GLPG2451

GLPG2451 is an investigational small molecule drug developed as a potentiator of the cystic fibrosis transmembrane conductance regulator (CFTR) protein for the treatment of cystic fibrosis. It enhances the activity of defective CFTR channels at the cell surface, improving chloride ion transport and potentially alleviating symptoms associated with cystic fibrosis. The drug was co-developed by Galapagos and AbbVie as part of a strategy to create combination therapies targeting multiple aspects of CFTR dysfunction. In preclinical studies, GLPG2451 demonstrated significant activity in primary cells from patients with defective CFTR and was metabolized to an active component called M31 with similar efficacy. Clinical development included Phase 1 trials evaluating safety, tolerability, and pharmacokinetics in healthy volunteers[1][3][4][5][7].