PTC PRESENTS DATA FOR CYSTIC FIBROSIS DRUG CANDIDATE

PTC Therapeutics announced that complete data from the Phase I single- and multiple-dose studies and design of the planned Phase II studies for PTC124 were presented at the 28th Annual European Cystic Fibrosis Conference in Crete, Greece.

PTC124 is a novel, orally administered drug that targets nonsense mutations and is being investigated initially as a treatment for cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD), with the potential to treat a number of other genetic disorders. It is estimated that 10 percent of CF patients and 15 percent of DMD patients have these diseases as a consequence of nonsense mutations.

The results of the single- and multiple-dose studies in healthy volunteers confirmed that PTC124 is generally well-tolerated and achieves target plasma concentrations that have been associated with activity in preclinical models. To demonstrate specificity of PTC124 effects, special tests were performed that revealed no evidence of undesirable, nonspecific readthrough of normal stop codons. Pharmacokinetic modeling of the Phase I results has allowed development of a dosing regimen for the planned Phase II studies in CF and DMD.