SOUTH PLAINFIELD, N.J., April 11 /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 that data from the Phase 1 multiple-dose
study of PTC124 were presented at the 57th Annual meeting of the American
Academy of Neurology meeting in Miami Beach, Florida. The study was also
featured in the Neurogenetics Highlights and Neuromuscular Highlights
sessions.
(Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )
PTC124 is a novel, orally administered drug that targets nonsense
mutations and is being investigated initially as a treatment for Duchenne
muscular dystrophy (DMD) and cystic fibrosis (CF), with the potential to treat
a number of other genetic disorders. It is estimated that 15% of DMD patients
and 10% of CF patients have these diseases as a consequence of nonsense
mutations.
The Phase 1 trial evaluated doses of up to 50 mg/kg twice-per-day for up
to 14 days of treatment in healthy volunteers. Endpoints included safety,
pharmacokinetics, and specificity of drug action as assessed by special safety
pharmacology testing. There were no symptomatic drug-related adverse events
at any dose level. Minor, reversible laboratory changes were noted in some of
the subjects. PTC124 administration achieved target plasma concentrations
associated with activity in preclinical models. PTC is working with
investigators and the FDA to finalize plans for Phase 2 studies in DMD and CF.
"The results of the multiple-dose study demonstrate the safety of PTC124
at the studied doses, as well as its specificity in reading through premature
termination codons," said Langdon L. Miller, M.D., Chief Medical Officer of
PTC. "We are very pleased with the rapid progress of PTC124 and honored to
have the opportunity to present our research findings. Our team looks forward
to advancing as quickly as possible into studying PTC124 in patients with DMD
or CF due to nonsense mutations."
"It is very gratifying to see PTC124 advance through preclinical and Phase
1 clinical development," indicated Stuart W. Peltz, Ph.D., President and CEO
of PTC. "We have been encouraged by the results of PTC124 thus far and look
forward to initiating Phase 2 studies."
About PTC Therapeutics, Inc.
PTC is a 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's compounds alter these processes by selectively
modulating how RNA is used to produce proteins. By applying this approach,
PTC has advanced its drug discovery programs rapidly from targets to
preclinical and clinical drug candidates, building a robust pipeline across
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. PTC124 allows the cellular machinery to bypass the
nonsense mutation and continue the translation process, restoring the
production of full-length, functional proteins. PTC124 has demonstrated the
ability to restore full-length functional protein in genetic disease models
harboring nonsense mutations. PTC124 represents a unique opportunity to use a
single small-molecule drug to address chronic and life-threatening diseases of
high unmet medical need. It is estimated that 10% of the cases of cystic
fibrosis (CF) and 15% of the cases of Duchenne muscular dystrophy (DMD) are
due to nonsense mutations. PTC has catalogued over 1,800 distinct genetic
disorders where nonsense mutations are the cause of the disease in an
appreciable percentage of patients. In addition to CF and DMD, other
potential indications under consideration for PTC124 include hemophilia,
neurofibromatosis, retinitis pigmentosa, epidermolysis bullosa, and lysosomal
storage disorders.
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 Cystic Fibrosis
Cystic fibrosis (CF) is a life-threatening, genetic disease affecting
approximately 30,000 people in the United States. A defective gene causes the
body to produce abnormally thick, sticky mucus that leads to chronic lung-
infections and impairs digestion. More information regarding CF is available
through the Cystic Fibrosis Foundation (http://www.cff.org). The Cystic
Fibrosis Foundation Therapeutics, Inc. (CFFT) has awarded PTC Therapeutics,
Inc. with a grant of $1.7 million for the development of PTC124 for the
treatment of cystic fibrosis (CF) due to a nonsense mutation in the cystic
fibrosis transmembrane regulator (CFTR) gene.
SOURCE PTC Therapeutics, Inc.
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Related links:
http://www.ptcbio.com
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; or Robert Stanislaro of
Noonan Russo, +1-212-845-4268, robert.stanislaro@eurorscg.com,
for PTC Therapeutics, Inc.
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