- PTC Announces Initiation of Additional Phase 2b Clinical Trial Sites in
Europe -
SOUTH PLAINFIELD, N.J., May 30 /PRNewswire/ -- PTC Therapeutics, Inc.
(PTC) today announced that PTC124, the company's orally delivered
investigational new drug for the treatment of genetic disorders due to
nonsense mutations, was featured in a symposium at Myology 2008, the Third
Annual Congress of Myology, on Friday, May 30 in Marseilles. The Congress
serves as the annual meeting of The Association Francaise contre les
Myopathies (French Muscular Dystrophy Association) and focuses on research,
therapies and clinical trials for various forms of muscular dystrophy.
(Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO)
PTC124 is being studied in Duchenne muscular dystrophy (DMD), a
progressive muscle disorder in which patients lack dystrophin, a protein
that is critical to the structural stability of muscle fibers. PTC recently
launched a global, registration-directed Phase 2b trial in DMD and Becker
muscular dystrophy (BMD) to evaluate the efficacy of PTC124 as measured by
improvements in the walking ability of patients with this progressive
genetic disease.
Data from the previous Phase 2a study were presented and discussed by a
panel of leading physicians and researchers, including: Dr. Thomas Voit,
M.D., Medical and Scientific Director of the Myology Institute and Dr.
Richard Finkel, M.D., Director of the Neuromuscular Program, Children's
Hospital of Philadelphia, PA.
"PTC124 represents a promising new therapy for DMD/BMD, as there are
currently no available treatments that address the underlying cause of this
disease," stated Dr. Voit. "The Phase 2b PTC124 clinical trial sets a gold
standard for future clinical trials in muscular dystrophies. I am delighted
to be part of these groundbreaking studies."
Presenters at the symposium also announced that the Phase 2b trial of
PTC124 was recently initiated in France, Belgium and Sweden and will begin
enrolling patients in additional European sites in the coming months,
broadening the reach of the clinical development program.
Genetic disorders, such as DMD and cystic fibrosis (CF), are caused by
genetic alterations, known as mutations. By targeting a specific type of
genetic alteration -- nonsense mutations -- PTC124 bypasses the defect and
leads to the restoration of a functional protein. The company has
catalogued over 2,400 distinct genetic disorders for which 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 of
these inherited diseases.
In preclinical models of genetic diseases harboring nonsense mutations,
PTC124 was shown to regulate post-transcriptional control processes by
restoring full-length, functional proteins. Post-transcriptional control
processes are the cellular regulatory events that take place after an RNA
molecule is copied from DNA. These processes are critical to proper
cellular function and provide an opportunity for therapeutic intervention
in a broad range of genetic disorders through the modulation of protein
levels.
"We are pleased to have the opportunity to share our exciting clinical
progress regarding PTC124 with the myology community," said Langdon Miller,
M.D., Chief Medical Officer of PTC. "Through its specificity and novel
mechanism of action, PTC124 has shown potential to address a broad range of
genetic disorders due to nonsense mutations. We look forward to continuing
to advance our clinical programs in DMD and CF and evaluating PTC124 in a
number of additional nonsense-mutation-mediated genetic disorders."
About DMD/BMD
Duchenne and Becker muscular dystrophy (DMD/BMD) are progressive muscle
disorders that cause the loss of both muscle function and independence.
DMD/BMD 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). It is estimated that one in 10 DMD patients
are likely to have a Becker presentation, a milder form of the disease that
is associated with later manifestation of symptoms. In essence, DMD and BMD
represent a continuum of the same disease. More information regarding DMD
and BMD is available through the Muscular Dystrophy Association
(http://www.mdausa.org), Parent Project Muscular Dystrophy
(http://www.parentprojectmd.org), and the Association Francaise contre les
Myopathies (http://www.afm-france.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 stop 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
Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF).
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). For additional information on the PTC124 clinical trial, please
visit http://www.clinicaltrials.gov and search using the keyword: PTC124.
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
http://www.ptcbio.com.
SOURCE PTC Therapeutics, Inc.
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Related links:
http://www.ptcbio.com
http://www.mdausa.org http://www.parentprojectmd.org
http://www.afm-france.org
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:
Investors and Media, Jane Baj of PTC
Therapeutics, Inc., +1-908-912-9167, jbaj@ptcbio.com; or Sheryl
Seapy of Pure Communications, +1-949-608-0841,
sheryl@purecommunicationsinc.com, for PTC Therapeutics, Inc.; or
Patients, Patients' Families, Investigators and Patient
Organizations, Diane Goetz of PTC Therapeutics, Inc.,
+1-908-912-9256, patientinfo@ptcbio.com
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