PTC Therapeutics Receives $1.5 Million Grant from Muscular Dystrophy Association

Main Category: Muscular Dystrophy / ALS
Article Date: 21 Apr 2005 - 14:00 PDT

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Therapeutics, Inc (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of small-molecule drugs targeting post-transcriptional control mechanisms, today announced that the company has been awarded a $1.5 million grant from the Muscular Dystrophy Association (MDA). The award will be utilized to fund the development of PTC124 for the treatment of Duchenne muscular dystrophy (DMD) due to a nonsense mutation in the dystrophin gene. It is estimated that 15% of the cases of DMD 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. Single-dose and multiple-dose Phase 1 studies involving healthy volunteers have been recently completed and PTC is working with investigators and the FDA to finalize plans for Phase 2 studies in DMD and cystic fibrosis.

"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. We commend PTC for their use of innovative treatment approaches for genetic diseases like Duchenne muscular dystrophy," commented MDA President and CEO Robert Ross.

"We are honored to receive this grant from the MDA and appreciate their continued support of PTC124," said Stuart W. Peltz, Ph.D., President and CEO of PTC. "The funding by the MDA is fundamental to the development of PTC124. The MDA is among the most active associations 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 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 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. Phase 2 studies in patients with Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF) are planned for 2005.

It is estimated that 10% of the cases of cystic fibrosis (CF) and 15% of the cases of Duchenne muscular dystrophy (DMD) are due to nonsense mutations. PTC has catalogued over 1,800 distinct genetic disorders where 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, epidermolysis bullosa, and lysosomal storage disorders. In addition to the MDA grant, Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) has awarded PTC Therapeutics, Inc. a grant of $1.7 million for the development of PTC124 for the treatment of CF due to a nonsense mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. The FDA has granted PTC124 fast track designation for the treatment of CF and orphan drug designations for the treatment of CF and DMD due to nonsense mutations.

About the Muscular Dystrophy Association (MDA) MDA is a voluntary health agency dedicated to helping the more than 1 million Americans affected by neuromuscular disorders through programs of worldwide research, comprehensive services, and far-reaching professional and public health education. For more information please visit http://www.mdausa.org.

About Duchenne muscular dystrophy (DMD)

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 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 regarding Duchenne muscular dystrophy is available through the Muscular Dystrophy Association (http://www.mdausa.org).

PTC Therapeutics, Inc.
http://www.ptcbio.com
http://www.mdausa.org