vivo Use Guide
Sources: https://clinicaltrials.gov/ct2/show/NCT03184584
Alström Syndrome: four 200 mg capsules (800 mg total) administered orally, once daily
Route of Administration:
Oral
Substance Class |
Chemical
Created
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879OVM0Y1S
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Validated (UNII)
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EU-Orphan Drug |
EU/3/16/1810
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ACTIVE MOIETY |
Originator: ProMetic Life Sciences; Developer: ProMetic Life Sciences, Vanderbilt University; Class: Anti-inflammatory, Antifibrotic, Small molecule; Mechanism of Action: Cell differentiation modulator, Colony stimulating factor stimulant, Cytokine modulator, Erythropoiesis stimulant, Extracellular matrix protein inhibitor, Extracellular matrix protein modulator, Immunomodulator, Inflammation mediator inhibitor, Inflammation mediator modulators;
Orphan Drug Status: Yes for Idiopathic pulmonary fibrosis; New Molecular Entity: Yes; Highest Development Phases: Phase II for Idiopathic pulmonary fibrosis, Inborn genetic Disorders, Metabolic syndrome/ Phase I/II for Diabetic nephropathies/ Preclinical for Cystic fibrosis, Renal fibrosis, Scleroderma/; No development reported for Anaemia, Haematological disorders, Neutropenia
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