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<a target=_blank class=ftalternatingbarlinklarge href="http://www.prnewswire.com/cgi-bin/stories.pl?ACCT=104&STORY=/www/story/02-04-2008/0004749209&EDATE=
">http://www.prnewswire.com/cgi-...008/0004749209&EDATE=
</a>
Data Show PTC124 Addresses Underlying Cause of Genetic Disorders and
Restores Protein Function in Cystic Fibrosis Model
SOUTH PLAINFIELD, N.J., Feb. 4 /PRNewswire/ -- PTC Therapeutics, Inc.
today announced the publication of new preclinical data in the February 12,
2008 edition of the Proceedings of the National Academy of Sciences (PNAS)
which show that PTC124, a novel drug designed to bypass nonsense mutations,
was active in a preclinical model of cystic fibrosis (CF). These results
support and add to research published last year in the journal Nature,
which demonstrated the activity of PTC124 in a preclinical model of
Duchenne muscular dystrophy (DMD). PTC124 has demonstrated pharmacodynamic
proof of concept in Phase 2a clinical trials in nonsense-mutation-mediated
CF and DMD.
(Logo: <a target=_blank class=ftalternatingbarlinklarge href="http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO">http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO</a> )
PTC has catalogued over 2,400 distinct genetic disorders where nonsense
mutations are the cause of the disease in a significant percentage of
patients. Nonsense mutations inactivate gene function and are known to
cause anywhere from five to 70 percent of the individual cases of most
inherited diseases, such as cystic fibrosis (10%) and Hurler's syndrome
(70%).
"The preclinical and clinical data on PTC124 support our hope that this
drug will be an important disease-modifying therapy for cystic fibrosis,"
said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis
Foundation. "This is an exciting potential new treatment for patients
afflicted with nonsense-mutation-mediated CF. We look forward to the next
stage of clinical development to demonstrate the benefits of this promising
new investigational drug."
As with the DMD data published in Nature, the results published in PNAS
further demonstrate that PTC124 targets genetic mutations in a completely
new way. PTC124 functions by overcoming the premature stop signal and
reading through the complete genetic instructions, resulting in the
restoration of a full-length, functional protein. Patients with CF lack the
CFTR protein, a chloride channel that maintains proper hydration of
epithelial cells in the lung, pancreas, and liver. The data in PNAS
demonstrate that PTC124 allows CFTR to be made in cells in which it was
previously absent, to be delivered to the proper cellular location, and to
induce chloride channel function. Collectively, these results indicate that
this new approach may ultimately be applicable to a subset of patients
across a large number of genetic disorders.
The paper entitled, "PTC124 is an orally bioavailable compound that
promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse
model," will be available in an advanced online publication of PNAS during
the week of February 4, 2008 (<a target=_blank class=ftalternatingbarlinklarge href="http://www.pnas.org/papbyrecent.shtml).">http://www.pnas.org/papbyrecent.shtml).</a> This
publication is the result of collaborative efforts between the University
of Alabama at Birmingham and PTC Therapeutics.
"These preclinical data further demonstrate the potential applicability
of the PTC124 mechanism of action to multiple genetic disorders," said
Stuart W. Peltz, Ph.D., President and Chief Executive Officer of PTC
Therapeutics. "We look forward to further evaluating PTC124 in
registration-directed clinical trials in both CF and DMD this year, as well
as in studies in additional genetic disorders in the future."
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. PTC124 has demonstrated pharmacodynamic
proof of concept in Phase 2a clinical trials 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 Subpart E
designation for expedited development, evaluation and marketing and has
granted 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
has been 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 the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the leading organization devoted to
curing and controlling cystic fibrosis. Headquartered in Bethesda, Md., the
Foundation funds CF research, has 80 chapter and branch offices, and
supports and accredits a nationwide network of 115 CF care centers, which
provide vital treatments and other CF resources to patients and families.
For more information, visit <a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org.
">http://www.cff.org.
</a>
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. For more information, visit the company's website,
<a target=_blank class=ftalternatingbarlinklarge href="http://www.ptcbio.com.
">http://www.ptcbio.com.
</a>
SOURCE PTC Therapeutics, Inc.
">http://www.prnewswire.com/cgi-...008/0004749209&EDATE=
</a>
Data Show PTC124 Addresses Underlying Cause of Genetic Disorders and
Restores Protein Function in Cystic Fibrosis Model
SOUTH PLAINFIELD, N.J., Feb. 4 /PRNewswire/ -- PTC Therapeutics, Inc.
today announced the publication of new preclinical data in the February 12,
2008 edition of the Proceedings of the National Academy of Sciences (PNAS)
which show that PTC124, a novel drug designed to bypass nonsense mutations,
was active in a preclinical model of cystic fibrosis (CF). These results
support and add to research published last year in the journal Nature,
which demonstrated the activity of PTC124 in a preclinical model of
Duchenne muscular dystrophy (DMD). PTC124 has demonstrated pharmacodynamic
proof of concept in Phase 2a clinical trials in nonsense-mutation-mediated
CF and DMD.
(Logo: <a target=_blank class=ftalternatingbarlinklarge href="http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO">http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO</a> )
PTC has catalogued over 2,400 distinct genetic disorders where nonsense
mutations are the cause of the disease in a significant percentage of
patients. Nonsense mutations inactivate gene function and are known to
cause anywhere from five to 70 percent of the individual cases of most
inherited diseases, such as cystic fibrosis (10%) and Hurler's syndrome
(70%).
"The preclinical and clinical data on PTC124 support our hope that this
drug will be an important disease-modifying therapy for cystic fibrosis,"
said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis
Foundation. "This is an exciting potential new treatment for patients
afflicted with nonsense-mutation-mediated CF. We look forward to the next
stage of clinical development to demonstrate the benefits of this promising
new investigational drug."
As with the DMD data published in Nature, the results published in PNAS
further demonstrate that PTC124 targets genetic mutations in a completely
new way. PTC124 functions by overcoming the premature stop signal and
reading through the complete genetic instructions, resulting in the
restoration of a full-length, functional protein. Patients with CF lack the
CFTR protein, a chloride channel that maintains proper hydration of
epithelial cells in the lung, pancreas, and liver. The data in PNAS
demonstrate that PTC124 allows CFTR to be made in cells in which it was
previously absent, to be delivered to the proper cellular location, and to
induce chloride channel function. Collectively, these results indicate that
this new approach may ultimately be applicable to a subset of patients
across a large number of genetic disorders.
The paper entitled, "PTC124 is an orally bioavailable compound that
promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse
model," will be available in an advanced online publication of PNAS during
the week of February 4, 2008 (<a target=_blank class=ftalternatingbarlinklarge href="http://www.pnas.org/papbyrecent.shtml).">http://www.pnas.org/papbyrecent.shtml).</a> This
publication is the result of collaborative efforts between the University
of Alabama at Birmingham and PTC Therapeutics.
"These preclinical data further demonstrate the potential applicability
of the PTC124 mechanism of action to multiple genetic disorders," said
Stuart W. Peltz, Ph.D., President and Chief Executive Officer of PTC
Therapeutics. "We look forward to further evaluating PTC124 in
registration-directed clinical trials in both CF and DMD this year, as well
as in studies in additional genetic disorders in the future."
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. PTC124 has demonstrated pharmacodynamic
proof of concept in Phase 2a clinical trials 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 Subpart E
designation for expedited development, evaluation and marketing and has
granted 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
has been 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 the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the leading organization devoted to
curing and controlling cystic fibrosis. Headquartered in Bethesda, Md., the
Foundation funds CF research, has 80 chapter and branch offices, and
supports and accredits a nationwide network of 115 CF care centers, which
provide vital treatments and other CF resources to patients and families.
For more information, visit <a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org.
">http://www.cff.org.
</a>
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. For more information, visit the company's website,
<a target=_blank class=ftalternatingbarlinklarge href="http://www.ptcbio.com.
">http://www.ptcbio.com.
</a>
SOURCE PTC Therapeutics, Inc.