Details of the Drug

General Information of Drug (ID: DM4GXW3)

Drug Name

Ataluren

Synonyms

Ataluren; 775304-57-9; PTC124; 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid; PTC-124; 3-[5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid; PTC124 (Ataluren); PTC 124; UNII-K16AME9I3V; Ataluren (PTC124); K16AME9I3V; NCGC00168759-02; Translarna; AK-31904; Benzoic acid, 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]-; DSSTox_CID_26776; DSSTox_RID_81895; DSSTox_GSID_46776; Benzoic acid, 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)-; CAS-775304-57-9; Ataluren [USAN:INN]; Translarna (TN); PTC124,Ataluren; Ataluren(ptc124)

Indication

Disease Entry	ICD 11	Status	REF
Cystic fibrosis	CA25	Phase 3	[1]
Dravet syndrome	8A61.11	Phase 2	[2]
Muscular dystrophy	8C70	Phase 2	[2]
Aniridia	LA11.3	Phase 1	[1]
Mucopolysaccharidosis	5C56.3	Phase 1	[1]
Duchenne dystrophy	8C70	Application submitted	[2]
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Drug Type

Small molecular drug

Structure

3D MOL

2D MOL

#Ro5 Violations (Lipinski): 0

Molecular Weight (mw)

284.24

Logarithm of the Partition Coefficient (xlogp)

3.1

Rotatable Bond Count (rotbonds)

Hydrogen Bond Donor Count (hbonddonor)

Hydrogen Bond Acceptor Count (hbondacc)

ADMET Property

Elimination: In the urine, unchanged ataluren and the acyl glucuronide metabolite account for less than 1% and 49%, respectively, of the administered dose [3]
Half-life: The concentration or amount of drug in body reduced by one-half in 2 - 6 hours [3]
Metabolism: The drug is metabolized via the conjugation via uridine diphosphate glucuronosyltransferase (UGT) enzymes (predominantly UGT1A9) in liver and intestine [3]

Chemical Identifiers

Formula: C15H9FN2O3
IUPAC Name: 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid
Canonical SMILES: C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F
InChI: InChI=1S/C15H9FN2O3/c16-12-7-2-1-6-11(12)14-17-13(18-21-14)9-4-3-5-10(8-9)15(19)20/h1-8H,(H,19,20)
InChIKey: OOUGLTULBSNHNF-UHFFFAOYSA-N

Cross-matching ID

PubChem CID: 11219835
ChEBI ID: CHEBI:94805
CAS Number: 775304-57-9
DrugBank ID: DB05016
TTD ID: D09IRQ
INTEDE ID: DR0149

Repurposed Drugs (RPD)

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Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Dystrophin nonsense mutant (DMD)	TT2TNRM	DMD_HUMAN	Modulator	[4]

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Drug-Metabolizing Enzyme (DME)

DME Name	DME ID	UniProt ID	MOA	REF
UDP-glucuronosyltransferase 1A9 (UGT1A9)_{Main DME}	DE85D2P	UD19_HUMAN	Substrate	[5]

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Molecular Interaction Atlas (MIA)

MIA Detail

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References

1	Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
2	Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
3	EMA Product Label
4	URL: http://www.guidetopharmacology.org Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 7341).
5	Ataluren pharmacokinetics in healthy Japanese and Caucasian subjects. Clin Pharmacol Drug Dev. 2019 Feb;8(2):172-178.