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PTC Therapeutics Completes Enrollment of Phase 3 Trial of Ataluren in Patients with Cystic Fibrosis
Date:12/21/2010

cally experience more severe disease symptoms than those with lower-risk genotypes, including a greater than twofold increased risk of death, a higher probability of end-stage lung disease and a higher prevalence of pancreatic insufficiency. A simple genetic test can determine if a patient's disease is caused by a nonsense mutation.

"We are pleased to have completed enrollment of our second pivotal clinical trial of ataluren in patients with a nonsense mutation genetic disorder.  This is a tremendous achievement and a testament to the commitment of clinical trial patients and their families, as well as study investigators and trial site staff," stated Stuart Peltz, Ph.D., president and CEO of PTC Therapeutics.  "PTC is committed to improving the quality of life of patients with serious and life-threatening diseases through our innovative scientific approach to the discovery of novel treatments."

PHASE 3 STUDY DESIGN

The primary objective of the registration-directed, double-blind, placebo-controlled study is to determine whether ataluren can improve lung function in patients with nmCF, as measured by forced expiratory volume in 1 second (FEV1). Additional secondary endpoints are evaluating other aspects of patient function, drug activity, and safety.  The 48-week trial enrolled 238 patients, ages six years and older, at multiple sites in North America, Europe, and Israel.  Patients were randomly assigned to one of two treatment arms: ataluren (10 mg/kg morning, 10 mg/kg midday, 20 mg/kg evening) or placebo (morning, midday, evening).

ABOUT ATALUREN

An investigational new drug discovered by PTC Therapeutics, ataluren is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.  A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. 
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SOURCE PTC Therapeutics, Inc.
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