Ataluren - CAS 775304-57-9
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Not Intended for Therapeutic Use. For research use only.
Ataluren is an investigational new drug designed to enable the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation. Ataluren is currently being investigated for use in patients with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) and cystic fibrosis (nmCF).
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PTC124; PTC-124.
Current Developer:
PTC Therapeutics.
1.Therapeutic effect of prenatal alkalization and PTC124 in Na(+)/HCO3(-) cotransporter 1 p.W516* knock-in mice.
Fang YW1, Yang SS2, Chau T3, Nakamura M4, Yamazaki O4, Seki G4, Yamada H4, Hsu HM5, Cheng CJ2, Lin SH2. Gene Ther. 2015 May;22(5):374-81. doi: 10.1038/gt.2015.7. Epub 2015 Feb 26.
We created Na(+)/HCO3(-) cotransporter 1 (NBCe1) p.W516* knock-in mice as a model of isolated proximal renal tubular acidosis showing early lethality associated with severe metabolic acidosis to investigate the therapeutic effects of prenatal alkalization or posttranscriptional control 124 (PTC124). NBCe1(W516*/W516*) mice were treated with non-alkalization (control, n=12), prenatal alkalization postcoitus (prenatal group, n=7) and postnatal alkalization from postnatal day 6 (postnatal group, n=12). Mutation-specific therapy, PTC124 (60 mg kg(-1)) or gentamicin (30 mg kg(-1)), was administered intraperitoneally from postnatal day 6. Blood and urine biochemistry, acid-base analysis, survival rate and renal histology were examined. NBCe1 protein, mRNA abundance and activity ex vivo were assessed after PTC124 and gentamicin treatment. Prenatal group mice had similar initial body weight to wild-type mice and achieved significant weight gain thereafter compared with controls.
2.Strategies in early clinical development for the treatment of basic defects of cystic fibrosis.
Dhooghe B1, Haaf JB1, Noel S1, Leal T1. Expert Opin Investig Drugs. 2016 Apr;25(4):423-36. doi: 10.1517/13543784.2016.1154041. Epub 2016 Mar 7.
INTRODUCTION: Twenty-six years after the identification of the gene responsible for cystic fibrosis (CF), controversies still surround the pathogenesis of the disease that continues to burden and shorten lives. Therefore, finding effective therapeutic strategies that target the basic defect of CF is crucially needed.
3.Enhancement of premature stop codon readthrough in the CFTR gene by Ataluren (PTC124) derivatives.
Pibiri I1, Lentini L2, Melfi R2, Gallucci G2, Pace A3, Spinello A2, Barone G1, Di Leonardo A4. Eur J Med Chem. 2015 Aug 28;101:236-44. doi: 10.1016/j.ejmech.2015.06.038. Epub 2015 Jun 21.
Premature stop codons are the result of nonsense mutations occurring within the coding sequence of a gene. These mutations lead to the synthesis of a truncated protein and are responsible for several genetic diseases. A potential pharmacological approach to treat these diseases is to promote the translational readthrough of premature stop codons by small molecules aiming to restore the full-length protein. The compound PTC124 (Ataluren) was reported to promote the readthrough of the premature UGA stop codon, although its activity was questioned. The potential interaction of PTC124 with mutated mRNA was recently suggested by molecular dynamics (MD) studies highlighting the importance of H-bonding and stacking π-π interactions. To improve the readthrough activity we changed the fluorine number and position in the PTC124 fluoroaryl moiety. The readthrough ability of these PTC124 derivatives was tested in human cells harboring reporter plasmids with premature stop codons in H2BGFP and FLuc genes as well as in cystic fibrosis (CF) IB3.
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