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PTC Therapeutics Announces Pharmacokinetic And Safety Results From Phase 2 Study Of PTC124 In Duchenne Muscular Dystrophy
PTC Therapeutics, Inc.
(PTC), a biopharmaceutical company focused on the discovery, development
and commercialization of small-molecule drugs targeting
post-transcriptional control mechanisms, announced pharmacokinetic
and safety data from a Phase 2 clinical trial of PTC124 in patients with
Duchenne muscular dystrophy (DMD) due to a nonsense mutation. The results,
which include data from the third and final cohort of the study, show that
treatment with PTC124 appeared well tolerated at all three dose levels and
target plasma concentrations were achieved at the mid- and high-dose
levels. These data were presented at the 36th Annual Meeting of the
Child Neurology Society (CNS) in Quebec City, Canada.
In the study, patients received 28 days of PTC124 treatment at one of
three dose levels. All clinical trial participants are boys with a nonsense
mutation in the dystrophin gene, substantially elevated serum creatine
kinase levels, and symptoms associated with DMD. The analysis presented
today showed that PTC124 appeared well tolerated among the 38 boys included
in the study. Adverse events were infrequent, mild to moderate in severity,
and did not result in therapy interruptions or discontinuations. There were
no concerns based on physical examinations, vital sign measurements,
electrocardiograms or laboratory parameters. Compliance with PTC124
treatment was excellent at all three dose levels. Target plasma
concentrations associated with activity in a preclinical model of DMD were
achieved at the mid- and high-dose levels.
"DMD is a disorder with a significant need for better treatment options
and we are encouraged by the results we have seen to date with PTC124,"
said Brenda Wong, M.D., Associate Professor of Pediatrics and Neurology,
Cincinnati Children's Hospital Medical Center, Cincinnati, OH, who
presented the data today at CNS and is one of the trial's lead
investigators. "Based on the findings from this study, we believe that the
safety profile of PTC124 supports continued testing in longer-term
studies."
"These results add to the growing body of safety data for PTC124, which
has now been evaluated in more than 150 subjects, including patients with
both DMD and cystic fibrosis. The safety profile has consistently shown
that PTC124 appears well tolerated," said Langdon Miller, M.D., Chief
Medical Officer of PTC. "We are looking forward to presenting additional
activity data from this study next week at the World Muscle Society meeting
in Italy."
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 PTC124
PTC124 is an orally delivered investigational new drug 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 restored production of full-length,
functional proteins 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 read
through 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 CF and DMD by the European Commission. PTC124's
development is supported by grants from the Muscular Dystrophy Association
(MDA), Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), Parent Project
Muscular Dystrophy (PPMD), FDA's Office of Orphan Products Development
(OOPD) and by General Clinical Research Center grants from the National
Center for Research Resources (NCRR).
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 regulate the rate and timing of
protein production and are of central importance to proper cellular
function. PTC's internally- discovered pipeline addresses multiple
therapeutic areas, including genetic disorders, oncology and infectious
diseases. In addition, PTC has developed proprietary technologies and
extensive knowledge of post-transcriptional control processes that it
applies in its drug discovery and development activities, including the
Gene Expression Modulation by Small-molecules (GEMS) technology platform,
which has been the basis for collaborations with leading pharmaceutical and
biotechnology companies such as Pfizer, Celgene, CV Therapeutics and
Schering-Plough.
PTC Therapeutics, Inc.
http://www.ptcbio.com
PTC terapeutice Announces farmacocinetice ºi siguranþã rezultate de la faza 2 Studierea PTC124 În Duchenne Muscular Dystrophy - PTC Therapeutics Announces Pharmacokinetic And Safety Results From Phase 2 Study Of PTC124 In Duchenne Muscular Dystrophy - articole medicale engleza - startsanatate