VX-809

VX-809 is a small molecule that corrects the processing of cystic fibrosis transmembrane conductance regulator (CFTR) proteins bearing the F508 deletion (F508del) mutation carried by 90% of cystic fibrosis patients. It enhances F508del-CFTR protein maturation and restores chloride transport function in FRT cells (EC50s = 100 and 500 nM, respectively). VX-809 increases the amount of F508del-CFTR protein exiting the endoplasmic reticulum in HEK293 cells expressing the mutant receptor. It also increases chloride and fluid transport in cultured human bronchial epithelial cells isolated from cystic fibrosis patients carrying the F508del-CFTR mutation. Formulations containing VX-809 are being investigated clinically for the treatment of cystic fibrosis.Formal Name: 3-[6-[[[1-(2,2-difluoro-1,3-benzodioxol-5-yl)cyclopropyl]carbonyl]amino]-3-methyl-2-pyridinyl]-benzoic acid. CAS Number: 936727-05-8. Synonyms: Lumacaftor, VRT-826809. Molecular Formula: C24H18F2N2O5. Formula Weight: 452.4. Purity: >98%. Formulation: (Request formulation change), A crystalline solid. Solubility: DMF: 30 mg/ml, DMSO: 30 mg/ml, DMSO:PBS (pH 7.2) (1:3): 0.25 mg/ml, Ethanol: 25 mg/ml. lambdamax: 217, 291 nm. SMILES: OC(C1=CC(C2=C(C)C=CC(NC(C3(CC3)C4=CC(OC(F)(F)O5)=C5C=C4)=O)=N2)=CC=C1)=O. InChi Code: InChI=1S/C24H18F2N2O5/c1-13-5-8-19(27-20(13)14-3-2-4-15(11-14)21(29)30)28-22(31)23(9-10-23)16-6-7-17-18(12-16)33-24(25,26)32-17/h2-8,11-12H,9-10H2,1H3,(H,29,30)(H,27,28,31). InChi Key: UFSKUSARDNFIRC-UHFFFAOYSA-N.