PTC Therapeutics, Inc. Announces $15.4 Million National Institutes of Health (NIH) Research Grant for Duchenne Muscular Dystrophy
SOUTH PLAINFIELD, N.J., July 10 /PRNewswire-USNewswire/ -- PTC 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 National Institute of Neurological Disorders and Stroke and the National Institute of Arthritis and Musculoskeletal and Skin Diseases of the National Institutes of Health (NIH) have awarded a $15.4 million, five-year U54 grant to fund further research into Duchenne muscular dystrophy (DMD). The grant award will support a research collaboration between PTC and the University of Pennsylvania School of Medicine and will build upon previous research and discovery work supported by Parent Project Muscular Dystrophy (PPMD) under an initiative known as Project Catalyst.
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"This grant supports promising preclinical research on a number of targets believed to be medically relevant for DMD," said H. Lee Sweeney, Ph.D., Professor and Chairman of the Department of Physiology at the University of Pennsylvania School of Medicine, Scientific Advisor to PPMD and principal investigator on this NIH grant. "Over the course of this grant, our goal is to have Project Catalyst compounds ready for advancement into clinical trials for patients with DMD." Ellen Welch, Ph.D., Group Leader, Genetic Disorders at PTC and principal investigator for PTC on this grant, commented, "We are very excited about this grant award as it expands our ability to advance promising programs that we have been developing over the past several years."
In 2003, PTC and PPMD initiated Project Catalyst, a research collaboration funded by PPMD to identify new treatments for patients with DMD. Project Catalyst leveraged PTC's proprietary drug discovery technology called GEMS (Gene Expression Modulation by Small-molecules) to search for new potential drugs for DMD patients. The GEMS technology allows PTC to identify small molecules that up- or down-regulate the production of proteins. Utilizing the GEMS technology, promising lead compounds were identified for several targets believed to be medically relevant for DMD.
"We are extremely pleased that the initial progress of Project Catalyst will be extended through this NIH grant award," stated Pat Furlong, Executive Director and Founder of Parent Project Muscular Dystrophy. "PTC and UPenn have demonstrated a long-standing commitment to research and clinical development for DMD and have made remarkable progress to date. The support of the NIH for Project Catalyst's efforts offers great hope to all members of the DMD community."
"This grant is a model of the type of translational research we seek to support," said John D. Porter, Ph.D., Program Director, Neurogenetics Cluster and Technology Development Program, NIH National Institutes of Neurological Disorders and Stroke. "The strength of this collaboration is that it brings together advocacy groups, academia, industry, and government in order to make an impact on the treatment of rare disorders such as DMD."
"We are honored to receive this award from the NIH in conjunction with our colleagues at UPenn," stated Stuart W. Peltz, Ph.D., President and Chief Executive Officer of PTC Therapeutics. "These funds help support our commitment to developing multiple treatments for neuromuscular disorders. With PTC124 in Phase 2 trials for nonsense-mediated DMD, we are establishing the clinical and regulatory path for new treatments for DMD in which the Project Catalyst compounds can follow. Our goal is to help provide treatment options for all DMD patients."
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 lethal genetic 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 DMD is available through the Muscular Dystrophy Association (www.mdausa.org ) and the Parent Project Muscular Dystrophy (www.parentprojectmd.org ).
About Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophy (PPMD) is the largest nonprofit organization in the United States focused entirely on Duchenne muscular dystrophy. A small group of parents founded PPMD in 1994, and it has progressed into the world's leading Duchenne muscular dystrophy organization. PPMD's expertise is unparalleled, and they hold the highest ethical standards. They are the only organization taking a comprehensive approach in the fight against Duchenne-raising awareness, funding research, promoting advocacy, connecting the community, and broadening treatment options--and their efforts will ultimately lead to a cure.
About University of Pennsylvania School of Medicine
PENN Medicine is a $2.9 billion enterprise dedicated to the related missions of medical education, biomedical research, and high-quality patient care. PENN Medicine consists of the University of Pennsylvania School of Medicine (founded in 1765 as the nation's first medical school) and the University of Pennsylvania Health System.
Penn's School of Medicine is ranked #2 in the nation for receipt of NIH research funds; and ranked #3 in the nation in U.S. News & World Report's most recent ranking of top research-oriented medical schools. Supporting 1,400 full-time faculty and 700 students, the School of Medicine is recognized worldwide for its superior education and training of the next generation of physician-scientists and leaders of academic medicine.
About the National Institute of Neurological Disorders and Stroke
The mission of National Institute of Neurological Disorders and Stroke (NINDS) is to reduce the burden of neurological disease -- a burden borne by every age group, by every segment of society, by people all over the world.
The project described in this press release was supported by Grant Number U54NS058572 from the National Institute of Neurological Disorders and Stroke. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NINDS or the National Institute of Health.
About PTC Therapeutics, Inc.
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, CV Therapeutics and Schering-Plough.
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