VX-661

• CAT Number: I000562
• CAS Number: 1152311-62-0
• Molecular Formula: C26H27F3N2O6
• Molecular Weight: 520.50
• Purity: 98%
• Synonyms: 1-(2,2-difluoro-1,3-benzodioxol-5-yl)-N-[1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)indol-5-yl]cyclopropane-1-carboxamide
• Target: CFTR
• Target Protein: P13569
• Solubility: DMSO 100 mg/ml; Water < 1 mg/ml
• Appearance: Solid
• Storage: Dry, dark and at 2 - 8 °C for six months or -20°C for two years.
• IC50: Tezacaftor (2 μM, 24 h) increases the expression level of N1303K-CFTR in HEK293 and CFBE cells
• InChI: InChI=1S/C26H27F3N2O6/c1-24(2,13-33)22-8-14-7-18(17(27)10-19(14)31(22)11-16(34)12-32)30-23(35)25(5-6-25)15-3-4-20-21(9-15)37-26(28,29)36-20/h3-4,7-10,16,32-34H,5-6,11-13H2,1-2H3,(H,30,35)/t16-/m1/s1
• InChIKey: MJUVRTYWUMPBTR-MRXNPFEDSA-N
• SMILES: CC(C)(CO)C1=CC2=CC(=C(C=C2N1C[C@H](CO)O)F)NC(=O)C3(CC3)C4=CC5=C(C=C4)OC(O5)(F)F

VX-661 (Cat No.: I000562) is a compound that has been developed as a potential therapy for cystic fibrosis (CF). It is a corrector agent designed to address the underlying genetic defect found in CF, which is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. VX-661 acts by increasing the trafficking and stability of the CFTR protein at the cell surface, thereby improving its function. It is often used in combination with other CFTR modulators, such as ivacaftor, to enhance the therapeutic effect. Clinical studies have shown promising results in improving lung function and reducing symptoms in CF patients with specific CFTR mutations.

Reference

[1]. Study of VX-661 Alone and in Combination With VX-770 in Subjects Homozygous to the F508del-CFTR Mutation
Abstract
The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) effects of VX-661 alone and when coadministered with VX-770 in subjects with CF who are homozygous for the F508del-CFTR mutation.