PTC Therapeutics has initiated a long-term Phase 3 clinical trial of ataluren (formerly referred to as PTC124®) in patients with nonsense mutation cystic fibrosis (nmCF). The trial is a multi-center, double-blind, placebo-controlled trial. The main goals of the trial are to understand whether ataluren can improve lung function in patients with nmCF and whether the drug can be given safely for a long period of time. The study also evaluates whether ataluren can decrease nmCF-related exacerbations (episodic increases in the severity of CF symptoms) and improve patient quality of life.

The study will enroll about 208 patients with nmCF at CF research centers in North America, Europe, and Israel. Each participant will be randomly assigned to one of two treatment groups. One treatment group will receive ataluren and the other will receive a placebo (a substance that looks and tastes like ataluren but does not actually contain the drug). Both groups will take the assigned study medication (ataluren or placebo) three times a day – morning, midday, and evening – for 48 weeks. Participants who successfully complete the trial will be eligible to enter into an extension study lasting at least another 48 weeks, in which every participant will receive ataluren.

Requirements for Taking Part in this Study
To be eligible for this trial, patients must have had a DNA blood test, or genetic test, to evaluate the CFTR gene and determine that a nonsense mutation is present. Patients must also meet the following criteria:

• Be at least 6 years of age
• Weigh at least 16 kg (about 35 pounds)
• Be able to complete a breathing test (spirometry) with values that are between 40% and 90% of those predicted for healthy people of similar age and height
• Be free of serious abnormalities of liver, kidney, or adrenal function or life-threatening complications of CF or other diseases
• Not be pregnant

Other eligibility requirements will be explained at the trial site.

• Trial sites
• Frequently Asked Questions
• ClinicalTrials.gov overview
• Ataluren overview
• Genetic Testing

Additional details about this ataluren clinical trial can be found at www.clinicaltrials.gov or by contacting PTC Therapeutics Patient and Professional Advocacy at
(866) 282-5873 or (908) 912-9256 or PatientInfo@ptcbio.com.