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PTC Announces Data Showing That PTC124 Causes Statistically Significant Improvements in Chloride Channel Function in Cystic Fibrosis Patients
Date:10/24/2008

ned to alleviate the symptoms of the disease. These treatments include chest physical therapy to clear the thick mucus from the lungs, antibiotics to treat lung infections, and a mucus-thinning drug designed to reduce the number of lung infections and improve lung function. In addition, the majority of cystic fibrosis patients take pancreatic enzyme supplements to assist with food absorption in digestion. There is a significant unmet medical need for treatments that address the underlying cause of CF. More information regarding CF is available through the Cystic Fibrosis Foundation (http://www.cff.org).

About PTC124

PTC124 is an orally delivered, investigational new drug discovered by PTC Therapeutics. The drug is being developed for the treatment of nonsense mutation genetic disorders. Nonsense mutations are single-point alterations in the genetic code that prematurely stop the mRNA translation process, leading to production of truncated, non-functional proteins. PTC124 induces the cellular translation machinery to read through nonsense mutations in mRNA, inducing production of full-length, functional proteins. PTC124 has demonstrated proof of concept in Phase 2a clinical trials. Across all clinical studies to date, PTC124 has been generally well tolerated. PTC124 is currently in Phase 2b development with the goal of demonstrating that increasing functional protein levels in patients with nonsense mutation genetic disorders will safely provide clinical benefits.

PTC124 has been granted orphan drug status by the FDA and the European Commission for the treatment of cystic fibrosis and Duchenne muscular dystrophy due to nonsense mutations. The FDA has also granted PTC124 Subpart E designation for expedited development, evaluation, and marketing.

PTC has an exclusive collaboration with Genzyme Corporation to develop and commercialize PTC124 outside the United States and Canada. The development o
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SOURCE PTC Therapeutics, Inc.
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