Ataluren

Summary

Ataluren (CHEMBL256997) is an approved small molecule (ATC M09AX03); indicated across 7 conditions including duchenne muscular dystrophy and cystic fibrosis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: M09AX03
• Indications: 7 conditions
• Clinical trials: 32
• Chemistry: 284.24 Da · C15H9FN2O3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL256997
Name	Ataluren
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	11219835
ATC	M09AX03
Molecular formula	C15H9FN2O3
Molecular weight	284.24
InChIKey	OOUGLTULBSNHNF-UHFFFAOYSA-N

SMILES: C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F

IUPAC name: 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid

Also known as: Ataluren, Atalureno, PTC-124, PTC124, Translarna, SID85852879, SID87334013, ATALUREN, SID144206184, SID99460885, SID170466862, PTC124 (ATALUREN)

Patent coverage: 597 distinct patent families (1,920 SureChEMBL compound mentions), from 4 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 4 (assay-derived). Sample: Nuclear receptor ROR-gamma, Protein deacetylase HDAC6, Amine oxidase [flavin-containing] A, Flavin reductase (NADPH).

Bioactivity

ChEMBL activities: 5 potent at pChembl ≥ 5 of 5 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
BLVRB	6.7	Kd	200	nM	CHEMBL_ACT_24380459
BLVRB	6.7	Kd	199.5	nM	CHEMBL_ACT_24380460
HDAC6	6.52	IC50	300.9	nM	CHEMBL_ACT_23140991
P51450	6.15	Potency	707.9	nM	CHEMBL_ACT_4100429
MAOA	5.28	AC50	5300	nM	CHEMBL_ACT_25160287

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

7 indications (2 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
Duchenne muscular dystrophy	4	MONDO:0010679	MONDO:0010311
cystic fibrosis	3	MONDO:0009061	MONDO:0009061
epilepsy	2	MONDO:0005027	EFO:0000474
hemophilia A	2	MONDO:0010602	MONDO:0010602
hemophilia B	2	MONDO:0010604	MONDO:0010604
aniridia	2	MONDO:0019172	MONDO:0019172

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 32.

Phase distribution

Phase	Trials
PHASE2	18
PHASE3	10
PHASE4	2
PHASE1/PHASE2	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03256799	PHASE4	COMPLETED	Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations
NCT03256968	PHASE4	COMPLETED	PTC Study to Evaluate Ataluren in Combination With Ivacaftor
NCT00803205	PHASE3	COMPLETED	Study of Ataluren (PTC124™) in Cystic Fibrosis
NCT01140451	PHASE3	COMPLETED	Extension Study of Ataluren (PTC124) in Cystic Fibrosis
NCT01247207	PHASE3	COMPLETED	Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
NCT01557400	PHASE3	COMPLETED	Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
NCT01826487	PHASE3	COMPLETED	Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT02090959	PHASE3	TERMINATED	An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT02107859	PHASE3	TERMINATED	Study of Ataluren (PTC124) in Cystic Fibrosis
NCT02139306	PHASE3	COMPLETED	Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
NCT02456103	PHASE3	TERMINATED	Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
NCT03179631	PHASE3	COMPLETED	Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT04014530	PHASE1/PHASE2	RECRUITING	Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO Study
NCT00234663	PHASE2	COMPLETED	PTC124 for Cystic Fibrosis
NCT00237380	PHASE2	COMPLETED	Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis
NCT00264888	PHASE2	COMPLETED	Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
NCT00351078	PHASE2	COMPLETED	PTC124 for the Treatment of Cystic Fibrosis
NCT00458341	PHASE2	COMPLETED	A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
NCT00592553	PHASE2	COMPLETED	Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT00759876	PHASE2	TERMINATED	Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
NCT00847379	PHASE2	TERMINATED	Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT00947193	PHASE2	TERMINATED	Study of Ataluren (PTC124) in Hemophilia A and B
NCT01009294	PHASE2	TERMINATED	Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
NCT01141075	PHASE2	TERMINATED	Ataluren for Nonsense Mutation Methylmalonic Acidemia
NCT02647359	PHASE2	COMPLETED	Study of Ataluren in Participants With Nonsense Mutation Aniridia
NCT02758626	PHASE2	COMPLETED	Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
NCT02819557	PHASE2	COMPLETED	Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
NCT03648827	PHASE2	COMPLETED	A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT03796637	PHASE2	COMPLETED	A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
NCT04117880	PHASE2	WITHDRAWN	A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
NCT04336826	PHASE2	COMPLETED	A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT02409004	PHASE1	COMPLETED	Effects of Rifampin on the Pharmacokinetics of Ataluren

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 3 clinical and 12 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).