Posenacaftor (PTI-801) sodium is a newly developed p.Phe508del-CFTR corrector with EC50 of 0.52 uM (HS-YFP quenching rate). Posenacaftor (PTI-801) at 1 and 3 μM concentrations also demonstrated greater efficacy in rescuing p.Phe508del-CFTR function than VX-661 at 5 μM concentration. Posenacaftor (PTI-801) rescues p.Phe508del-CFTR processing, PM trafficking, and channel function (upon agonist stimulation) with greater correction effects in combination with ABBV-2222, FDL-169, VX-661, or VX-809, but not with VX-445. Posenacaftor (PTI-801) exhibits no potentiator activity on low temperature- and corrector-rescued p.Phe508del-CFTR, displays similar behavior to that of VX-445 on genetic revertants.
Physicochemical Properties
M.Wt
467.50
Formula
C27H26NNaO5
Appearance
Solid
CAS No.
Storage
Solide Powder
-20°C 12 Months; 4°C 6 Months
In Solvent
-80°C 6 Months; -20°C 6 Months
Shipping
Solubility
10 mM in DMSO
Chemical Name/SMILES
4-Quinolinecarboxylic acid, 8-methyl-2-(3-methyl-2-benzofuranyl)-5-[(1R)-1-(tetrahydro-2H-pyran-4-yl)ethoxy]-, sodium salt (1:1)
References
1. Filipa C Ferreira, et al. Eur J Pharmacol. 2024 Mar 15:967:176390.
