Ataluren (PTC124)

Catalog No.S6003

For research use only.

Ataluren (PTC124) selectively induces ribosomal read-through of premature but not normal termination codons, with EC50 of 0.1 μM in HEK293 cells, may provide treatment for genetic disorders caused by nonsense mutations (e.g. CF caused by CFTR nonsense mutation). Phase 3.

Ataluren (PTC124) Chemical Structure

CAS No. 775304-57-9

Selleck's Ataluren (PTC124) has been cited by 44 publications

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Biological Activity

Description Ataluren (PTC124) selectively induces ribosomal read-through of premature but not normal termination codons, with EC50 of 0.1 μM in HEK293 cells, may provide treatment for genetic disorders caused by nonsense mutations (e.g. CF caused by CFTR nonsense mutation). Phase 3.
Features Demonstrates oral bioavailability, and an appropriate safety toxicology profile.
Targets
CFTR (nonsense mutant) [1]
(HEK293 cells)
In vitro

Compared with Gentamicin which is only active at much higher concentrations, PTC124 is a more potent nonsense-suppressing agent and exhibits 4- to 15-fold stimulation of read-through relative to controls. PTC124 (0.01-3 μM) promotes dose-dependent read-through of all three nonsense codons in HEK293 cells harboring LUC-190 nonsense alleles with the highest read-through at UGA, followed by UAG and then UAA, but it does not suppress multiple proximal nonsense codons. Like Gentamicin, PTC124 is most active when a pyrimidine (in particular cytosine, C) follows the nonsense codon. Consistent with the stable cell line reporter assay, PTC124 (17 μM) promotes significant production of dystrophin in primary muscle cells from Duchenne muscular dystrophy (DMD) patients or MDXMDX mice expressing dystrophin nonsense alleles. PTC124 selectively promotes ribosomal read-through of premature termination but not normal termination codons, even at concentrations substantially greater than the values achieving maximal activity. [1]

Cell Data
Cell Lines Assay Type Concentration Incubation Time Formulation Activity Description PMID
BAC_MMA∗EGFP MlL6SpVv[3Srb36gRZN{[Xl? NXfkclJ7OjEEoN88US=> MnexO|LDqGkEoB?= M2PkOWROW09? NHfyTphz\XO3bITzJIlvKGFic3nncolncWOjboSgbY5kemWjc3WgbY4hdWWmaXHuJJBm[WtiZnz1c5Jme2OnbnPl MVeyN|A1OTF6OR?=
Mut−/−MUTStop+/− NETKbnZHfW6ldHnvckBCe3OjeR?= MXWyNOKh|ryP MUO3NuKhcMLi NGDRSYpFVVOR NV\RXYZScW6lcnXhd4V{KHSqZTDhcY92dnRib3dCpG1WXMLibWLORS=> MUSyN|A1OTF6OR?=
HEK293T NIPJeWRHfW6ldHnvckBCe3OjeR?= M4TibVExKML3Zz;tcC=> M3XtWWROW09? MYPy[ZN1d3KnczDmeYxtNWynbnf0bEBp[XKvb37pckBiOSBq4pk8PFAhc0SjKTDpckBxNlJ|MWiteJJidnOoZXP0[YQh[2WubIO= NFX2TVAzOzB{N{[0NC=>
ML1 NFPUSYFHfW6ldHnvckBCe3OjeR?= NWf6R2ZYOy5|L{GwxsDPxE1? M1jPbVQ5KGh? MVnEUXNQ MX;pcoNz\WG|ZYOgRXJUSiCjY4Tpeol1gQ>? MWGyNlk4OTl3OR?=
ML2 M4D3SGZ2dmO2aX;uJGF{e2G7 NXvnNVBROy5|L{GwxsDPxE1? MVi0PEBp NX7SO|ZJTE2VTx?= MWHpcoNz\WG|ZYOgRXJUSiCjY4Tpeol1gQ>? M{nyRVIzQTdzOUW5
In vivo Due to functional recovery of dystrophin production, oral, intraperitoneal or combined dosing of PTC124 for 2-8 weeks partially rescues functional strength deficit in dystrophic muscles of MDX mice, and results in partial protection against contraction-induced injury in the extensor digitorum longus (EDL) muscles, as well as significant reductions in serum creatine kinase values. [1] In Cftr-/- mice expressing a human CFTR-G542X transgene, subcutaneous or oral administration of PTC124 (~60 mg/kg) suppresses the G542X nonsense mutation in a dose-dependent manner, leading to a significant restoration of human (h)CFTR protein expression and function without any effect on nonsense-mediated mRNA decay (NMD) or other aspects of mRNA stability. PTC124 treatment (60 mg/kg) restores 29% of the normal intestinal transepithelial cAMP-stimulated shortcircuit currents observed in Cftr+/+ mice, displaying a significant advantage compared with Gentamicin. [2]

Protocol (from reference)

Animal Research:[2]
  • Animal Models: Cftr-/- hCFTR-G542X transgenic mice
  • Dosages: ~60 mg/kg/day
  • Administration: Subcutaneous injection or oral administration

Solubility (25°C)

In vitro

In vivo

Add solvents to the product individually and in order
(Data is from Selleck tests instead of citations):
1% DMSO+30% polyethylene glycol+1% Tween 80
For best results, use promptly after mixing.

30 mg/mL

Chemical Information

Molecular Weight 284.24
Formula

C15H9FN2O3

CAS No. 775304-57-9
Storage 3 years -20°C powder
2 years -80°C in solvent
Smiles C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F

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Method for preparing in vivo formulation: Take μL DMSO master liquid, next addμL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O, mix and clarify.

Method for preparing in vivo formulation: Take μL DMSO master liquid, next add μL Corn oil, mix and clarify.

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Clinical Trial Information

NCT Number Recruitment Interventions Conditions Sponsor/Collaborators Start Date Phases
NCT04336826 Recruiting Drug: Ataluren Nonsene Mutation Duchenne Muscular Dystrophy PTC Therapeutics August 11 2021 Phase 2
NCT04014530 Recruiting Drug: Ataluren + Pembrolizumab Colorectal Cancer|Endometrium Cancer Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)|Merck Sharp & Dohme Corp.|PTC Therapeutics August 1 2019 Phase 1|Phase 2
NCT02758626 Completed Drug: ataluren|Drug: Placebo Epilepsy NYU Langone Health|PTC Therapeutics November 2016 Phase 2
NCT02819557 Completed Drug: Ataluren Duchenne Muscular Dystrophy PTC Therapeutics June 9 2016 Phase 2
NCT02090959 Terminated Drug: Ataluren Muscular Dystrophy Duchenne|Muscular Dystrophies|Muscular Disorders Atrophic|Muscular Diseases|Musculoskeletal Diseases|Neuromuscular Diseases|Nervous System Diseases|Genetic Diseases X-Linked|Genetic Diseases Inborn PTC Therapeutics March 20 2014 Phase 3
NCT01140451 Completed Drug: Ataluren Cystic Fibrosis PTC Therapeutics|Cystic Fibrosis Foundation August 12 2010 Phase 3

(data from https://clinicaltrials.gov, updated on 2022-01-17)

Tech Support

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