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MedKoo Cat#: 205786 | Name: Ataluren
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Description:

WARNING: This product is for research use only, not for human or veterinary use.

Ataluren (PTC124) is an orally bioavailable small molecule that promotes ribosomal readthrough of premature termination codons (PTCs), enabling the production of full-length, functional proteins in genetic disorders caused by nonsense mutations. In vitro, Ataluren demonstrates an EC₅₀ of 0.1 μM in HEK293 cells, effectively inducing readthrough of all three stop codons (UGA > UAG > UAA) .

Chemical Structure

Ataluren
Ataluren
CAS#775304-57-9

Theoretical Analysis

MedKoo Cat#: 205786

Name: Ataluren

CAS#: 775304-57-9

Chemical Formula: C15H9FN2O3

Exact Mass: 284.0597

Molecular Weight: 284.24

Elemental Analysis: C, 63.38; H, 3.19; F, 6.68; N, 9.86; O, 16.89

Price and Availability

Size Price Availability Quantity
100mg USD 550.00 2 Weeks
200mg USD 950.00 2 Weeks
500mg USD 1,450.00 2 Weeks
1g USD 2,450.00 2 Weeks
2g USD 3,850.00 2 Weeks
5g USD 5,950.00 2 Weeks
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Related CAS #
No Data
Synonym
PTC124; PTC 124; PTC-124; Ataluren.
IUPAC/Chemical Name
3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid
InChi Key
OOUGLTULBSNHNF-UHFFFAOYSA-N
InChi Code
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)
SMILES Code
O=C(O)C1=CC=CC(C2=NOC(C3=CC=CC=C3F)=N2)=C1
Appearance
Solid powder
Purity
>98% (or refer to the Certificate of Analysis)
Shipping Condition
Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.
Storage Condition
Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).
Solubility
Soluble in DMSO, not in water
Shelf Life
>5 years if stored properly
Drug Formulation
This drug may be formulated in DMSO
Stock Solution Storage
0 - 4 C for short term (days to weeks), or -20 C for long term (months).
HS Tariff Code
2934.99.9001
More Info
        

Preparing Stock Solutions

The following data is based on the product molecular weight 284.24 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.

Recalculate based on batch purity %
Concentration / Solvent Volume / Mass 1 mg 5 mg 10 mg
1 mM 1.15 mL 5.76 mL 11.51 mL
5 mM 0.23 mL 1.15 mL 2.3 mL
10 mM 0.12 mL 0.58 mL 1.15 mL
50 mM 0.02 mL 0.12 mL 0.23 mL
1: McDonald CM, Muntoni F, Penematsa V, Jiang J, Kristensen A, Bibbiani F, Goodwin E, Gordish-Dressman H, Morgenroth L, Werner C, Li J, Able R, Trifillis P, Tulinius M; Study 019 investigators. Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients. J Comp Eff Res. 2022 Feb;11(3):139-155. doi: 10.2217/cer-2021-0196. Epub 2021 Nov 18. PMID: 34791888; PMCID: PMC8787621. 2: Michorowska S. Ataluren-Promising Therapeutic Premature Termination Codon Readthrough Frontrunner. Pharmaceuticals (Basel). 2021 Aug 9;14(8):785. doi: 10.3390/ph14080785. PMID: 34451881; PMCID: PMC8398184. 3: Fox H, Millington L, Mahabeer I, van Ruiten H. Duchenne muscular dystrophy. BMJ. 2020 Jan 23;368:l7012. doi: 10.1136/bmj.l7012. PMID: 31974125. 4: McDonald CM, Campbell C, Torricelli RE, Finkel RS, Flanigan KM, Goemans N, Heydemann P, Kaminska A, Kirschner J, Muntoni F, Osorio AN, Schara U, Sejersen T, Shieh PB, Sweeney HL, Topaloglu H, Tulinius M, Vilchez JJ, Voit T, Wong B, Elfring G, Kroger H, Luo X, McIntosh J, Ong T, Riebling P, Souza M, Spiegel RJ, Peltz SW, Mercuri E; Clinical Evaluator Training Group; ACT DMD Study Group. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2017 Sep 23;390(10101):1489-1498. doi: 10.1016/S0140-6736(17)31611-2. Epub 2017 Jul 17. PMID: 28728956. 5: Sermet-Gaudelus I, Boeck KD, Casimir GJ, Vermeulen F, Leal T, Mogenet A, Roussel D, Fritsch J, Hanssens L, Hirawat S, Miller NL, Constantine S, Reha A, Ajayi T, Elfring GL, Miller LL. Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis. Am J Respir Crit Care Med. 2010 Nov 15;182(10):1262-72. doi: 10.1164/rccm.201001-0137OC. Epub 2010 Jul 9. PMID: 20622033. 6: Wang D, Xue X, Gunn G, Du M, Siddiqui A, Weetall M, Keeling KM. Ataluren suppresses a premature termination codon in an MPS I-H mouse. J Mol Med (Berl). 2022 Aug;100(8):1223-1235. doi: 10.1007/s00109-022-02232-0. Epub 2022 Jul 20. PMID: 35857082; PMCID: PMC9329424. 7: Berger J, Li M, Berger S, Meilak M, Rientjes J, Currie PD. Effect of Ataluren on dystrophin mutations. J Cell Mol Med. 2020 Jun;24(12):6680-6689. doi: 10.1111/jcmm.15319. Epub 2020 Apr 28. PMID: 32343037; PMCID: PMC7299694. 8: Ryan NJ. Ataluren: first global approval. Drugs. 2014 Sep;74(14):1709-14. doi: 10.1007/s40265-014-0287-4. PMID: 25193627. 9: Finkel RS. Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124). J Child Neurol. 2010 Sep;25(9):1158-64. doi: 10.1177/0883073810371129. Epub 2010 Jun 2. PMID: 20519671; PMCID: PMC3674569. 10: Lentini L, Melfi R, Cancemi P, Pibiri I, Di Leonardo A. Caffeine boosts Ataluren's readthrough activity. Heliyon. 2019 Jun 21;5(6):e01963. doi: 10.1016/j.heliyon.2019.e01963. PMID: 31294114; PMCID: PMC6595402. 11: Sheikh O, Yokota T. Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies. Expert Opin Investig Drugs. 2021 Feb;30(2):167-176. doi: 10.1080/13543784.2021.1868434. Epub 2021 Jan 6. PMID: 33393390. 12: Wilschanski M, Miller LL, Shoseyov D, Blau H, Rivlin J, Aviram M, Cohen M, Armoni S, Yaakov Y, Pugatsch T, Cohen-Cymberknoh M, Miller NL, Reha A, Northcutt VJ, Hirawat S, Donnelly K, Elfring GL, Ajayi T, Kerem E. Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis. Eur Respir J. 2011 Jul;38(1):59-69. doi: 10.1183/09031936.00120910. Epub 2011 Jan 13. Erratum in: Eur Respir J. 2011 Oct;38(4):996. Pugatch, T [corrected to Pugatsch, T]. PMID: 21233271. 13: Wu MH, Lu RY, Yu SJ, Tsai YZ, Lin YC, Bai ZY, Liao RY, Hsu YC, Chen CC, Cai BH. PTC124 Rescues Nonsense Mutation of Two Tumor Suppressor Genes NOTCH1 and FAT1 to Repress HNSCC Cell Proliferation. Biomedicines. 2022 Nov 16;10(11):2948. doi: 10.3390/biomedicines10112948. PMID: 36428516; PMCID: PMC9687978. 14: McDonald CM, Henricson EK, Abresch RT, Florence J, Eagle M, Gappmaier E, Glanzman AM; PTC124-GD-007-DMD Study Group; Spiegel R, Barth J, Elfring G, Reha A, Peltz SW. The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study. Muscle Nerve. 2013 Sep;48(3):357-68. doi: 10.1002/mus.23905. Epub 2013 Jul 17. PMID: 23674289; PMCID: PMC3826053. 15: Dori A, Guglieri M, Scutifero M, Passamano L, Trabacca A, Politano L. Can symptomatic nmDuchenne carriers benefit from treatment with ataluren? Results of 193-month follow-up. Acta Myol. 2022 Jun 30;41(2):101. PMID: 35919205; PMCID: PMC9299955. 16: Devinsky O, King L, Bluvstein J, Friedman D. Ataluren for drug-resistant epilepsy in nonsense variant-mediated Dravet syndrome and CDKL5 deficiency disorder. Ann Clin Transl Neurol. 2021 Mar;8(3):639-644. doi: 10.1002/acn3.51306. Epub 2021 Feb 4. PMID: 33538404; PMCID: PMC7951093. 17: Ma J, Risher N, Northcutt V, Moon YC, Weetall M, Welch E, Colacino J, Almstead N, Kong R. Ataluren metabolism: Ataluren-O-1β-acyl glucuronide is a stable circulating metabolite in mouse, rat, dog and human. Drug Metab Pharmacokinet. 2021 Jun;38:100393. doi: 10.1016/j.dmpk.2021.100393. Epub 2021 Mar 30. PMID: 33872944. 18: Huang S, Bhattacharya A, Ghelfi MD, Li H, Fritsch C, Chenoweth DM, Goldman YE, Cooperman BS. Ataluren binds to multiple protein synthesis apparatus sites and competitively inhibits release factor-dependent termination. Nat Commun. 2022 May 6;13(1):2413. doi: 10.1038/s41467-022-30080-6. PMID: 35523781; PMCID: PMC9076611. 19: Peltz SW, Welch EM, Jacobson A, Trotta CR, Naryshkin N, Sweeney HL, Bedwell DM. Nonsense suppression activity of PTC124 (ataluren). Proc Natl Acad Sci U S A. 2009 Jun 23;106(25):E64; author reply E65. doi: 10.1073/pnas.0901936106. Epub 2009 Jun 8. PMID: 19506240; PMCID: PMC2700894. 20: Zainal Abidin N, Haq IJ, Gardner AI, Brodlie M. Ataluren in cystic fibrosis: development, clinical studies and where are we now? Expert Opin Pharmacother. 2017 Sep;18(13):1363-1371. doi: 10.1080/14656566.2017.1359255. Epub 2017 Aug 1. PMID: 28730885.