SOUTH PLAINFIELD, N.J., March 30 /PRNewswire/ -- PTC Therapeutics, Inc.
(PTC), a biopharmaceutical company focused on the discovery, development, and
commercialization of small-molecule drugs that target post-transcriptional
control processes, today announced that the company has been granted Fast
Track designation from the United States Food and Drug Administration (FDA)
for the development of PTC124 for the treatment of Duchenne muscular dystrophy
(DMD) due to a nonsense mutation in the dystrophin gene. In December 2004,
PTC124 was granted Orphan Drug designation by the FDA for the treatment of
DMD. PTC124 is currently in Phase 2 clinical trials in DMD and cystic
fibrosis (CF) in cases in which a nonsense mutation is the cause of the
disease. PTC expects to complete these Phase 2 clinical trials in the second
half of 2006.
(LOGO: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )
The Fast Track program is designed to facilitate the development and
expedite the review of new drugs that are intended to treat serious or
life-threatening conditions and that demonstrate the potential to address
unmet medical needs. Development programs receiving Fast Track designations
typically receive FDA priority review (6-month vs. standard 10-month review).
"Fast Track designation in DMD is an additional important element in the
development of PTC124," said Stuart W. Peltz, Ph.D., President and CEO of PTC.
"DMD is an unmet medical need where only palliative options are currently
available. We hope PTC124 will represent a therapeutic option for patients
with DMD due to a nonsense mutation."
About 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 are of central importance to the regulation of the rate and
timing of cellular protein production. 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.
About PTC124
PTC124 is an orally delivered investigational product candidate in
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. In pre-clinical trials, the administration of PTC124
allowed the restoration of the production of full-length, functional proteins.
PTC124 has demonstrated activity in preclinical genetic disease models
harboring nonsense mutations. 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. Pharmacokinetic modeling of the Phase 1
results allowed development of a dosing regimen for the Phase 2 studies in
cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD). It is estimated
that 10% of the cases of CF and 15% 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 designation 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 (http://www.mdausa.org ) and the Parent Project Muscular
Dystrophy (http://www.parentprojectmd.org ).
SOURCE PTC Therapeutics, Inc.
 back to top
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
PRN Photo Desk, photodesk@prnewswire.com
CONTACT:
Investors & Media: Jane Baj,
+1-908-222-7000, ext. 167, jbaj@ptcbio.com, or Patients,
Patients' Families, Investigators and Patient Organizations:
Kerri Donnelly, +1-908-222-7000, ext. 112, kdonnelly@ptcbio.com,
both of PTC Therapeutics, Inc.
|
