PTC Therapeutics Initiates a Phase 1 Multiple-Dose Study Of PTC124, An Investigational Drug for Cystic Fibrosis and Duchenne Muscular Dystrophy
Thursday December 23, 3:59 pm ET
Novel Treatment Has Potential Applicability Across Multiple Genetic Disorders
SOUTH PLAINFIELD, N.J., Dec. 23 /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 the initiation of a Phase 1 multiple-dose study of PTC124 to evaluate escalating dose levels in healthy volunteers. 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.
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PTC124 recently completed testing in a Phase 1 single-dose, dose-escalation study in healthy volunteers. Preliminary results confirmed that the drug is orally bioavailable, well tolerated, and safely achieves target plasma concentrations. Final results of Phase 1 studies are anticipated by the first quarter of 2005. Pending FDA concurrence, these results are expected to form the basis for initiation of Phase 2 studies in children and young adults with CF and DMD in the second quarter of 2005.
"The Phase 1 multiple-dose study will allow us to thoroughly examine the safety and pharmacokinetic profile of PTC124," stated Langdon Miller, M.D., Chief Medical Officer at PTC. "Upon conclusion of this study, we look forward to a rapid transition to Phase 2 studies in children and young adults with CF and DMD due to a nonsense mutation."
"Development of PTC124 offers a unique strategy for the treatment of genetic disorders, coupling a specific type of genetic defect with a small- molecule drug that has the potential to overcome that genetic defect," stated Stuart W. Peltz, Ph.D., President and CEO of PTC. "Our team is extremely enthusiastic about the progress to date. Given the unmet medical need in many genetic disorders, we are committed to rapidly advancing clinical trials of PTC124."
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, bullous skin diseases, and lysosomal storage disorders.
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).
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).
Additional Resources
Genetic testing for cystic fibrosis is available through Ambry Genetics (http://www.ambrygen.com).
Information regarding genetic testing for Duchenne muscular dystrophy is available through the University of Utah (http://www.genome.utah.edu/DMD/clinical_test.shtml).
Additional information regarding genetic disorders is available through the National Organization for Rare Disorders (http://www.rarediseases.org).
For more information:
Kerri Donnelly Robert Stanislaro
PTC Therapeutics, Inc. Euro RSCG Life NRP
(908) 222-7000, x112 (212) 845-4268
kdonnelly@ptcbio.com robert.stanislaro@eurorscg.com |
