ACT-709478   Click here for help

GtoPdb Ligand ID: 12647

Synonyms: ACT709478 | compound 66b [PMID: 29116786] | NBI-827104 | NBI827104
PDB Ligand
Compound class: Synthetic organic
Comment: ACT-709478 is an orally available inhibitor of T-type voltage-gated calcium channels (Cav3.1, Cav3.2, Cav3.3) [1]. Blocking activity of these channels is proposed as a mechanism to treat idiopathic generalized epilepsies.
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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

ACT-709478 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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Click here for structure editor
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 6
Hydrogen bond donors 1
Rotatable bonds 8
Topological polar surface area 80.85
Molecular weight 425.41
XLogP 2.13
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 C1=C(C=CC(=C1)C2(CC2)C(F)(F)F)CC(=O)NC3=NN(C=C3)CC4=CC=C(C#N)C=N4
Isomeric SMILES C1CC1(C2=CC=C(C=C2)CC(=O)NC3=NN(C=C3)CC4=NC=C(C=C4)C#N)C(F)(F)F
InChI InChI=1S/C22H18F3N5O/c23-22(24,25)21(8-9-21)17-4-1-15(2-5-17)11-20(31)28-19-7-10-30(29-19)14-18-6-3-16(12-26)13-27-18/h1-7,10,13H,8-9,11,14H2,(H,28,29,31)
InChI Key LSYANGLAZUZYFX-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
ACT-709478 was progressed to clinical studies to determine anti-epileptic efficacy.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT03165097 Study to Evaluate Safety and Tolerability of ACT-709478 in Healthy Subjects Phase 1 Interventional Idorsia Pharmaceuticals Ltd.
NCT03239691 A Study to Evaluate the Effect of ACT-709478 in Photosensitive Epilepsy Patients Phase 2 Interventional Idorsia Pharmaceuticals Ltd.
NCT04123288 A Study to Compare a Single-dose of Two Different Formulations of ACT-709478 in Healthy Men Phase 1 Interventional Idorsia Pharmaceuticals Ltd.
NCT05301894 Extension Study to Evaluate NBI-827104 in Pediatric Subjects With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep Phase 2 Interventional Neurocrine Biosciences
NCT04625101 Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-827104 in Pediatric Subjects With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep Phase 2 Interventional Neurocrine Biosciences