Muscular Dystrophy Association Awards
$1.5 Million Grant to PTC Therapeutics

The Muscular Dystrophy Association (MDA) today announced a $1.5 million award to PTC Therapeutics Inc. (PTC), a biopharmaceutical company focused on the discovery, development and commercialization of small-molecule drugs targeting post-transcriptional control mechanisms. The award will fund the development of PTC124 for the treatment of Duchenne muscular dystrophy (DMD) due to a nonsense mutation in the dystrophin gene. It’s estimated that 15 percent of DMD cases are due to a nonsense mutation.

PTC124 represents a first-in-class, 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 halt the translation process, producing a shortened, non-functional protein. PTC124 allows the cellular machinery to bypass the nonsense mutation and continue the translation process, restoring the production of full-length, functional proteins. PTC124 is currently being evaluated in Phase 1 clinical trials involving healthy volunteers.

“This award through MDA’s Translational Research Program represents a major commitment by the Association to partner with industry in the development of new drugs,” MDA President and CEO Bob Ross said.

“We are honored to receive this grant from the MDA and appreciate their continued support of PTC124,” PTC President and CEO Stuart W. Peltz said. “The funding by MDA is fundamental to the development of PTC124. The MDA is the most active association in the United States supporting research from scientists across multiple disciplines in an effort to identify new treatments for neuromuscular diseases. We feel privileged to work with the MDA, and share their commitment to finding a treatment for DMD.”

ABOUT PTC THERAPEUTICS INC.

PTC is a biopharmaceutical company focused on the discovery, development and commercialization of small-molecule drugs targeting post-transcriptional control mechanisms. Post-transcriptional control processes are the sequence of events in the cell that ultimately regulate the rate and timing of all protein production. PTC’s compounds alter these processes by selectively modulating how RNA is used to produce proteins. By applying this approach, PTC has advanced its drug discovery programs rapidly from targets to preclinical and clinical drug candidates, building a robust pipeline across genetic disorders, oncology and infectious diseases.

ABOUT PTC124

PTC124 has demonstrated the ability to restore full-length functional protein in genetic disease models harboring nonsense mutations. PTC124 represents a unique opportunity to use a single small-molecule drug to address chronic and life-threatening diseases of high unmet medical need. It’s estimated that 10 percent of the cases of cystic fibrosis (CF) and 15 percent of the cases of Duchenne muscular dystrophy are due to nonsense mutations. PTC has catalogued over 1,800 distinct genetic disorders in which nonsense mutations are the cause of the disease in an appreciable percentage of patients. In addition to CF and DMD, other potential indications under consideration for PTC124 include hemophilia, neurofibromatosis, retinitis pigmentosa, bullous skin diseases and lysosomal storage disorders.

ABOUT THE MUSCULAR DYSTROPHY ASSOCIATION

MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education.

ABOUT DUCHENNE MUSCULAR DYSTROPHY

Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common inherited lethal disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).

More information about DMD and MDA is available at www.mda.org, while information about PTC Therapeutics and PTC124 can be found at www.ptcbio.com.