Proteostasis Therapeutics (NASDAQ: PTI) dosed the first patient in a 28-day Phase 2 trial evaluating combinations of PTI-801, PTI-808 and PTI-428 for the treatment of cystic fibrosis (CF).
CF is caused by a mutation in cystic fibrosis transmembrane conductance regulator (CFTR) gene, which impairs epithelial fluid transport in the lungs.
PTI-801 is a third-generation CFTR corrector and PTI-808 is a novel potentiator. PTI-428 is designed to amplify CFTR.
The trial will assess the efficacy of Proteostasis’ doublet (PTI-808 and PTI-801) and triplet (PTI-808, PTI-801 and PTI-428) combinations in up to 60 CF patients.
“Our next stage in development will target the recruitment of patients comparable to those used in other CFTR modulator combination studies which, together with optimal dose levels and longer treatment duration, could potentially further enhance the magnitude of pulmonary benefit,” Dr. Geoffrey Gilmartin, Proteostasis’ CMO, said in a statement.
Data from the Phase 2 study are expected in the first quarter of 2020.
