SOUTH PLAINFIELD, N.J., Jan. 27 /PRNewswire/ -- 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 the initiation of a Phase 2 study of
PTC124 in patients with Duchenne muscular dystrophy (DMD) due to a nonsense
mutation. PTC124 is a novel, orally administered drug that targets nonsense
mutations and is being investigated initially as a treatment for DMD and
cystic fibrosis (CF), with the potential to treat a number of other genetic
disorders caused by nonsense mutations.
(Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )
"The initiation of the Phase 2 study of PTC124 in DMD marks a wonderful
milestone for PTC in its search for new treatments for DMD," commented Stuart
W. Peltz, Ph.D., President and CEO of PTC, adding, "We share the gratification
of this achievement with patients, their families, patient advocacy groups,
and investigators who have supported and helped guide our efforts. We hope
that information gained from this study will support the further development
of PTC124, and complement our efforts to discover and develop additional
therapies for boys with DMD."
The Phase 2 clinical study is enrolling patients who have DMD due to the
presence of a nonsense mutation in the dystrophin gene. The primary endpoint
of this Phase 2 multi-site, open-label, dose-ranging clinical study is
assessment of muscle dystrophin expression in response to treatment with
PTC124. Other assessments include the presence of dystrophin mRNA and
dystrophin-related proteins, muscle function, compliance with treatment,
safety, and pharmacokinetics.
"With the initiation of the Phase 2 study, we hope to establish proof-of-
concept for PTC124 in nonsense-mutation-mediated DMD," said Langdon Miller,
M.D., Chief Medical Officer at PTC. "Building on the positive preclinical
data with PTC124 that Doctors Lee Sweeney and Elisabeth Barton have generated
in their laboratories at the University of Pennsylvania, we have worked
closely with scientists and investigators to design a study that can examine
whether the encouraging preclinical findings with PTC124 translate to the
clinical setting. Demonstration of pharmacological activity in this study
would be an important step toward evaluating the longer-term clinical benefits
of PTC124."
Dr. Valerie Cwik, Medical Director of the Muscular Dystrophy Association
(MDA), commented: "The start of the PTC124 study opens a new era in clinical
trials for DMD, by testing a drug specifically aimed at overcoming the genetic
defect that causes the disease. We at MDA are very excited that this
potential treatment is now in clinical trials for DMD."
PTC has commenced recruitment for the Phase 2 study in DMD at the
Children's Hospital of Philadelphia in Philadelphia, PA, the Cincinnati
Children's Hospital Medical Center in Cincinnati, OH and the University of
Utah in Salt Lake City, UT. More details regarding the design and conduct of
this study are available at http://www.clinicaltrials.gov.
"Given the lack of effective targeted treatment for DMD, there is
tremendous interest in the development of PTC124 within the DMD patient and
scientific communities," commented Dr. Richard Finkel, Director, Neuromuscular
Program, Children's Hospital of Philadelphia. "We are excited by the results
in the animal model and are delighted to collaborate with PTC and the other
investigational sites in designing and conducting this important study in boys
with DMD."
PTC also has Phase 2 clinical trials for PTC124 underway with CF patients
in the United States and in Israel. More information regarding these trials
can be found at http://www.clinicaltrials.gov.
About PTC Therapeutics, Inc.
PTC is a privately-held 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 discovers and
develops small molecule drugs that alter these processes by selectively
modulating how RNA is used to produce proteins. This approach enables PTC to
advance its drug discovery programs rapidly from targets to preclinical and
clinical drug candidates, building a pipeline across multiple therapeutic
areas including 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. In pre-clinical trials, PTC124 allowed the cellular
machinery to bypass the nonsense mutation, continue the translation process,
and thereby restore the production of a full-length, functional protein.
PTC124 has demonstrated activity in preclinical genetic disease models
harboring nonsense mutations. In Phase 1 clinical studies, 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 has 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. In addition to CF and DMD, other potential indications include
hemophilia, neurofibromatosis, retinitis pigmentosa, epidermolysis bullosa,
and lysosomal storage disorders. PTC124 may represent a unique opportunity to
use a single small-molecule drug to address multiple chronic and life-
threatening diseases of high unmet medical need. 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. 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.
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Related links:
http://www.ptcbio.com
http://www.mdausa.org http://www.parentprojectmd.org
http://www.clinicaltrials.gov
Photo Notes:http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO
CONTACT:
Investors & Media, Jane Baj of PTC
Therapeutics, Inc., +1-908-222-7000, x167, jbaj@ptcbio.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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