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PTC Therapeutics Announces Publication of Preclinical Data in PNAS
Date:2/4/2008

Data Show PTC124 Addresses Underlying Cause of Genetic Disorders and

Restores Protein Function in Cystic Fibrosis Model

SOUTH PLAINFIELD, N.J., Feb. 4 /PRNewswire/ -- PTC Therapeutics, Inc. today announced the publication of new preclinical data in the February 12, 2008 edition of the Proceedings of the National Academy of Sciences (PNAS) which show that PTC124, a novel drug designed to bypass nonsense mutations, was active in a preclinical model of cystic fibrosis (CF). These results support and add to research published last year in the journal Nature, which demonstrated the activity of PTC124 in a preclinical model of Duchenne muscular dystrophy (DMD). PTC124 has demonstrated pharmacodynamic proof of concept in Phase 2a clinical trials in nonsense-mutation-mediated CF and DMD.

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

PTC has catalogued over 2,400 distinct genetic disorders where nonsense mutations are the cause of the disease in a significant percentage of patients. Nonsense mutations inactivate gene function and are known to cause anywhere from five to 70 percent of the individual cases of most inherited diseases, such as cystic fibrosis (10%) and Hurler's syndrome (70%).

"The preclinical and clinical data on PTC124 support our hope that this drug will be an important disease-modifying therapy for cystic fibrosis," said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. "This is an exciting potential new treatment for patients afflicted with nonsense-mutation-mediated CF. We look forward to the next stage of clinical development to demonstrate the benefits of this promising new investigational drug."

As with the DMD data published in Nature, the results published in PNAS further demonstrate that PTC124 targets genetic mutations in a completely new way. PTC124 functions by overcomi
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SOURCE PTC Therapeutics, Inc.
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