research use only

Ataluren (PTC124) CFTR inhibitor

Name: Ataluren (PTC124)
Brand: Selleck Chemicals
SKU: S6003
Availability: InStock
Rating: 5 (47 reviews)

Cat.No.S6003

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

Chemical Structure

Molecular Weight: 284.24

Jump to Mechanism of Action Cell Culture & Working Concentration Solubility Chemical Information, Storage & Stability Specificity

Quality Control

Batch: Purity: 99.96%

99.96

Related Targets	CD markers AChR Calcium Channel Sodium Channel Potassium Channel GABA Receptor TRP Channel ATPase GluR NMDAR P2 Receptor Proton Pump P-gp SGLT Chloride Channel MCT Amino acid transporter GLUT CRM1 VDAC BCRP NKCC OCT NCX OAT VMAT Aquaporin EAAT GlyT GlyR
Related Products	Tezacaftor (VX-661) Elexacaftor (VX-445) CFTRinh-172 GLPG1837 FDL169 Galicaftor (ABBV-2222) IOWH032 GlyH-101 PPQ-102 Vanzacaftor (VX-121) Nesolicaftor (PTI-428) VX-561(CTP-656) KM11060 Icenticaftor (QBW251) Steviol (Hydroxydehydrostevic acid)

Cell Culture, Treatment & Working Concentration

Cell Lines	Assay Type	Concentration	Incubation Time	Formulation	Activity Description
BAC_MMA∗EGFP	Function Assay	20 μM	72 h	DMSO	results in a significant increase in median peak fluorescence
Mut−/−MUTStop+/−	Function Assay	20 μM	72 h	DMSO	increases the amount of MUT mRNA
HEK293T	Function Assay	10 µg/ml		DMSO	restores full-length harmonin a1 (∼80 kDa) in p.R31X-transfected cells
ML1	Function Assay	3.3/10 μM	48 h	DMSO	increases ARSB activity
ML2	Function Assay	3.3/10 μM	48 h	DMSO	increases ARSB activity
Click to View More Cell Line Experimental Data

Chemical Information, Storage & Stability

Molecular Weight	284.24	Formula	C₁₅H₉FN₂O₃	Storage (From the date of receipt)	3 years-20°Cpowder
CAS No.	775304-57-9	Download SDF		Storage of Stock Solutions	1 year-80°Cin solvent 1 month-20°Cin solvent
Synonyms	N/A			Smiles	C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F

Solubility

In vitro
Batch:

DMSO : 57 mg/mL (200.53 mM)
(Moisture-contaminated DMSO may reduce solubility. Use fresh, anhydrous DMSO.)

Water : Insoluble

Ethanol : Insoluble

Molarity Calculator

Mass	Concentration	Volume	Molecular Weight
Mass value Mass unit	Concentration value Concentration unit	Volume value Volume unit	Molecular weight (g/mol)

Dilution Calculator Molecular Weight Calculator

In vivo batch selection In vivo Batch:
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.
Clear solution	5%DMSO 1 40%PEG300 2 5%Tween80 3 50%ddH2O Validated by Selleck labs. Should you need adjustments to this formulation, contact our sales team for custom testing.	0.700mg/ml (2.46mM)	Taking the 1 mL working solution as an example, add 50 μL of 14 mg/ml clarified DMSO stock solution to 400 μL of PEG300, mix evenly to clarify it; add 50 μL of Tween80 to the above system, mix evenly to clarify; then continue to add 500 μL of ddH2O to adjust the volume to 1 mL. The mixed solution should be used immediately for optimal results.
Clear solution	5% DMSO 1 95% Corn oil Validated by Selleck labs. Should you need adjustments to this formulation, contact our sales team for custom testing.	0.500mg/ml (1.76mM)	Taking the 1 mL working solution as an example, add 50 μL of 10 mg/ml clear DMSO stock solution to 950 μL of corn oil and mix evenly. The mixed solution should be used immediately for optimal results.
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.

In vivo Formulation Calculator (Clear solution)

Step 1: Enter information below (Recommended: An additional animal making an allowance for loss during the experiment)

Dosage: mg/kg

Average weight of animals: g

Dosing volume per animal: μL

Number of animals:

Step 2: Enter the in vivo formulation (This is only the calculator, not formulation. Please contact us first if there is no in vivo formulation at the solubility Section.)

% DMSO

% Tween 80

% ddH₂O

% DMSO

Calculation results:

Working concentration: mg/ml;

Method for preparing DMSO master liquid: mg drug pre-dissolved in μL DMSO ( Master liquid concentration mg/mL, Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug. )

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.

Note: 1. Please make sure the liquid is clear before adding the next solvent.
2. Be sure to add the solvent(s) in order. You must ensure that the solution obtained, in the previous addition, is a clear solution before proceeding to add the next solvent. Physical methods such
as vortex, ultrasound or hot water bath can be used to aid dissolving.

Mechanism of Action

Features	Demonstrates oral bioavailability, and an appropriate safety toxicology profile.
Targets/IC₅₀/Ki	CFTR (nonsense mutant) ^[1] (HEK293 cells)
In vitro	Ataluren (PTC124) is a more potent nonsense-suppressing agent and exhibits 4- to 15-fold stimulation of read-through relative to controls. This compound (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. It 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. It 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]
In vivo	Due to functional recovery of dystrophin production, oral, intraperitoneal or combined dosing of Ataluren (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 this compound (~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. Treatment with it (60 mg/kg) restores 29% of the normal intestinal transepithelial cAMP-stimulated shortcircuit currents observed in Cftr+/+ mice, displaying a significant advantage. ^[2]
References	[1] https://pubmed.ncbi.nlm.nih.gov/17450125/ [2] https://pubmed.ncbi.nlm.nih.gov/18272502/

Clinical Trial Information

(data from https://clinicaltrials.gov, updated on 2024-05-22)

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT04336826	Completed	Nonsene Mutation Duchenne Muscular Dystrophy	PTC Therapeutics	December 29 2021	Phase 2
NCT04014530	Recruiting	Colorectal Cancer\|Endometrium Cancer	Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)\|Merck Sharp & Dohme LLC\|PTC Therapeutics	August 1 2019	Phase 1\|Phase 2
NCT02758626	Completed	Epilepsy	NYU Langone Health\|PTC Therapeutics	November 2016	Phase 2
NCT02819557	Completed	Duchenne Muscular Dystrophy	PTC Therapeutics	June 9 2016	Phase 2
NCT02090959	Terminated	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	Cystic Fibrosis	PTC Therapeutics\|Cystic Fibrosis Foundation	August 12 2010	Phase 3

Tech Support

Handling Instructions

Tel: +1-832-582-8158 Ext:3

If you have any other enquiries, please leave a message.

C1=C0/X	C1:	LOG(C1):
C2=C1/X	C2:	LOG(C2):
C3=C2/X	C3:	LOG(C3):
C4=C3/X	C4:	LOG(C4):
C5=C4/X	C5:	LOG(C5):
C6=C5/X	C6:	LOG(C6):
C7=C6/X	C7:	LOG(C7):
C8=C7/X	C8:	LOG(C8):