PTC Therapeutics of the USA says that it has initiated a Phase Iclinical trial of PTC124, a small-molecule drug which is initially under evaluation for the treatment of cystic fibrosis and Duchenne muscular dystrophy.
The Phase I study will evaluate the safety, tolerability and pharmacokinetic profile of PTC124 in healthy volunteers. In preclinical studies, the agent was found to be orally-available and well-tolerated, noted the firm.
PTC124 targets nonsense mutations, with 15% of the cases of DMD and 10% of the cases for CF being due to these single-point alterations in the DNA.
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