- Preliminary Data to be Presented at Parent Project UK Muscular Dystrophy
Conference -
SOUTH PLAINFIELD, N.J., Oct. 19 /PRNewswire/ -- PTC Therapeutics, Inc.
(PTC), a biopharmaceutical company focused on the discovery and development
of small-molecule drugs targeting post-transcriptional control mechanisms,
today announced it has been awarded a two-year grant from the Office of
Orphan Products Development (OOPD) Grant Program of the U.S. Food and Drug
Administration (FDA). The grant will support the patient-related costs of
the company's ongoing U.S.-based Phase 2 clinical trial of PTC124 for the
treatment of nonsense-mutation-mediated Duchenne muscular dystrophy (DMD).
(Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )
"It is an honor to receive this grant from the FDA in support of our
clinical development efforts," stated Stuart Peltz, Ph.D., President and
CEO of PTC Therapeutics. "Last year, PTC was awarded a grant from the FDA
for the development of PTC124 as a potential treatment for cystic fibrosis
(CF), which we have used to advance our Phase 2 CF program. Similarly, this
new award will support the development of PTC124 as a potential treatment
for DMD."
PTC initiated the Phase 2 DMD clinical trial of PTC124 in January 2006.
Patients with DMD lack dystrophin, a protein that is critical to the
structural stability of muscle fibers. This Phase 2 multi-site, open-label,
dose-ranging clinical trial is evaluating muscle dystrophin expression in
patients with nonsense-mutation-mediated DMD. Blood levels of
muscle-derived creatine kinase are being measured as assessments of muscle
integrity. PTC124 safety, compliance, and pharmacokinetics are also being
evaluated.
Preliminary data from this study will be presented at the Parent
Project UK Muscular Dystrophy 4th International DMD conference on Saturday,
October 21st by Dr. Richard Finkel, Director of the Neuromuscular Program
at The Children's Hospital of Philadelphia and one of the lead
investigators of the PTC DMD trial. The conference will be held at the
Institute of Education in London, England, October 21st and 22nd, 2006.
About The Office of Orphan Products Development
The Office of Orphan Products Development (OOPD) has been dedicated to
promoting the development of products that demonstrate promise for the
diagnosis and/or treatment of rare diseases or conditions since it was
created in 1982. OOPD interacts with the medical and research communities,
professional organizations, academia, and the pharmaceutical industry, as
well as rare disease groups. The OOPD administers the major provisions of
the Orphan Drug Act (ODA) which provide incentives for sponsors to develop
products for rare diseases. The ODA has been very successful -- more than
200 drugs and biological products for rare diseases have been brought to
market since 1983. In contrast, the decade prior to 1983 saw fewer than ten
such products come to market. In addition, the OOPD administers the Orphan
Products Grants Program which provides funding for clinical research in
rare diseases.
About PTC124
PTC124 is an orally delivered investigational product candidate in
Phase 2 clinical development 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 has demonstrated activity in
preclinical genetic disease models harboring nonsense mutations allowing
the restoration of the production of full-length, functional proteins. In
Phase 1 clinical trials, PTC124 was generally well tolerated, achieved
target plasma concentrations that have been associated with activity in
preclinical models, and did not induce ribosomal readthrough of normal stop
codons. PTC is currently conducting Phase 2 clinical trials of PTC124 in
nonsense-mutation- mediated cystic fibrosis (CF) and Duchenne muscular
dystrophy (DMD).
It is estimated that 10% of the cases of CF and 13% of the cases of DMD
are due to nonsense mutations. PTC believes that PTC124 is potentially
applicable to a broad range of other genetic disorders in which a nonsense
mutation is the cause of the disease. The FDA has granted PTC124 Fast-Track
designations and Orphan Drug designations for the treatment of CF and DMD
due to nonsense mutations. PTC124 has also been granted orphan drug status
for the treatment of DMD and CF by the Committee for Orphan Medicinal
Products (COMP) of the European Medicines Agency (EMEA). PTC124's
development is supported by grants from the Muscular Dystrophy Association
(MDA), Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), FDA's Office
of Orphan Products Development (OOPD), and by General Clinical Research
Center grants from the National Center for Research Resources (NCRR).
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 and the Parent Project Muscular
Dystrophy.
About PTC Therapeutics, Inc.
PTC is a biopharmaceutical company focused on the discovery and
development 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 has assembled
proprietary technologies and extensive knowledge of post-transcriptional
control processes that it applies in its drug discovery and development
activities. PTC's current pipeline of clinical and preclinical product
candidates addresses multiple indications, including genetic disorders,
oncology, and infectious diseases.
SOURCE PTC Therapeutics, Inc.
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Related links:
http://www.ptcbio.com
Photo Notes:http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO
CONTACT:
Jane Baj of PTC Therapeutics, Inc.,
+1-908-222-7000, x167, jbaj@ptcbio.com; or Sheryl Seapy of Pure
Communications, +1-949-608-0841,
sheryl@purecommunicationsinc.com; or Patients, Patients'
Families, Investigators and Patient Organizations - Kerri
Donnelly of PTC Therapeutics, Inc., +1-908-222-7000, x112,
kdonnelly@ptcbio.com
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