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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

tudy (p<0.01). PTC124 was generally well tolerated in this study, resulting in excellent compliance (>95%).

In a separate presentation, Dr. Kerem provided a review of all PTC124 studies to date, including studies in patients with Duchenne muscular dystrophy, a genetic disorder that can also be caused by a nonsense mutation.

"We are enthusiastic about the growing body of data on PTC124, and this study specifically, which suggests the benefits of PTC124 treatment may increase over time," said Dr. Kerem. "The safety profile of the 12-week administration of PTC124 in patients with CF supports further investigations in long-term clinical trials."

"We are pleased to see PTC124 highlighted in so many presentations at NACFC," said Langdon Miller, M.D., Chief Medical Officer of PTC. "These data demonstrate the activity of PTC124 across a broad range of patients and suggest that it could be a valuable option for many patients living with CF. There is currently no available therapy to correct defective CFTR production and function. Instead, available treatments for CF are designed to alleviate the symptoms of the disease. We hope that PTC124 will be among the first treatments that addresses the underlying cause of the disease."

About Cystic Fibrosis

Cystic fibrosis (CF) is a life-threatening genetic disease that causes serious lung infections and digestive complications. According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and children in the United States and nearly 70,000 people worldwide. There is a commercially available genetic test to determine if a patient's CF is caused by a nonsense mutation, and it is estimated that nonsense mutations are the cause of CF in approximately 10 percent of patients in the United States and Europe and over 50 percent of patients in Israel. There is currently no available therapy to correct defective CFTR production and function. Instead, available treatments for CF are desig
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SOURCE PTC Therapeutics, Inc.
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