pti-428 + ivacaftor

PTI-428 + ivacaftor is an experimental oral combination therapy for cystic fibrosis consisting of **PTI-428**, a CFTR amplifier developed by Proteostasis Therapeutics, and **ivacaftor**, a CFTR potentiator developed by Vertex Pharmaceuticals. PTI-428 works by amplifying the production of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, while ivacaftor increases the activity of CFTR channels at the cell surface, improving chloride ion transport. This combination has been studied in patients with cystic fibrosis, including those with the F508del mutation, to assess whether PTI-428 can further enhance the therapeutic effect of existing CFTR modulators like ivacaftor, providing additive or synergistic improvements in lung function and other disease parameters[1][2][3][7].