bamocaftor   Click here for help

GtoPdb Ligand ID: 10553

Synonyms: compound 1 [WO2018064632A1] | VX-659 | VX659
Compound class: Synthetic organic
Comment: Bamocaftor (VX-659) is a next-generation CFTR corrector class molecule [2]. It is structurally related to elexacaftor and like other corrector molecules was designed to improve CFTR protein processing and trafficking in the presence of the F508del mutation. Bamocaftor's mechanism of action is different from that of the first generation CFTR corrector tezacaftor and its functional and clinical effects appear to be additive [2]. As such a triple combination therapeutic with tezacaftor, izacaftor (a CFTR potentiator that increases CFTR channel gating activity) and bamocaftor increases CFTR function beyond that achieved by current standard of care dual therapy tezacaftor/izacaftor, and improves clinical symptoms in patients with cystic fibrosis with one or two F508del alleles [3].
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2D Structure
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Physico-chemical Properties
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Hydrogen bond acceptors 7
Hydrogen bond donors 1
Rotatable bonds 11
Topological polar surface area 114.28
Molecular weight 591.21
XLogP 7.56
No. Lipinski's rules broken 1
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Canonical SMILES CC1CN(C(C1)(C)C)c1nc(ccc1C(=O)NS(=O)(=O)c1ccccc1)n1ccc(n1)OCCC1(CC1)C(F)(F)F
Isomeric SMILES C[C@@H]1CN(C(C1)(C)C)c1nc(ccc1C(=O)NS(=O)(=O)c1ccccc1)n1ccc(n1)OCCC1(CC1)C(F)(F)F
InChI InChI=1S/C28H32F3N5O4S/c1-19-17-26(2,3)35(18-19)24-21(25(37)34-41(38,39)20-7-5-4-6-8-20)9-10-22(32-24)36-15-11-23(33-36)40-16-14-27(12-13-27)28(29,30)31/h4-11,15,19H,12-14,16-18H2,1-3H3,(H,34,37)/t19-/m0/s1
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Summary of Clinical Use Click here for help
The VX-659/tezacaftor/izacaftor combined therapy has completd Phase 3 evaluation in patients who are homozygous for the F508del CFTR mutation, and in heterozygous carriers of the F508del allele plus a minimal function (MF) mutant allele. Click here to link to's full list of VX-659 studies.