SOUTH PLAINFIELD, N.J., June 29 /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 complete data from the Phase 1
single- and multiple-dose studies and design of the planned Phase 2 studies
for PTC124 were presented at the 28th Annual European Cystic Fibrosis
Conference in Crete, Greece.
(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 cystic
fibrosis (CF) and Duchenne muscular dystrophy (DMD), with the potential to
treat a number of other genetic disorders. It is estimated that 10% of CF
patients and 15% of DMD patients have these diseases as a consequence of
nonsense mutations.
The results of the single- and multiple-dose studies in healthy volunteers
confirmed that PTC124 is generally well-tolerated and achieves target plasma
concentrations that have been associated with activity in preclinical models.
To demonstrate specificity of PTC124 effects, special tests were performed
that revealed no evidence of undesirable, nonspecific readthrough of normal
stop codons. Pharmacokinetic modeling of the Phase 1 results has allowed
development of a dosing regimen for the planned Phase 2 studies in CF and DMD.
The primary objective of the CF Phase 2 studies is to determine the
pharmacological activity of PTC124 as evaluated by nasal potential difference
assessment of chloride secretion. Secondary objectives will include other
measures of drug activity, evaluation of drug safety, determination of drug
levels in the blood, and assessment of patient compliance. PTC is also
working with international organizations to develop studies of PTC124 in
parallel with US-based efforts.
"The encouraging results of the Phase 1 studies provide a solid groundwork
for the Phase 2 studies," said Langdon L. Miller, M.D., Chief Medical Officer
of PTC. "We are making rapid progress in the clinic and anticipate that the
data from the Phase 2 trial will lead to an understanding of the potential
activity of PTC124 in patients with CF due to a nonsense mutation."
"We are delighted about the progress of PTC124 to date and committed to
advancing our efforts to study PTC124 for the treatment of CF and DMD due to a
nonsense mutation. We are extremely pleased with the results of Phase 1 and
enthusiastic about the opportunity to study PTC124 in patients for the first
time. This is a wonderful milestone and we are very proud to enter this
exciting new phase," said Stuart W. Peltz, Ph.D., President and CEO of PTC.
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 Cystic Fibrosis
Cystic fibrosis (CF) is a life-threatening, genetic disease affecting
approximately 60,000 people worldwide. 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 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.
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 ). The Muscular Dystrophy
Association (MDA) has awarded PTC with a grant of $1.5 million for the
development of PTC124 for the treatment of Duchenne muscular dystrophy (DMD)
due to a nonsense mutation. The Parent Project Muscular Dystrophy (PPMD) has
awarded PTC with a grant of $1M for drug discovery efforts on targets believed
to be of therapeutic relevance for DMD.
SOURCE PTC Therapeutics, Inc.
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Related links:
http://www.ptcbio.com
http://www.cff.org http://www.mdausa.org
http://www.parentprojectmd.org
Photo Notes:http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO
CONTACT:
Jane Baj of PTC Therapeutics, Inc.,
+1-908-222-7000, ext. 167, jbaj@ptcbio.com; Robert Stanislaro of
Noonan Russo, +1-212-845-4268, robert.stanislaro@eurorscg.com,
for PTC Therapeutics, Inc.
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