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PTC Therapeutics Announces Pharmacokinetic and Safety Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy
Date:10/11/2007

- Data Presented at 36th Child Neurology Society Annual Meeting -

SOUTH PLAINFIELD, N.J., Oct. 11 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development and commercialization of small-molecule drugs targeting post-transcriptional control mechanisms, today announced pharmacokinetic and safety data from a Phase 2 clinical trial of PTC124 in patients with Duchenne muscular dystrophy (DMD) due to a nonsense mutation. The results, which include data from the third and final cohort of the study, show that treatment with PTC124 appeared well tolerated at all three dose levels and target plasma concentrations were achieved at the mid- and high-dose levels. These data were presented today at the 36th Annual Meeting of the Child Neurology Society (CNS) in Quebec City, Canada.

(Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )

In the study, patients received 28 days of PTC124 treatment at one of three dose levels. All clinical trial participants are boys with a nonsense mutation in the dystrophin gene, substantially elevated serum creatine kinase levels, and symptoms associated with DMD. The analysis presented today showed that PTC124 appeared well tolerated among the 38 boys included in the study. Adverse events were infrequent, mild to moderate in severity, and did not result in therapy interruptions or discontinuations. There were no concerns based on physical examinations, vital sign measurements, electrocardiograms or laboratory parameters. Compliance with PTC124 treatment was excellent at all three dose levels. Target plasma concentrations associated with activity in a preclinical model of DMD were achieved at the mid- and high-dose levels.

"DMD is a disorder with a significant need for better treatment options and we are encouraged by the results we have seen to date with PTC124," said Brend
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SOURCE PTC Therapeutics, Inc.
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