Compounds > idarucizumab
Page last updated: 2024-11-13

idarucizumab

Cross-References

ID SourceID
PubMed CID71464713
CHEMBL ID3940890
SCHEMBL ID15816954
MeSH IDM000601774

Synonyms (23)

Synonym
3-[3-(3,5-dimethyl-1h-pyrazol-4-yl)propoxy]-4-fluorobenzoic acid
16V ,
4HIQ
SCHEMBL15816954
1446711-81-4
unii-t12b44a1oe
who 11205
ag10
acoramidis
benzoic acid, 3-(3-(3,5-dimethyl-1h-pyrazol-4-yl)propoxy)-4-fluoro-
t12b44a1oe ,
acoramidis [usan]
CHEMBL3940890 ,
3-(3-(3,5-dimethyl-1h-pyrazol-4-yl)propoxy)-4-fluorobenzoic acid ,
ag-10
bdbm50197885
acoramidis [who-dd]
acoramidis [inn]
E79901
Q27451739
HY-109165
CS-0116350
AKOS040759821

Research Excerpts

Toxicity

ExcerptReferenceRelevance
"The misassembly of soluble proteins into toxic aggregates, including amyloid fibrils, underlies a large number of human degenerative diseases."( AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V122I transthyretin.
Alhamadsheh, MM; Baranczak, A; Chan, WK; Connelly, S; Graef, IA; Kelly, JW; Liedtke, M; Park, MS; Penchala, SC; Powers, ET; Rappley, I; Reixach, N; Vogel, H; Wang, Y; Wilson, IA; Witteles, RM; Zhao, L, 2013)		0.39
" Safety and tolerability were assessed by vital signs, electrocardiogram, adverse events, and clinical laboratory tests."( First-in-Human Study of AG10, a Novel, Oral, Specific, Selective, and Potent Transthyretin Stabilizer for the Treatment of Transthyretin Amyloidosis: A Phase 1 Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Healthy Adult Volunteers.
Fox, JC; Gretler, D; Hellawell, JL; Jernelius, J; Lumpkin, R; O'Reilly, T; Rao, S; Sinha, U, 2020)		0.56

Pharmacokinetics

ExcerptReferenceRelevance
" Pharmacodynamics were assessed via three pharmacodynamic assays of TTR stabilization."( First-in-Human Study of AG10, a Novel, Oral, Specific, Selective, and Potent Transthyretin Stabilizer for the Treatment of Transthyretin Amyloidosis: A Phase 1 Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Healthy Adult Volunteers.
Fox, JC; Gretler, D; Hellawell, JL; Jernelius, J; Lumpkin, R; O'Reilly, T; Rao, S; Sinha, U, 2020)		0.56

Bioavailability

ExcerptReferenceRelevance
" The oral bioavailability of AG10, combined with additional desirable drug-like features, makes it a very promising candidate to treat TTR amyloid cardiomyopathy."( AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V122I transthyretin.
Alhamadsheh, MM; Baranczak, A; Chan, WK; Connelly, S; Graef, IA; Kelly, JW; Liedtke, M; Park, MS; Penchala, SC; Powers, ET; Rappley, I; Reixach, N; Vogel, H; Wang, Y; Wilson, IA; Witteles, RM; Zhao, L, 2013)		0.39

Dosage Studied

ExcerptRelevanceReference
" Complete (>90%) stabilization of TTR was observed across the entire dosing interval at steady state on the highest dose tested."( First-in-Human Study of AG10, a Novel, Oral, Specific, Selective, and Potent Transthyretin Stabilizer for the Treatment of Transthyretin Amyloidosis: A Phase 1 Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Healthy Adult Volunteers.
Fox, JC; Gretler, D; Hellawell, JL; Jernelius, J; Lumpkin, R; O'Reilly, T; Rao, S; Sinha, U, 2020)		0.56
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Protein Targets (3)

Inhibition Measurements

ProteinTaxonomyMeasurementAverageMin (ref.)Avg (ref.)Max (ref.)Bioassay(s)
TransthyretinHomo sapiens (human)IC50 (µMol)0.16000.16004.292110.0000AID1810837
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Activation Measurements

ProteinTaxonomyMeasurementAverageMin (ref.)Avg (ref.)Max (ref.)Bioassay(s)
TransthyretinHomo sapiens (human)Kd0.00610.00301.348210.0000AID1386000; AID1896523
AlbuminHomo sapiens (human)Kd8.00000.08933.31358.0000AID1386001
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Other Measurements

ProteinTaxonomyMeasurementAverageMin (ref.)Avg (ref.)Max (ref.)Bioassay(s)
AlbuminHomo sapiens (human)PB13.05007.80007.80007.8000AID1386024; AID1386025
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Biological Processes (29)

Processvia Protein(s)Taxonomy
eye developmentRetinol-binding protein 4Homo sapiens (human)
positive regulation of immunoglobulin productionRetinol-binding protein 4Homo sapiens (human)
gluconeogenesisRetinol-binding protein 4Homo sapiens (human)
heart developmentRetinol-binding protein 4Homo sapiens (human)
visual perceptionRetinol-binding protein 4Homo sapiens (human)
maintenance of gastrointestinal epitheliumRetinol-binding protein 4Homo sapiens (human)
lung developmentRetinol-binding protein 4Homo sapiens (human)
positive regulation of insulin secretionRetinol-binding protein 4Homo sapiens (human)
response to retinoic acidRetinol-binding protein 4Homo sapiens (human)
retinol transportRetinol-binding protein 4Homo sapiens (human)
retinol metabolic processRetinol-binding protein 4Homo sapiens (human)
glucose homeostasisRetinol-binding protein 4Homo sapiens (human)
embryonic organ morphogenesisRetinol-binding protein 4Homo sapiens (human)
embryonic skeletal system developmentRetinol-binding protein 4Homo sapiens (human)
cardiac muscle tissue developmentRetinol-binding protein 4Homo sapiens (human)
female genitalia morphogenesisRetinol-binding protein 4Homo sapiens (human)
negative regulation of cardiac muscle cell proliferationRetinol-binding protein 4Homo sapiens (human)
embryonic retina morphogenesis in camera-type eyeRetinol-binding protein 4Homo sapiens (human)
uterus developmentRetinol-binding protein 4Homo sapiens (human)
vagina developmentRetinol-binding protein 4Homo sapiens (human)
urinary bladder developmentRetinol-binding protein 4Homo sapiens (human)
heart trabecula formationRetinol-binding protein 4Homo sapiens (human)
signal transductionTransthyretinHomo sapiens (human)
purine nucleobase metabolic processTransthyretinHomo sapiens (human)
cellular response to starvationAlbuminHomo sapiens (human)
negative regulation of mitochondrial depolarizationAlbuminHomo sapiens (human)
cellular response to calcium ion starvationAlbuminHomo sapiens (human)
cellular oxidant detoxificationAlbuminHomo sapiens (human)
transportAlbuminHomo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Molecular Functions (17)

Processvia Protein(s)Taxonomy
protein bindingRetinol-binding protein 4Homo sapiens (human)
retinal bindingRetinol-binding protein 4Homo sapiens (human)
retinol bindingRetinol-binding protein 4Homo sapiens (human)
retinol transmembrane transporter activityRetinol-binding protein 4Homo sapiens (human)
hormone activityTransthyretinHomo sapiens (human)
protein bindingTransthyretinHomo sapiens (human)
identical protein bindingTransthyretinHomo sapiens (human)
thyroid hormone bindingTransthyretinHomo sapiens (human)
oxygen bindingAlbuminHomo sapiens (human)
DNA bindingAlbuminHomo sapiens (human)
fatty acid bindingAlbuminHomo sapiens (human)
copper ion bindingAlbuminHomo sapiens (human)
protein bindingAlbuminHomo sapiens (human)
toxic substance bindingAlbuminHomo sapiens (human)
antioxidant activityAlbuminHomo sapiens (human)
pyridoxal phosphate bindingAlbuminHomo sapiens (human)
identical protein bindingAlbuminHomo sapiens (human)
protein-folding chaperone bindingAlbuminHomo sapiens (human)
exogenous protein bindingAlbuminHomo sapiens (human)
enterobactin bindingAlbuminHomo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Ceullar Components (12)

Processvia Protein(s)Taxonomy
extracellular regionRetinol-binding protein 4Homo sapiens (human)
extracellular spaceRetinol-binding protein 4Homo sapiens (human)
extracellular exosomeRetinol-binding protein 4Homo sapiens (human)
extracellular spaceRetinol-binding protein 4Homo sapiens (human)
extracellular regionTransthyretinHomo sapiens (human)
extracellular spaceTransthyretinHomo sapiens (human)
azurophil granule lumenTransthyretinHomo sapiens (human)
extracellular exosomeTransthyretinHomo sapiens (human)
extracellular spaceTransthyretinHomo sapiens (human)
extracellular regionAlbuminHomo sapiens (human)
extracellular spaceAlbuminHomo sapiens (human)
nucleusAlbuminHomo sapiens (human)
endoplasmic reticulumAlbuminHomo sapiens (human)
endoplasmic reticulum lumenAlbuminHomo sapiens (human)
Golgi apparatusAlbuminHomo sapiens (human)
platelet alpha granule lumenAlbuminHomo sapiens (human)
extracellular exosomeAlbuminHomo sapiens (human)
blood microparticleAlbuminHomo sapiens (human)
protein-containing complexAlbuminHomo sapiens (human)
cytoplasmAlbuminHomo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Bioassays (40)

Assay IDTitleYearJournalArticle
AID1322959Binding affinity to human plasma wild type TTR assessed as dissociation constants for first binding site of TTR by isothermal titration calorimetric method2016European journal of medicinal chemistry, Oct-04, Volume: 121A novel bis-furan scaffold for transthyretin stabilization and amyloid inhibition.
AID1386029Binding affinity to purified human transthyretin assessed as protein occupancy at 10 uM incubated for 6 hrs in pH 7.4 PBS buffer in presence of 600 uM albumin by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386024Binding affinity to human serum albumin assessed as compound level bound to protein at 30 uM measured immediately after gel filtration2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386037In-vivo binding affinity to transthyretin in beagle dog serum assessed as protein occupancy 20 mg/kg/day dosed daily dosed as single administration via oral gavage2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386011Binding affinity to transthyretin in whole human serum assessed as protein occupancy at 5 uM after 72 hrs by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386027Binding affinity to transthyretin (unknown origin) assessed as compound to TTR molar ratio at 5 uM measured after 24 hrs by gel filtration/dialysis assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386025Binding affinity to human serum albumin assessed as compound level bound to protein at 30 uM measured 124 hrs after dialysis2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1385999Binding affinity to transthyretin in dog serum assessed as protein stabilization at 10 uM by by Western blot2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386019Binding affinity to transthyretin (unknown origin) in human serum assessed as protein stabilization at 10 uM in pH 7.4 urea buffer by Western blot2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386028Binding affinity to purified human transthyretin assessed as protein occupancy at 10 uM incubated for 6 hrs in pH 7.4 PBS buffer by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386022Binding affinity to transthyretin in whole human serum assessed as protein occupancy at 20 uM after 72 hrs by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386009Binding affinity to transthyretin in human serum assessed as protein occupancy at 2:1 compound to TTR ratio after 72 hrs by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1896523Binding affinity to human TTR incubated for 20 mins by fluorescence polarization assay2022Journal of medicinal chemistry, 11-24, Volume: 65, Issue:22
Enhancing the Safety and Efficacy of PSMA-Based Small-Molecule Drug Conjugates by Linker Stabilization and Conjugation to Transthyretin Binding Ligand.
AID1386010Binding affinity to transthyretin in human serum assessed as protein stabilization at 2:1 compound to TTR ratio after 72 hrs by Western blot2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386006Binding affinity to transthyretin (unknown origin) assessed as free enthalpy changes by ITC method2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386026Binding affinity to transthyretin (unknown origin) assessed as compound to TTR molar ratio at 5 uM measured after 6 hrs by gel filtration/dialysis assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386003Potency index, ratio of Kd for tolcapone to Kd for test compound for binding affinity to transthyretin (unknown origin) by ITC method2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1810836Disruption of retional-induced bacterially expressed MBP-tagged RBP4-TTR (unknown origin) protein-protein interaction measured by d2 dye based HTFR assay2021Journal of medicinal chemistry, 07-08, Volume: 64, Issue:13
Identification of Transthyretin Tetramer Kinetic Stabilizers That Are Capable of Inhibiting the Retinol-Dependent Retinol Binding Protein 4-Transthyretin Interaction: Potential Novel Therapeutics for Macular Degeneration, Transthyretin Amyloidosis, and Th
AID1386036In-vivo binding affinity to transthyretin in beagle dog serum assessed as protein occupancy measured at 1 hr post dose on day 7 at 50 to 200 mg/kg/day dosed daily via oral gavage for 7 days by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386016Binding affinity to transthyretin (unknown origin) in human serum assessed as protein occupancy at 10 uM by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386007Binding affinity to transthyretin (unknown origin) assessed as free TdeltaS changes by ITC method2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386008Binding affinity to transthyretin (unknown origin) in pH 7.4 PBS buffer assessed as protein occupancy at 1:1 compound to TTR ratio by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386023Binding affinity to transthyretin in whole human serum assessed as protein stabilization at 20 uM after 72 hrs by Western blot2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386033Binding affinity to transthyretin in dog serum assessed as protein occupancy at 10 uM by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1810838Displacement of [3H]-all trans retinol from human urine biotinylated RBP4 incubated for 16 hrs measured by Scintillation Proximity Assay2021Journal of medicinal chemistry, 07-08, Volume: 64, Issue:13
Identification of Transthyretin Tetramer Kinetic Stabilizers That Are Capable of Inhibiting the Retinol-Dependent Retinol Binding Protein 4-Transthyretin Interaction: Potential Novel Therapeutics for Macular Degeneration, Transthyretin Amyloidosis, and Th
AID1386004Potency index, ratio of Kd for diflunisal to Kd for test compound for binding affinity to transthyretin (unknown origin) by ITC method2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386021Binding affinity to transthyretin in whole human serum assessed as protein stabilization at 5 uM after 72 hrs by Western blot2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386030Binding affinity to purified human transthyretin assessed as protein occupancy at 10 uM incubated for 6 hrs in pH 7.4 PBS buffer in presence of 25 uM transferrin by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386001Binding affinity to human serum albumin by ITC method2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386034In-vivo binding affinity to transthyretin in beagle dog serum assessed as protein occupancy at 50 to 200 mg/kg/day dosed daily via oral gavage for 7 days by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386005Binding affinity to transthyretin (unknown origin) assessed as free energy changes by ITC method2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386031Binding affinity to purified human transthyretin assessed as protein occupancy at 10 uM incubated for 6 hrs in pH 7.4 PBS buffer in presence of 5 uM fibrinogen by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1810837Binding affinity to human plasma TTR tetramer assessed as displacement of diclofenac-coupled FITC by fluorescence polarization assay2021Journal of medicinal chemistry, 07-08, Volume: 64, Issue:13
Identification of Transthyretin Tetramer Kinetic Stabilizers That Are Capable of Inhibiting the Retinol-Dependent Retinol Binding Protein 4-Transthyretin Interaction: Potential Novel Therapeutics for Macular Degeneration, Transthyretin Amyloidosis, and Th
AID1386000Binding affinity to transthyretin (unknown origin) by ITC method2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386032Binding affinity to purified human transthyretin assessed as protein occupancy at 10 uM incubated for 6 hrs in pH 7.4 PBS buffer in presence of 70 uM IgG by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386015Dissociation constant, pKa of the compound2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1386038In-vivo binding affinity to transthyretin in beagle dog serum assessed as protein occupancy 5 mg/kg/day dosed daily dosed as single administration via oral gavage2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID1322958Binding affinity to human plasma wild type TTR assessed as dissociation constants for second binding site of TTR by isothermal titration calorimetric method2016European journal of medicinal chemistry, Oct-04, Volume: 121A novel bis-furan scaffold for transthyretin stabilization and amyloid inhibition.
AID1386035In-vivo binding affinity to transthyretin in beagle dog serum assessed as protein occupancy measured on day 7 at 50 to 200 mg/kg/day dosed daily via oral gavage for 7 days by fluorescent probe exclusion assay2018Journal of medicinal chemistry, 09-13, Volume: 61, Issue:17
Enthalpy-Driven Stabilization of Transthyretin by AG10 Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis.
AID977611Experimentally measured binding affinity data (Kd) for protein-ligand complexes derived from PDB2013Proceedings of the National Academy of Sciences of the United States of America, Jun-11, Volume: 110, Issue:24
AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V122I transthyretin.
[information is prepared from bioassay data collected from National Library of Medicine (NLM), extracted Dec-2023]

Research

Studies (11)

TimeframeStudies, This Drug (%)All Drugs %
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's0 (0.00)29.6817
2010's5 (45.45)24.3611
2020's6 (54.55)2.80
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Market Indicators

Research Demand Index: 71.47

According to the monthly volume, diversity, and competition of internet searches for this compound, as well the volume and growth of publications, there is estimated to be very strong demand-to-supply ratio for research on this compound.

MetricThis Compound (vs All)
Research Demand Index71.47 (24.57)
Research Supply Index2.64 (2.92)
Research Growth Index4.69 (4.65)
Search Engine Demand Index117.10 (26.88)
Search Engine Supply Index2.00 (0.95)

This Compound (71.47)

All Compounds (24.57)

Study Types

Publication TypeThis drug (%)All Drugs (%)
Trials2 (18.18%)5.53%
Reviews2 (18.18%)6.00%
Case Studies0 (0.00%)4.05%
Observational0 (0.00%)0.25%
Other7 (63.64%)84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]
SubstanceRelationship StrengthStudiesTrialsClassesRoles
diflunisal
Diflunisal: A salicylate derivative and anti-inflammatory analgesic with actions and side effects similar to those of ASPIRIN.. diflunisal : An organofluorine compound comprising salicylic acid having a 2,4-difluorophenyl group at the 5-position.		4.9430monohydroxybenzoic acid;
organofluorine compound		non-narcotic analgesic;
non-steroidal anti-inflammatory drug
thyroxine
Thyroxine: The major hormone derived from the thyroid gland. Thyroxine is synthesized via the iodination of tyrosines (MONOIODOTYROSINE) and the coupling of iodotyrosines (DIIODOTYROSINE) in the THYROGLOBULIN. Thyroxine is released from thyroglobulin by proteolysis and secreted into the blood. Thyroxine is peripherally deiodinated to form TRIIODOTHYRONINE which exerts a broad spectrum of stimulatory effects on cell metabolism.. thyroxine : An iodothyronine compound having iodo substituents at the 3-, 3'-, 5- and 5'-positions.		2.13102-halophenol;
iodophenol;
L-phenylalanine derivative;
non-proteinogenic L-alpha-amino acid;
thyroxine zwitterion;
thyroxine		antithyroid drug;
human metabolite;
mouse metabolite;
thyroid hormone
benzoxazoles
1,3-benzoxazole : A benzoxazole in which the benzene ring is fused to a 1,3-oxazole ring across positions 4 and 5.. benzoxazole : Compounds based on a fused 1,2- or 1,3-oxazole and benzene bicyclic ring skeleton.		6.11401,3-benzoxazoles;
mancude organic heterobicyclic parent
3,3',5,5'-tetrachlorobiphenyl-4,4'-diol
3,3',5,5'-tetrachlorobiphenyl-4,4'-diol : A member of the class of hydroxybiphenyls formed formally by chlorination of biphenyl-4,4'-diol at C-3, -3', -5 and -5'.		2.1310dichlorobenzene;
hydroxybiphenyls
docetaxel anhydrous
Docetaxel: A semisynthetic analog of PACLITAXEL used in the treatment of locally advanced or metastatic BREAST NEOPLASMS and NON-SMALL CELL LUNG CANCER.. docetaxel anhydrous : A tetracyclic diterpenoid that is paclitaxel with the N-benzyloxycarbonyl group replaced by N-tert-butoxycarbonyl, and the acetoxy group at position 10 replaced by a hydroxy group.		2.4110secondary alpha-hydroxy ketone;
tetracyclic diterpenoid		antimalarial;
antineoplastic agent;
photosensitizing agent
iododiflunisal
iododiflunisal: an amyloid inhibitor; structure in first source		2.1310
tolcapone
Tolcapone: A benzophenone and nitrophenol compound that acts as an inhibitor of CATECHOL O-METHYLTRANSFERASE, an enzyme involved in the metabolism of DOPAMINE and LEVODOPA. It is used in the treatment of PARKINSON DISEASE in patients for whom levodopa is ineffective or contraindicated.. tolcapone : Benzophenone substituted on one of the phenyl rings at C-3 and C-4 by hydroxy groups and at C-5 by a nitro group, and on the other phenyl ring by a methyl group at C-4. It is an inhibitor of catechol O-methyltransferase.		4.96302-nitrophenols;
benzophenones;
catechols		antiparkinson drug;
EC 2.1.1.6 (catechol O-methyltransferase) inhibitor
tafamidis
tafamidis: may be effective in treating transthyretin amyloid polyneuropathy. tafamidis : A member of the class of 1,3-benzoxazoles that is 1,3-benzoxazole-6-carboxylic acid in which the hydrogen at position 2 is replaced by a 3,5-dichlorophenyl group. Used (as its meglumine salt) for the amelioration of transthyretin-related hereditary amyloidosis.		6.71801,3-benzoxazoles;
dichlorobenzene;
monocarboxylic acid		central nervous system drug
monomethyl auristatin e
[no description available]		2.4110
oligonucleotides
[no description available]		5.6820
abt-199
venetoclax: A BCL-2 inhibitor with antineoplastic activity that is used in the treatment of CHRONIC LYMPHOCYTIC LEUKEMIA associated with chromosome 17p deletion; structure in first source.. venetoclax : A member of the class of pyrrolopyridines that is a potent inhibitor of the antiapoptotic protein B-cell lymphoma 2. It is used for treamtment of chronic lymphocytic leukemia with 17p deletion.		4.3310aromatic ether;
C-nitro compound;
monochlorobenzenes;
N-alkylpiperazine;
N-arylpiperazine;
N-sulfonylcarboxamide;
oxanes;
pyrrolopyridine		antineoplastic agent;
apoptosis inducer;
B-cell lymphoma 2 inhibitor
ConditionIndicatedRelationship StrengthStudiesTrials
Cardiomyopathies, Primary
[description not available]		05.8940
Amyloidosis
A group of sporadic, familial and/or inherited, degenerative, and infectious disease processes, linked by the common theme of abnormal protein folding and deposition of AMYLOID. As the amyloid deposits enlarge they displace normal tissue structures, causing disruption of function. Various signs and symptoms depend on the location and size of the deposits.		17.6430
Cardiomyopathies
A group of diseases in which the dominant feature is the involvement of the CARDIAC MUSCLE itself. Cardiomyopathies are classified according to their predominant pathophysiological features (DILATED CARDIOMYOPATHY; HYPERTROPHIC CARDIOMYOPATHY; RESTRICTIVE CARDIOMYOPATHY) or their etiological/pathological factors (CARDIOMYOPATHY, ALCOHOLIC; ENDOCARDIAL FIBROELASTOSIS).		05.8940
Amyloid Neuropathy Type 1
[description not available]		010.4105
Amyloid Neuropathies, Familial
Inherited disorders of the peripheral nervous system associated with the deposition of AMYLOID in nerve tissue. The different clinical types based on symptoms correspond to the presence of a variety of mutations in several different proteins including transthyretin (PREALBUMIN); APOLIPOPROTEIN A-I; and GELSOLIN.		112.4105
Disease Exacerbation
[description not available]		04.0810
Arrhythmia
[description not available]		04.0810
Cardiac Failure
[description not available]		08.9354
AL Amyloidosis
[description not available]		04.0810
Immunoglobulin Light-chain Amyloidosis
A nonproliferative disorder of the PLASMA CELL characterized by excessive production and misfolding of IMMUNOGLOBULIN LIGHT CHAINS that form insoluble amyloid fibrils (see AMYLOID DEPOSITS) in various tissues. Clinical features include LIVER FAILURE; MULTIPLE MYELOMA; NEPHROTIC SYNDROME; RESTRICTIVE CARDIOMYOPATHY, and neuropathies.		04.0810
Arrhythmias, Cardiac
Any disturbances of the normal rhythmic beating of the heart or MYOCARDIAL CONTRACTION. Cardiac arrhythmias can be classified by the abnormalities in HEART RATE, disorders of electrical impulse generation, or impulse conduction.		04.0810
Heart Failure
A heterogeneous condition in which the heart is unable to pump out sufficient blood to meet the metabolic need of the body. Heart failure can be caused by structural defects, functional abnormalities (VENTRICULAR DYSFUNCTION), or a sudden overload beyond its capacity. Chronic heart failure is more common than acute heart failure which results from sudden insult to cardiac function, such as MYOCARDIAL INFARCTION.		08.9354
Aging
The gradual irreversible changes in structure and function of an organism that occur as a result of the passage of time.		04.1510
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