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PTC Therapeutics Announces $25 Million Award From Cystic Fibrosis Foundation Therapeutics for Development of PTC124

- Expanded Collaboration Supports Pivotal Phase 2b Clinical Trials of First Potential Therapy to Address an Underlying Cause of Cystic Fibrosis -

SOUTH PLAINFIELD, N.J. and BETHESDA, Md., July 16 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) and Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit affiliate of the Cystic Fibrosis Foundation, today announced the expansion of an existing collaboration to support development of PTC124, the PTC's new investigational oral drug for the treatment of cystic fibrosis (CF) caused by nonsense mutations. PTC will receive up to $25 million from CFFT in support of key Phase 2b clinical trials for PTC124 in CF.

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"We are pleased to expand our collaboration with PTC Therapeutics on the development of the therapy PTC124," said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. "The encouraging data from the Phase 2a studies indicate that PTC may become a very important drug for the treatment of CF in the 10 percent of patients who carry nonsense mutations. These exciting results lead us to believe PTC124 has the potential to be one of the first oral drugs on the market to address the basic defect in cystic fibrosis."

"We are honored both to receive this award and to continue our productive collaboration with the Foundation," stated Stuart W. Peltz, Ph.D., president and chief executive officer of PTC Therapeutics. "The significant progress that we have made with PTC124 is a result of our unique drug discovery approach as well as the extraordinary support that we have received from the physician, patient, advocacy and regulatory communities. We look forward to initiating registration-directed studies of PTC124 for nonsense-mutation- mediated CF within the next few months."

In June 2008, PTC announced encouraging new data from recently completed Phase 2a studies in adult and pediatric CF patients at the European Cystic Fibrosis Conference. Results from these studies demonstrated statistically significant improvements in the primary outcome measure-nasal potential difference (NPD)-across a variety of age ranges, geographies, and nonsense mutation types. In the three-month, Phase 2a extension study of adult patients, results demonstrated a statistically significant mean decrease [28 percent] in the frequency of cough, one of the most prominent and burdensome CF-related symptoms.

About Cystic Fibrosis

Cystic fibrosis (CF) is a life-threatening genetic disease that causes serious lung infections and digestive complications. According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and children in the United States and nearly 70,000 people worldwide. CF occurs in approximately one of every 3,500 live births, with approximately 1,000 new cases diagnosed each year in the United States. There is a commercially available genetic test to determine if a patient's CF is caused by a nonsense mutation, and it is estimated that nonsense mutations are the cause of CF in approximately 10 percent of patients in the United States. There is currently no available therapy to correct defective CFTR production and function. Instead, available treatments for CF are designed to alleviate the symptoms of the disease. These treatments include chest physical therapy to clear the thick mucus from the lungs, antibiotics to treat lung infections and a mucus-thinning drug designed to reduce the number of lung infections and improve lung function. In addition, the majority of CF patients take pancreatic enzyme supplements to assist with food absorption in digestion. There is a significant unmet medical need for treatments for the underlying cause of CF. More information regarding CF is available through the Cystic Fibrosis Foundation ( ).

About PTC124

PTC124 is an orally delivered investigational new drug for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely stop the translation process, preventing production of a functional protein. In Phase 2a clinical trials in nonsense-mutation-mediated cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD), PTC124 has demonstrated the ability to produce protein across a variety of nonsense mutation types. Across all clinical studies to date, PTC124 has been generally well tolerated and has achieved target plasma concentrations that have been associated with activity in preclinical models. PTC124 is currently in Phase 2b development in DMD with the goal of demonstrating that increasing functional protein levels in patients with nonsense-mutation-mediated genetic disorders may provide clinical benefits.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation, the leading organization focused on curing and controlling cystic fibrosis, has invested nearly $300 million in drug research with biotech companies since 1998 to develop therapies to fight CF. As a result, the Foundation has built a drug pipeline with more than 30 promising therapies in development. Virtually all the approved CF therapies available today were made possible because of the support of the Foundation. Based in Bethesda, MD, the Foundation has 80 chapters and branch offices, and supports and accredits a nationwide network of 115 CF care centers that provide treatment and vital resources to patients and families. For more information visit

About PTC Therapeutics Inc.

PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary, small-molecule drugs that target post-transcriptional control processes. Post- transcriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. PTC's internally-discovered pipeline addresses multiple therapeutic areas, including genetic disorders, oncology and infectious diseases. In addition, PTC has developed proprietary technologies and extensive knowledge of post- transcriptional control processes that it applies in its drug discovery and development activities, including the Gene Expression Modulation by Small- molecules (GEMS) technology platform, which has been the basis for collaborations with leading pharmaceutical and biotechnology companies such as Pfizer, Celgene, CV Therapeutics and Schering-Plough. For more information, visit PTC's website,

SOURCE PTC Therapeutics, Inc.
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