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 /EMWNews/ -- 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.



    



    "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 ( http://www.cff.org ).



    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 http://www.cff.org



    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, http://www.ptcbio.com