Ataluren (PTC124)

Catalog No.S6003

Ataluren (PTC124) Chemical Structure

Molecular Weight(MW): 284.24

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.

Size Price Stock Quantity  
In DMSO USD 97 In stock
USD 75 In stock
USD 170 In stock
USD 270 In stock
USD 370 In stock
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Cited by 19 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 M3jqOmZ2dmO2aX;uJGF{e2G7 NYTncGdzOjEEoN88US=> MXG3NuKhcMLi MX;EUXNQ NYfrZmZbemW|dXz0d{BqdiCjIIPp[45q\mmlYX70JIlv[3KnYYPlJIlvKG2nZHnhckBx\WGtIH\seY9z\XOlZX7j[S=> M3f0SFI{ODRzMUi5
Mut−/−MUTStop+/− MXTGeY5kfGmxbjDBd5NigQ>? MVmyNOKh|ryP MYK3NuKhcMLi Mme0SG1UVw>? M1\vTolv[3KnYYPld{B1cGViYX3veY51KG:owrDNWXTDqG2UTlG= NHvVfXQzOzB2MUG4PS=>
HEK293T M1[5VGZ2dmO2aX;uJGF{e2G7 NF3IRVUyOCEEtXevcYw> NXjtc4VvTE2VTx?= MnjzdoV{fG:{ZYOg[pVtdC2uZX7neIghcGG{bX;ubY4h[TFiKPMIwFgxKGuGYTmgbY4heC6UM{HYMZRz[W6|ZnXjeIVlKGOnbHzz MXWyN|AzPzZ2MB?=
ML1 MXrGeY5kfGmxbjDBd5NigQ>? NIWzbJk{NjNxMUFCpO69VQ>? NGX0UIM1QCCq NEC2foxFVVOR NXHQVFl4cW6lcnXhd4V{KEGUU1KgZYN1cX[rdIm= M{\IflIzQTdzOUW5
ML2 NYfSPWFiTnWwY4Tpc44hSXO|YYm= NX\aTpFTOy5|L{GwxsDPxE1? Ml7NOFghcA>? M1[wdGROW09? MmTkbY5kemWjc3XzJGFTW0JiYXP0bZZqfHl? MWmyNlk4OTl3OR?=

... Click to View More Cell Line Experimental Data

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

Animal Research:[2]
+ Expand
  • Animal Models: Cftr-/- hCFTR-G542X transgenic mice
  • Formulation: Dissolved in DMSO, and diluted in saline
  • Dosages: ~60 mg/kg/day
  • Administration: Subcutaneous injection or oral administration
    (Only for Reference)

Solubility (25°C)

In vitro DMSO 57 mg/mL (200.53 mM)
Water Insoluble
Ethanol Insoluble
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

* Please note that Selleck tests the solubility of all compounds in-house, and the actual solubility may differ slightly from published values. This is normal and is due to slight batch-to-batch variations.

Chemical Information

Molecular Weight 284.24
Formula

C15H9FN2O3

CAS No. 775304-57-9
Storage powder
in solvent
Synonyms N/A

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Clinical Trial Information

NCT Number Recruitment interventions Conditions Sponsor/Collaborators Start Date Phases
NCT04014530 Not yet 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 Active not recruiting Drug: ataluren|Drug: Placebo Epilepsy NYU Langone Health|PTC Therapeutics November 2016 Phase 2
NCT02456103 Terminated Drug: Ataluren Cystic Fibrosis PTC Therapeutics July 31 2015 Phase 3
NCT02090959 Completed 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 31 2014 Phase 3
NCT01826487 Completed Drug: Ataluren|Drug: Placebo 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 2013 Phase 3
NCT01557400 Completed Drug: Ataluren Duchenne Muscular Dystrophy|Becker Muscular Dystrophy|Dystrophinopathy PTC Therapeutics May 31 2012 Phase 3

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Cell Lines Assay Type Concentration Incubation Time Formulation Activity Description PMID