PTC124 shows activity in cystic fibrosis; Phase 2 proof-of-concept data published in Lancet

1 of the first therapies to address the underlying cause of cystic fibrosis; oral drug may represent new treatment paradigm SOUTH PLAINFIELD, NJ – August 20, 2008 – New phase 2 data published today in The Lancet show that the investigational oral drug PTC124 demonstrates activity in nonsense-mutation cystic fibrosis (CF). The data show that treatment with PTC124 results in statistically significant improvements in the chloride channel function of patients with nonsense-mutation CF. The study was conducted at the Hadassah Hebrew University Hospital in Jerusalem, Israel and sponsored by PTC Therapeutics (PTC). Patients with CF lack adequate levels of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, a chloride channel which is required for normal function of the lung, pancreas, liver, and other organs.... [read full story]