Grant Extends Research Originally Supported by Parent Project 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.
(Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )
"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 (http://www.mdausa.org ) and the Parent Project
Muscular Dystrophy (http://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.
SOURCE PTC Therapeutics, Inc.
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Related links:
http://www.ptcbio.com
http://www.mdausa.org http://www.parentprojectmd.org
Photo Notes:
NewsCom: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO
AP Archive: http://photoarchive.ap.org
PRN Photo Desk, photodesk@prnewswire.com
CONTACT:
Jane Baj of PTC Therapeutics, Inc.,
+1-908-222-7000 x167, jbaj@ptcbio.com; Sheryl Seapy of Pure
Communications, +1-949-608-0841,
sheryl@purecommunicationsinc.com, for PTC Therapeutics, Inc.
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