A couple of PTC Therapeutics PR's..looks like they're firing up a few cylindars
please let me know if a more appropriate thread
ptcbio.com
SCHERING-PLOUGH AND PTC THERAPEUTICS ANNOUNCE COLLABORATION FOR DEVELOPMENT OF PTC’S PRECLINICAL HEPATITIS C COMPOUNDS
KENILWORTH, N.J., and SOUTH PLAINFIELD, N.J., March 20, 2006 - Schering-Plough Corporation (NYSE: SGP) and PTC Therapeutics, Inc. (PTC) today announced that they have entered into an exclusive collaboration and licensing agreement for the development of PTC’s preclinical compounds for the oral treatment of hepatitis C virus (HCV) infection and other viral diseases. PTC’s small molecules are designed to inhibit the HCV Internal Ribosome Entry Site (IRES) mediated production of viral proteins. The IRES is highly conserved among all HCV genotypes and is required for the expression of all viral proteins involved in replication of the hepatitis C virus.
PTC identified the compounds in its HCV program through the company’s proprietary Gene Expression Modulation by Small-molecules (GEMS) technology.
“The goal of this alliance is to develop new oral therapies to improve treatment for patients with hepatitis C, one of the most serious and common blood borne infections in the world,” said Catherine D. Strader, Ph.D., executive vice president, discovery research, Schering-Plough Research Institute. “Schering-Plough is continuing its leadership in hepatitis C through internal research programs and
$200 million. Schering-Plough will receive exclusive worldwide commercialization rights for any approved products and pay PTC royalties on worldwide net sales.
PTC’s proprietary Gene Expression Modulation by Small-molecules (GEMS) technology exploits the regulatory mechanisms found in the untranslated regions of messenger RNA for the identification of small molecule drugs that can treat diseases by selectively increasing or decreasing the expression of key proteins.
Chronic hepatitis C is estimated to affect more than 10 million people in major world markets, including in Canada, Europe and the United States. It is a leading cause of chronic liver disease and one of the most common reasons for liver transplant.
PTC Therapeutics is a privately-held 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
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development of and market for drugs that treat hepatitis C. Forward-looking statements relate to expectations or forecasts of future events. Schering-Plough does not assume the obligation to update any forward-looking statement. Many factors could cause actual results to differ materially from Schering-Plough’s forward-looking statements, including market forces, economic factors, product availability, patent and other intellectual property protection, current and future branded, generic or over-the-counter competition, the regulatory process, and any developments following regulatory approval, among other uncertainties. For further details about these and other factors that may impact the forward-looking statements, see Schering-Plough’s Securities and Exchange Commission filings, including Item 1A. Risk Factors in the Company’s 2005 10-K.
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strategic collaborations with innovative companies such as PTC, with a focus on developing targeted viral inhibitors,” she said.
“We are delighted to enter into this collaboration with Schering-Plough, as our teams share a commitment to develop new HCV therapies that will benefit patients,” said Stuart Peltz, president & CEO of PTC Therapeutics. “Schering-Plough’s expertise in the research and development of antiviral therapies and its global hepatitis franchise affords PTC the opportunity to realize the full potential of our IRES inhibitors. This collaboration provides additional validation for the GEMS technology and nicely complements our strategy as we advance internally the development of our clinical programs.”
Under the terms of the agreement, PTC and Schering-Plough will conduct a joint research program, and Schering-Plough will be responsible for development and commercialization efforts worldwide. Schering-Plough will make an upfront payment to PTC of $12 million and provide funding for PTC’s research efforts. Additionally, PTC can earn milestone payments if specific development, regulatory and commercial goals are achieved. Total payments to PTC could exceed $200 million. Schering-Plough will receive exclusive worldwide commercialization rights for any approved products and pay PTC royalties on worldwide net sales.
PTC’s proprietary Gene Expression Modulation by Small-molecules (GEMS) technology exploits the regulatory mechanisms found in the untranslated regions of messenger RNA for the identification of small molecule drugs that can treat diseases by selectively increasing or decreasing the expression of key proteins.
PTC THERAPEUTICS RECEIVES FAST TRACK DESIGNATION FOR PTC124 IN THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
ptcbio.com
SOUTH PLAINFIELD, N.J., March 30, 2006 - PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of small-molecule drugs that target post-transcriptional control processes, today announced that the company has been granted Fast Track designation from the United States Food and Drug Administration (FDA) for the development of PTC124 for the treatment of Duchenne muscular dystrophy (DMD) due to a nonsense mutation in the dystrophin gene. In December 2004, PTC124 was granted Orphan Drug designation by the FDA for the treatment of DMD. PTC124 is currently in Phase 2 clinical trials in DMD and cystic fibrosis (CF) in cases in which a nonsense mutation is the cause of the disease. PTC expects to complete these Phase 2 clinical trials in the second half of 2006.
The Fast Track program is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Development programs receiving Fast Track designations typically receive FDA priority review (6-month vs. standard 10-month review).
ABOUT PTC124
PTC124 is an orally delivered investigational product candidate in 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. In pre-clinical trials, the administration of PTC124 allowed the restoration of the production of full-length, functional proteins. PTC124 has demonstrated activity 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 readthrough of normal stop codons. Pharmacokinetic modeling of the Phase 1 results allowed development of a dosing regimen for the Phase 2 studies in cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD). It is estimated that 10% of the cases of CF and 15% 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 designation and Orphan Drug development of and market for drugs that treat hepatitis C. Forward-looking statements relate to expectations or forecasts of future events. Schering-Plough does not assume the obligation to update any forward-looking statement. Many factors could cause actual results to differ materially from Schering-Plough’s forward-looking statements, including market forces, economic factors, product availability, patent and other intellectual property protection, current and future branded, generic or over-the-counter competition, the regulatory process, and any developments following regulatory approval, among other uncertainties. For further details about these and other factors that may impact the forward-looking statements, see Schering-Plough’s Securities and Exchange Commission filings, including Item 1A. Risk Factors in the Company’s 2005 10-K.
“Fast Track designation in DMD is an additional important element in the development of PTC124,” said Stuart W. Peltz, Ph.D., President and CEO of PTC. “DMD is an unmet medical need where only palliative options are currently available. We hope PTC124 will represent a therapeutic option for patients with DMD due to a nonsense mutation.”
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 are of central importance to the regulation of the rate and timing of cellular protein production. PTC has assembled proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities. PTC’s current pipeline of clinical and preclinical product candidates addresses multiple indications, including genetic disorders, oncology, and infectious diseases.
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designations for the treatment of CF and DMD due to nonsense mutations. PTC124 has also been granted orphan drug status for the treatment of DMD and CF by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA). PTC124’s development is supported by grants from the Muscular Dystrophy Association (MDA), Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), FDA’s Office of Orphan Products Development (OOPD), and by General Clinical Research Center grants from the National Center for Research Resources (NCRR).
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 (www.mdausa.org) and the Parent Project Muscular Dystrophy (www.parentprojectmd.org). |
