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PTC Therapeutics Announces Data from Additional Clinical Studies of PTC124 in Cystic Fibrosis Confirming Activity
Date:6/12/2008

measurement is particularly notable given that cough is one of the major symptomatic manifestations of the underlying disease process in CF. The reduction in cough achieved with PTC124 in this three-month study suggests secondary clinical effects of drug activity. Based on these findings, we believe that objective measurement of cough may offer a meaningful new way to evaluate drug efficacy in future CF clinical trials."

In a separate Phase 2a study in Europe, data are currently available from 21 children who received two 14-day treatment courses of oral PTC124 therapy at two different dose levels. Patients ranged in age from six to 18 years. All had nonsense-mutation-mediated disease, pathological lung infection, and pancreatic insufficiency. Statistically significant (p< 0.05) increases in the proportion of epithelial cells showing surface staining with the CFTR protein were observed. In addition, statistically significant (p< 0.05) improvements in CFTR-mediated chloride conductance as measured by TEPD were evident. PTC124 was generally well tolerated in pediatric patients and mean compliance with treatment was excellent ( >95%).

"We are encouraged to see the activity of PTC124 observed in adults reproduced in a pediatric population in France and Belgium," commented Dr. Sermet-Gaudelus. "PTC124 causes the missing protein to be made, to be located in the right place in the cell, and to have functional effect. Combined with the generally well-tolerated profile of PTC124, we believe these data support inclusion of pediatric patients in future clinical trials."

"We are gratified by these results from our Israeli, French, and Belgian investigators, which demonstrated the activity of PTC124 in patients across a variety of age ranges, geographies, and nonsense mutation types," stated Langdon Miller, M.D., Chief Medical Officer of PTC. "These data offer the basis for initiating a randomized, controlled Phase 2b study later this year to evaluate the cl
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SOURCE PTC Therapeutics, Inc.
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