acoramidis   Click here for help

GtoPdb Ligand ID: 13628

Synonyms: AG-10 | AG10 | Attruby®
Approved drug PDB Ligand
acoramidis is an approved drug (FDA (2024))
Compound class: Synthetic organic
Comment: Acoramidis (AG10) is an orally bioactive, small molecule transthyretin (TTR) stabiliser that functions to reduce the formation of pathogenic amyloid plaques that accumulate in the organs of individuals with misassembled TTR proteins [1].
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Ligand Activity Visualisation Charts

These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.

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View interactive charts of activity data across species

acoramidis is commercially available from our sponsors

2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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Physico-chemical Properties
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Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/). All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

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Hydrogen bond acceptors 5
Hydrogen bond donors 2
Rotatable bonds 6
Topological polar surface area 70.92
Molecular weight 292.31
XLogP 2.07
No. Lipinski's rules broken 0

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)

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Canonical SMILES CC1=C(CCCOC2=CC(=CC=C2F)C(=O)O)C(=NN1)C
Isomeric SMILES CC1=C(C(=NN1)C)CCCOC2=C(C=CC(=C2)C(=O)O)F
InChI InChI=1S/C15H17FN2O3/c1-9-12(10(2)18-17-9)4-3-7-21-14-8-11(15(19)20)5-6-13(14)16/h5-6,8H,3-4,7H2,1-2H3,(H,17,18)(H,19,20)
InChI Key WBFUHHBPNXWNCC-UHFFFAOYSA-N

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
No information available.
Summary of Clinical Use Click here for help
Acoramidis (AG10) was progressed to clinical evaluations for hereditary forms of TTR amyloidosis (variant ATTR), including polyneuropathy and cardiomyopathy of familial (ATTR). It is also being trialled for efficacy in ATTR cardiomyopathy (CM) of old age (wild-type ATTR-CM). The FDA approved acoramidis in November 2024, for both variant and wild-type ATTR-CM.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT04882735 Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN) Phase 3 Interventional Eidos Therapeutics, a BridgeBio company
NCT04988386 Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants Phase 3 Interventional Eidos Therapeutics, a BridgeBio company
NCT06563895 Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant Phase 3 Interventional Eidos Therapeutics, a BridgeBio company