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PTC Therapeutics Initiates A Phase 1 Multiple-Dose Study of Its Novel VEGF Inhibitor, PTC299

- Data from Phase 1a single-dose study to be presented at EORTC-NCI-AACR Conference -

SOUTH PLAINFIELD, NJ – November 7, 2006 - PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of small--molecule drugs targeting post-transcriptional control mechanisms, today announced the initiation of a Phase 1 multiple-dose study to evaluate escalating dose levels of PTC299 in healthy volunteers. PTC299 is a novel, orally bioavailable drug designed to modulate RNA-mediated protein expression to inhibit the production of vascular endothelial growth factor (VEGF) in tumors.

PTC has recently completed a Phase 1a escalating, single-dose safety and pharmacokinetic study of PTC299 in healthy volunteers. Preliminary results confirmed that the drug is orally bioavailable, well tolerated, and safely achieves the desired target plasma concentrations. Data from this trial will be presented in Poster Number 51, entitled, “Phase 1 Single-dose Safety, PK, and Food-effect Study of PTC299, a Novel VEGF Expression Inhibitor for Treatment of Solid Tumors” at the 18th Annual EORTC-NCI-AACR Symposium “Molecular Targets and Cancer Therapeutics.” The conference will take place from November 7th to the 10th in Prague, Czech Republic. 

“The favorable safety profile of PTC299 in preclinical toxicology testing has allowed us to perform these initial Phase 1 single-dose and multiple-dose studies in healthy volunteers,” noted Langdon Miller, M.D., Chief Medical Officer at PTC Therapeutics, Inc. “The safety and pharmacokinetic data obtained from these trials should allow a prompt transition to Phase 1-2 clinical trials in cancer patients. The advantage of this approach is that we have already established PTC299 dose levels that are likely to be well-tolerated and achieve plasma concentrations associated with preclinical testing.”

“We are very pleased with the continued progress of PTC299,” commented Stuart W. Peltz, Ph.D., President and CEO of PTC Therapeutics, Inc. “PTC299 is the second compound that entered clinical development from our post-transcriptional control technologies that identify small molecules which affect RNA biology. In particular, PTC299 is an important validation of our GEMS technology. Given the very high unmet medical need in oncology, we are committed to rapidly advancing clinical trials of PTC299.”

PTC will also present two preclinical PTC299 posters at the conference: Poster Number 52, entitled “Preclinical Development of PTC299: An Orally Bioavailable Small-molecule Drug That Selectively Inhibits VEGF Protein Production, Tumor Growth, and Microvessel Density” and Poster Number 88, entitled “PTC299 Inhibits VEGF Expression Through Its 5´-UTR.” All PTC posters will be on display on Wednesday, November 8th from noon to 3pm. More information regarding the conference can be found at http://www.fecs.be/emc.asp?pageId=973. 

About PTC299

PTC299 is a novel, orally administered small-molecule compound that inhibits the production of the protein vascular endothelial growth factor, or VEGF, in tumors. PTC discovered PTC299 through PTC’s proprietary GEMS (Gene Expression Modulation by Small-Molecules) technology by targeting the post-transcriptional processes that regulate VEGF formation. Over expression of VEGF plays a key role in the growth of many types of cancers. PTC299 inhibits VEGF production through a mechanism that is distinct from other VEGF inhibitors and has potential application in the treatment of multiple solid tumor types. In September 2006, PTC announced that it had received a three-year, $2.2 million grant from the Department of Defense (DoD) to fund preclinical and clinical development of PTC299 as a potential treatment for breast cancer.

About GEMS

Gene Expression Modulation by Small-molecules (GEMS) is PTC’s novel and proprietary screening technology for the identification of small-molecules that modulate post-t-transcriptional control mechanisms. Compounds identified through the GEMS technology target processes that act through the untranslated regions (UTRs) of messenger RNA (mRNA) molecules. GEMS was the basis for the discovery of PTC299 and is being used in multiple ongoing drug discovery programs.

About PTC Therapeutics, Inc.

PTC is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small-molecule drugs that target post-st-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 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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PTC Therapeutics Announces Positive Phase 2 Results For PTC124 in Cystic Fibrosis

- Data Indicate that PTC124 Addresses Underlying Cause of Cystic Fibrosis Through Improvements in CFTR Function -

SOUTH PLAINFIELD, NJ – November 3, 2006 - PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of small-molecule drugs targeting post-transcriptional control processes, today announced the findings from two Phase 2 clinical trials of PTC124 in patients with cystic fibrosis (CF) due to a nonsense mutation. The results suggest that PTC124 can restore function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in airway cells and significantly reduce blood neutrophil counts that are a hallmark of the CF disease process. The initial data were presented today at the North American Cystic Fibrosis Conference in Denver, Colorado.

“These results are very exciting because they provide the first indication that an oral therapy may address the underlying cause of CF through restoration of CFTR function,” said J.P. Clancy, M.D., Director of the Pediatric Pulmonary Division, University of Alabama at Birmingham, and the lead investigator of the U.S. Phase 2 PTC124 trial. “These data indicate that PTC124 warrants further clinical investigation in this patient population, which currently can only be treated with supportive therapies.”

"We are encouraged that PTC124 provides hope for a particular group of individuals with CF, as well as offering important scientific information that may have broader implications for pharmacological approaches to CF,” said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. “It is gratifying to see the Cystic Fibrosis Foundation Therapeutics’ support of this drug yield encouraging results." 

Patients with CF lack the CFTR protein, a chloride channel that maintains proper hydration of epithelial cells in the lung, pancreas, and liver. Patients with CF develop highly viscous secretions in these organs that result in inflammation, chronic colonization of pathogenic bacteria and progressive organ destruction. Pulmonary involvement usually causes the greatest disability as blood neutrophils migrate into the lungs, contributing to clogging of the airways. Patients experience chronic shortness of breath, coughing and production of thick, sticky sputum filled with neutrophils and bacteria.

PTC sponsored a multi-site, open-label, dose-ranging Phase 2 clinical trial program to determine whether PTC124 can induce production of active CFTR protein. Identical studies in the U.S. and Israel evaluated nasal transepithelial potential difference (TEPD) as a surrogate for CFTR protein production. A change in CFTR-mediated chloride transport toward normal during PTC124 treatment would suggest that PTC124 is inducing the cells to make full-length, functional CFTR protein. TEPD is measured using a small plastic catheter to assess electrical differences across the cell membrane in each nostril. Also assessed were circulating blood neutrophil and liver enzyme values, lung function, and body weight, as well as safety, compliance, and pharmacokinetics. Patients received two sequential 14-day courses of treatment of PTC124, first at a lower and then at a higher dose level. 

All patients were adults with a nonsense mutation in one of their CFTR genes. Over 90% had severe CF as characterized by colonization of the lung with Pseudomonas aeruginosa bacteria, pancreatic insufficiency requiring pancreatic enzyme replacement to prevent malabsorption of food, and relative elevations of neutrophil levels in blood. Patients tended to be underweight due to complications of CF.

Across the two studies, at both PTC124 dose levels tested, TEPD assessments showed statistically significant (p<0.03) improvements of mean CFTR-dependent chloride secretion in the airways. By the end of the first cycle of treatment, 43% (18/42) had responded with a change of at least -5 mV in chloride secretion TEPD and 36% (15/42) had chloride secretion TEPD values in the generally accepted normal range (more electrically negative than -5 mV). CFTR chloride secretion responses were observed in both the U.S. and Israel among several of the most common nonsense mutation genotypes affecting patients with CF. 

In evaluating the studies separately, the results from the Israeli study demonstrated statistical significance for chloride secretion TEPD response for the population as a whole while the interim results from the U.S. study demonstrated such responses in several patients but the trends did not reach statistical significance. The differences in detection of chloride secretion response between the studies may result from a number of factors, including differences in use of concomitant inhaled medications. Blood neutrophil counts were also monitored before and during PTC124 treatment because CF is a neutrophil-mediated disease, and reductions in blood neutrophil counts may be consistent with PTC124 activity. Statistically significant reductions (p<0.02) in blood neutrophil counts were observed in both the U.S. and Israeli studies. Furthermore, improvements in circulating levels of liver enzymes in the blood were seen in both trials, supporting the hypotheses that PTC124 would offer systemic benefits to patients with multiorgan compromise due to CF. Trends toward improved pulmonary function and body weight were also observed in patients participating in the Phase 2 program. Although a formal symptom assessment was not a component of the Phase 2 program, a number of patients described decreased sputum volume and thickness, decreased frequency and severity of coughing and a better sense of well-being during PTC124 therapy. PTC124 was well tolerated, resulting in excellent compliance with the treatment regimen (>95%).

Eitan Kerem, M.D., Head of Pediatrics and CF Center at the Hadassah University Hospital in Mount Scopus, Jerusalem commented, “More than 60% of CF patients in Israel have CF due to a nonsense mutation. Thus, we were very gratified to see such remarkable improvements in CFTR function and other parameters in just a two-week treatment period. Based on this clear demonstration of PTC124 activity we will be initiating a three-month study of PTC124 in patients with nonsense-mutation-mediated CF to further evaluate its potential for providing clinical benefit.”

Based on the results of the PTC124 CF studies, PTC will be conducting a longer term study in Israel at the Hadassah Medical Center in Jerusalem, and will initiate a pediatric clinical trial in France with a lead investigator who has extensive experience in nonsense-mutation-mediated CF. Collective results from the Phase 2 studies will be reviewed with regulatory authorities with the intent of initiating an international Phase 3 trial program within 2007.

"The results of these trials clearly establish clinical support for the potential of PTC124 in genetic disorders due to a nonsense mutation,” said Stuart W. Peltz, Ph.D., President and Chief Executive Officer, PTC Therapeutics. “When considered together with the encouraging results recently announced in patients with Duchenne muscular dystrophy, we have established a strong basis for advancing the clinical development of PTC124 and we are currently conducting preclinical studies with the intent to extend this concept into other disorders.”

The patients included in the analyses were enrolled at a single center in Israel, at the Hadassah Medical Center, Jerusalem and at five clinical sites in the United States: University of Alabama at Birmingham, Birmingham, AL; the Johns Hopkins Hospital, Baltimore, MD; Rainbow Babies’ and Children’s Hospital, Cleveland, OH; Denver Children’s Hospital, Denver, CO; and Stanford University Medical Center, Stanford, CA. All of these sites are member institutions of the Cystic Fibrosis Foundation Therapeutics Development Network (CFF-TDN), which is supported by the Cystic Fibrosis Foundation (CFF), and is collaborating with PTC in the development of PTC124. 

PTC will also conduct a longer-term Phase 2b cystic fibrosis program in Israel at the Hadassah Medical Center, Jerusalem and will initiate a study in France in children with CF.

 

About Cystic Fibrosis

CF is among the most common life-threatening genetic disorders worldwide. According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and children in the United States and, according to the European Cystic Fibrosis Foundation, it affects a similar number of patients in Europe. CF occurs in approximately one of every 3,500 live births, with approximately 1,000 new cases diagnosed each year in the United States. There is a commercially available genetic test to determine if a patient’s CF is caused by a nonsense mutation and it is estimated that nonsense mutations are the cause of CF in approximately 10% of patients in the United States. There is currently no available therapy to correct defective CFTR production and function. Instead, available treatments for CF are designed to alleviate the symptoms of the disease. These treatments include chest physical therapy to clear the thick mucus from the lungs, antibiotics to treat lung infections and a mucus-thinning drug designed to reduce the number of lung infections and improve lung function. In addition, the majority of cystic fibrosis patients take pancreatic enzyme supplements to assist with food absorption in digestion. There is a significant unmet medical need for a treatment for the underlying cause of CF. More information regarding CF is available through the Cystic Fibrosis Foundation (www.cff.org).

About PTC124

PTC124 is an orally delivered product candidate 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 demonstrated activity in preclinical genetic disease models harboring nonsense mutations allowing the restoration of the production of full-length, functional proteins. 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. 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 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), 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 University of Alabama at Birmingham

The University of Alabama at Birmingham (UAB) is a research university and academic health center that discovers, teaches and applies knowledge for the intellectual, cultural, social and economic benefit of Birmingham, the state and beyond. UAB encompasses 82 city blocks and has a student enrollment of more than 17,000. UAB also is home to a large graduate school, a world-renowned health care complex and more than 70 research centers, focusing on such diverse issues as AIDS vaccines and aging to the environment, urban affairs, and telecommunications.

About The Hadassah University Medical Center, Jerusalem

A state-of-the-art medical center incorporating all medical and surgical sub-specialties, with two hospitals at Ein Kerem and on Mt. Scopus; conducts more than half the hospital research in Israel.

Founded, owned and supported by Hadassah, the Women's Zionist Organization of America, the largest women's, largest Jewish and largest volunteer organization in America, with over 300,000 members in more than 1,000 chapters.

About PTC Therapeutics, Inc.

PTC is a biopharmaceutical company focused on the discovery and development 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 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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PTC Therapeutics Announces Encouraging Preliminary Phase 2 Results of PTC124 in Duchenne Muscular Dystrophy

SOUTH PLAINFIELD, NJ – October 21, 2006 - PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of small-molecule drugs targeting post-transcriptional control processes, today announced encouraging data from a Phase 2 clinical trial of PTC124 in patients with Duchenne muscular dystrophy (DMD) due to a nonsense mutation. The results imply pharmacological activity based on preliminary data that suggest increases in dystrophin in muscle biopsies in a number of patients and statistically significant improvements in muscle enzymes in serum. The preliminary data were presented today at the PPUK 4th International DMD Conference in London, England.

“These results are the first example of an oral therapy addressing the underlying cause of DMD by restoring dystrophin production,” said Dr. Richard Finkel, Director of the Neuromuscular Program, Children’s Hospital of Philadelphia, PA, one of the trial’s lead investigators. “There are limited therapeutic options for patients living with DMD, and these data strongly indicate PTC124 warrants further clinical investigation in this patient population, which has a great unmet medical need.”

Langdon Miller, M.D., Chief Medical Officer, PTC, stated, “These preliminary results in patients with DMD provide confirmation of proof of concept that PTC124 can induce ribosomal readthrough of nonsense mutations as an approach to treating genetic disorders. Given that PTC124 was very well-tolerated and activity was observed at lower-than-expected plasma concentrations, we are amending this trial to evaluate higher dose levels and the potential to further increase dystrophin expression.”

“In the first half of 2007, we expect to present the final data set from this Phase 2 clinical trial and meet with regulatory authorities to determine the next steps for further clinical development of PTC124. Following these discussions, we hope to initiate longer-term clinical trials for PTC124 in 2007,” said Dr. Miller.

Patients with DMD lack dystrophin, a protein that is critical to the structural stability of muscle fibers. This Phase 2 multi-site, open-label, dose-ranging clinical trial is evaluating muscle dystrophin expression in patients with nonsense-mutation-mediated DMD. Blood levels of muscle-derived creatine kinase are being measured as assessments of muscle integrity. PTC124 safety, compliance, and pharmacokinetics are also being evaluated.

Patients included in the interim analysis were enrolled at three clinical sites in the United States: Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; and the University of Utah, Salt Lake City, Utah. In the study, patients received 28 days of PTC124 treatment at one of two dose levels. All patients were boys with a nonsense mutation in the dystrophin gene, substantially elevated serum creatine kinase, and symptoms associated with DMD.

Assessment of the in vitro effects of PTC124 on dystrophin expression showed dose-dependent production of full-length dystrophin in myocytes obtained from multiple study subjects; these data suggest the potential for response across a range of early to late nonsense mutations within the dystrophin gene. Evaluation of the in vivo effects of PTC124 over the 28-day treatment course suggest an increase in dystrophin expression in muscle biopsies in a number of the boys participating in the trial, although quantitative analysis is not yet complete. Statistically significant reductions in the concentrations of muscle-derived creatine kinase levels in the blood were observed during PTC124 treatment. Although no formal questionnaire was used to collect data on changes in DMD-related symptoms, several parents and teachers reported that boys participating in the study had improvements in terms of greater activity level and increased endurance during treatment.

PTC124 was well tolerated among the 26 patients included in the study. Potentially drug-related adverse events were infrequent, mild to moderate in severity, did not result in therapy interruptions or discontinuations, and were reversible. There were no safety concerns based on physical examinations, vital sign measurements, electrocardiograms, or laboratory parameters. Compliance was excellent at both dose levels. 

“These preliminary results are very encouraging and add to the growing body of clinical evidence supporting the potential of PTC124 as a treatment for genetic disorders due to a nonsense mutation,” said Stuart W. Peltz, Ph.D., President and Chief Executive Officer of PTC Therapeutics. “The findings in the DMD trials are consistent with the results observed in Phase 2 clinical trials of PTC124 in patients with cystic fibrosis. We intend to extend this concept into other nonsense-mediated genetic disorders.”

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).

About PTC124

PTC124 is an orally delivered product candidate 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 demonstrated activity in preclinical genetic disease models harboring nonsense mutations allowing the restoration of the production of full-length, functional proteins. 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. 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 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), 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 and development 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 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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FDA Awards PTC Therapeutics Orphan Products Development Grant for the Development of PTC124 in Duchenne Muscular Dystrophy

- Preliminary Data to be Presented at Parent Project UK Muscular Dystrophy Conference -

SOUTH PLAINFIELD, NJ – October 19, 2006 - PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of small-molecule drugs targeting post-transcriptional control mechanisms, today announced it has been awarded a two-year grant from the Office of Orphan Products Development (OOPD) Grant Program of the U.S. Food and Drug Administration (FDA). The grant will support the patient-related costs of the company’s ongoing U.S.-based Phase 2 clinical trial of PTC124 for the treatment of nonsense-mutation-mediated Duchenne muscular dystrophy (DMD). 

“It is an honor to receive this grant from the FDA in support of our clinical development efforts,” stated Stuart Peltz, Ph.D., President and CEO of PTC Therapeutics. “Last year, PTC was awarded a grant from the FDA for the development of PTC124 as a potential treatment for cystic fibrosis (CF), which we have used to advance our Phase 2 CF program. Similarly, this new award will support the development of PTC124 as a potential treatment for DMD.”

PTC initiated the Phase 2 DMD clinical trial of PTC124 in January 2006. Patients with DMD lack dystrophin, a protein that is critical to the structural stability of muscle fibers. This Phase 2 multi-site, open-label, dose-ranging clinical trial is evaluating muscle dystrophin expression in patients with nonsense-mutation-mediated DMD. Blood levels of muscle-derived creatine kinase are being measured as assessments of muscle integrity. PTC124 safety, compliance, and pharmacokinetics are also being evaluated.

Preliminary data from this study will be presented at the Parent Project UK Muscular Dystrophy 4th International DMD conference on Saturday, October 21st by Dr. Richard Finkel, Director of the Neuromuscular Program at The Children’s Hospital of Philadelphia and one of the lead investigators of the PTC DMD trial. The conference will be held at the Institute of Education in London, England, October 21st and 22nd, 2006. 

About The Office of Orphan Products Development

The Office of Orphan Products Development (OOPD) has been dedicated to promoting the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions since it was created in 1982. OOPD interacts with the medical and research communities, professional organizations, academia, and the pharmaceutical industry, as well as rare disease groups. The OOPD administers the major provisions of the Orphan Drug Act (ODA) which provide incentives for sponsors to develop products for rare diseases. The ODA has been very successful - more than 200 drugs and biological products for rare diseases have been brought to market since 1983. In contrast, the decade prior to 1983 saw fewer than ten such products come to market. In addition, the OOPD administers the Orphan Products Grants Program which provides funding for clinical research in rare diseases.

About PTC124

PTC124 is an orally delivered investigational product candidate 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 demonstrated activity in preclinical genetic disease models harboring nonsense mutations allowing the restoration of the production of full-length, functional proteins. 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. 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 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 and the Parent Project Muscular Dystrophy.

About PTC Therapeutics, Inc.

PTC is a biopharmaceutical company focused on the discovery and development 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 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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PTC Therapeutics Announces $17.2 Million Award from The United States Defense Threat Reduction Agency

--Contract Addresses Discovery and Development of Compounds to Combat Infectious Bacteria Used as Biowarfare and Bioterrorism Agents--

SOUTH PLAINFIELD, NJ – October 2, 2006 - PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes, today announced that it has received a $17.2 million award from the United States Defense Threat Reduction Agency (DTRA) for the discovery and development of broad-spectrum small-molecule antibacterials that may be applicable against biowarfare or bioterrorism agents. PTC plans to utilize the three-year contract to fund the discovery and development of small-molecule compounds through the initiation of Phase 1 clinical trials.

PTC has applied its expertise in addressing RNA biology with small-molecule drugs to identify compounds to combat bacterial infections through post-transcriptional control processes. In in vitro tests, the lead compounds in PTC’s antibacterial program demonstrated significant activity against several drug-resistant strains of bacteria, including methicillin-resistant and multidrug-resistant Staphylococcus aureus and vancomycinin-resistant Enterococci. These antibacterial effects have been achieved without toxicity in human cell lines with the lead compounds exhibiting favorable pharmacokinetic properties.

“We are honored to receive this award from the Defense Threat Reduction Agency to develop novel antibacterial agents. The DTRA award is another example of the potential applicability of our post-transcriptional drug discovery technologies to a wide range of therapeutic areas,” commented Stuart W. Peltz, President and CEO of PTC Therapeutics, Inc. “The DTRA award allows us to diversify our pipeline to include antibacterial agents targeting post-transcriptional control mechanisms.”

About the Defense Threat Reduction Agency (DTRA)

The Defense Threat Reduction Agency (DTRA) safeguards America and its allies from weapons of mass destruction by providing capabilities to reduce, eliminate, and counter the threat, and mitigate its effects. This Department of Defense agency is located at Fort Belvoir, Va., and operates field offices worldwide.

The Chemical and Biological Technologies Directorate within DTRA provides direct Science and Technology (S & T) support for the nation's defense through its many medical and physical (non-medical) S & T programs and initiatives to improve U.S. defensive capabilities against Chemical and Biological Weapons and to protect the safety of U. S. forces operating in a contaminated environment.

About PTC Therapeutics, Inc.

PTC is a biopharmaceutical company focused on the discovery and development 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 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.

PTC Therapeutics Receives Department of Defense Clinical Translational Research Award

-$2.2 Million Grant Will Fund Preclinical and Clinical Development of PTC299 in Breast Cancer-

SOUTH PLAINFIELD, NJ – September 26, 2006 - PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes, today announced that it has received a three-year, $2.2 million grant from the Department of Defense (DoD) to fund preclinical and clinical development of the company’s product candidate PTC299 as a potential treatment for breast cancer. 

PTC299 was discovered through PTC’s proprietary Gene Expression Modulation by Small-molecules (GEMS) technology. PTC299 is an orally-administered small molecule designed to inhibit the production of vascular endothelial growth factor, or VEGF. PTC299 functions by targeting the post-transcriptional control processes that regulate VEGF formation. PTC299 inhibits VEGF production through a mechanism that is distinct from other VEGF inhibitors. VEGF plays a major role in the growth of most tumors, therefore PTC299 may have applicability in many cancer types, including breast cancer. PTC299 is currently in Phase 1 clinical trials in healthy volunteers. 

“We are very pleased to receive this award from the DoD to assist us in the Phase 1-2 development of PTC299 patients with breast cancer,” stated Langdon Miller, M.D., Chief Medical Officer, PTC Therapeutics, Inc. and principal investigator for the grant. “The grant provides important validation to PTC’s innovative scientific approach of modulating post-transcriptional control elements with a small molecule to treat human diseases.”

PTC is collaborating with researchers at the New York University (NYU) Medical Center in New York City. Dr. Robert Schneider, Director of Translational Cancer Research, Co-Director of the Breast Cancer Research Program and an Associate Director of the NYU Cancer Institute, who serves as a co-principal investigator for the DoD award, commented, “This collaboration combines the preclinical and clinical development expertise of an experienced biopharmaceutical company and a DoD Breast Cancer Center of Excellence. This funding will support translation of preclinical pharmacological and biomarker data into the clinic while offering the potential to address the high unmet medical need of patients with breast cancer.”

About Department of Defense Clinical Translational Research Award

The DoD Clinical Translational Research Award sponsors innovative preclinical and clinical/translational research that may result in substantial improvements over current approaches to breast cancer chemoprevention and therapy. As part of the Congressionally Directed Medical Research Programs, it administers funds for peer-reviewed research toward specific diseases and supports research that positively affects the health and well-being of Americans. For this grant (grant number W81XWH-06-1-0629), the U.S. Army Medical Research Acquisition Activity, 820 Chandler Street, Fort Detrick, MD 21702-5014 is the awarding and administering acquisitions office. The content of this press release does not necessarily reflect the position or the policy of the Government, and no official endorsement should be inferred.

About NYU Medical Center Breast Cancer Research

Located in the heart of New York City, NYU Medical Center is one of the nation's premier centers of excellence in health care, biomedical research, and medical education. The mission of the NYU Cancer Institute is to decrease and eliminate cancer as a significant health problem throughout New York, the nation, and the world, by developing and maintaining excellent programs in patient care, research, education, and prevention.

About GEMS

Gene Expression Modulation by Small-molecules (GEMS) is PTC’s novel and proprietary screening technology for the identification of small-molecules that modulate post-transcriptional control mechanisms. Compounds identified through the GEMS technology modulate gene expression by targeting the post-transcriptional control processes that act through the untranslated regions (UTRs) of messenger RNA (mRNA) molecules. GEMS was the basis for the discovery of PTC299 and is being used in multiple ongoing drug discovery programs.

About PTC Therapeutics, Inc.

PTC is a biopharmaceutical company focused on the discovery and development 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 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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PTC Therapeutics and CV Therapeutics Announce Collaboration and License Arrangement

SOUTH PLAINFIELD, NJ, and PALO ALTO, CA, June 12, 2006 — PTC Therapeutics, Inc. and CV Therapeutics, Inc. (NASDAQ: CVTX) announced today that they have entered into an exclusive research collaboration and licensing arrangement for the development of orally bioavailable small molecules through the application of PTC's proprietary GEMS (Gene Expression Modulation by Small-Molecules) technology. Pursuant to the collaboration, PTC and CV Therapeutics will jointly select five targets, including targets with the potential to raise HDL, through a collaborative process of determining the applicability of the GEMS technology to targets of interest.

Under the terms of the collaboration and license agreement, CV Therapeutics will make an initial payment to PTC of $10 million. The initial payment consists of a cash upfront payment of $2 million and two loans for an aggregate of $8 million. One of the loans is forgivable over the course of the research term provided the agreement remains in effect, and the other is convertible into PTC equity. CV Therapeutics has the exclusive option to enter into one or more worldwide, exclusive licenses, on a target-by-target basis, for collaboration compounds it decides to take forward, and will be obligated to pay PTC royalties on worldwide net sales of products developed pursuant to the collaboration. If CV Therapeutics licenses and commercializes a product based on each of the five selected targets during the term of the agreement, PTC could earn milestone payments of up to $335 million if all specified development, regulatory and commercial goals are achieved over the product life cycle. PTC retains the option to co-fund research and development for increased royalties or co-promotion rights.

"We are very excited to start our collaboration with PTC, which we believe has the potential to deliver innovative oral treatments for well-validated targets in important areas of cardiovascular disease, such as dyslipidemia and diabetes," said Brent K. Blackburn, Ph.D., senior vice president, drug discovery and development of CV Therapeutics. "This alliance complements our internal research efforts with PTC's proprietary GEMS technology that allows the potential development of small-molecule drugs to address challenging targets."

"We are extremely pleased to enter into this collaboration with CV Therapeutics," said Stuart Peltz, president and chief executive officer of PTC Therapeutics. "This collaboration is part of our strategy of applying our GEMS technology to therapeutic areas outside of our core research areas through collaborators who bring disease-specific expertise. With CV Therapeutics, we have a collaborator with a significant presence and experience in molecular cardiology and drug development."

About CV Therapeutics
CV Therapeutics, Inc., headquartered in Palo Alto, California, is a biopharmaceutical company focused on applying molecular cardiology to the discovery, development and commercialization of novel, small molecule drugs for the treatment of cardiovascular diseases.

CV Therapeutics' approved products include Ranexa^® (ranolazine extended-release tablets) and ACEON^® (perindopril erbumine) Tablets. Ranexa is indicated for the treatment of chronic angina in patients who have not achieved an adequate response with other antianginal drugs, and should be used in combination with amlodipine, beta-blockers or nitrates. In addition, CV Therapeutics co-promotes ACEON®, an ACE inhibitor, for reduction of the risk of cardiovascular mortality or nonfatal myocardial infarction in patients with stable coronary artery disease and treatment of essential hypertension.

CV Therapeutics also has other clinical and preclinical drug development candidates and programs, including regadenoson, which is being developed for potential use as a pharmacologic stress agent in myocardial perfusion imaging studies. Regadenoson has not been approved for marketing by any regulatory authorities.

CV Therapeutics Disclosure Statement
Except for the historical information contained herein, the matters set forth in this press release, including statements as to drug research and development, clinical studies, regulatory review and approval, and commercialization of products, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially, including, early stage of development; regulatory review and approval of our products; the conduct and timing of clinical trials; commercialization of products; market acceptance of products; and other risks detailed from time to time in CV Therapeutics' SEC reports, including its Quarterly Report on Form 10-Q for the quarter ended March 31, 2006. CV Therapeutics disclaims any intent or obligation to update these forward-looking statements

About PTC Therapeutics, Inc.
PTC is a biopharmaceutical company focused on the discovery and development 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 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.

About GEMS
Gene Expression Modulation by Small-Molecules (GEMS) is PTC's novel and proprietary screening technology for the identification of small-molecules that modulate post-transcriptional control mechanisms. Compounds identified through the GEMS technology modulate gene expression by targeting the post-transcriptional control processes that act through the untranslated regions (UTRs) of messenger RNA (mRNA) molecules. GEMS was the basis for the discovery of PTC's product candidate, PTC299 and also for compounds in PTC's Hepatitis C virus program. PTC is using GEMS in multiple ongoing drug discovery programs.

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PTC Therapeutics Inititates Phase 1A Clinical Trial of PTC299

SOUTH PLAINFIELD, N.J., May 12, 2006 — PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes, today announced that it had commenced a Phase 1a clinical trial of the company's product candidate PTC299 in healthy volunteers. The trial is being conducted in Belgium. PTC299 was discovered through PTC's proprietary Gene Expression Modulation by Small-molecules (GEMS) technology.

This Phase 1a trial is a single-site, randomized, double-blind, placebo-controlled escalating single-dose safety and pharmacokinetic study in healthy volunteers between the ages of 18 and 55. The primary objective of this trial is to determine a dose range for PTC299 that is well tolerated, achieves pharmacologically active plasma concentrations and is appropriate for use in a subsequent Phase 1b multiple-dose study. PTC expects to complete this trial in the second quarter of 2006. If this trial is successful, PTC plans to initiate further clinical trials of PTC299 in solid tumor cancer patients.

About PTC Therapeutics, Inc.
PTC is a biopharmaceutical company focused on the discovery and development 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 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.

About PTC299
PTC299 is a novel, orally administered small-molecule compound designed to inhibit the production of the protein vascular endothelial growth factor, or VEGF, in tumors. PTC has designed PTC299 to inhibit VEGF production in tumors by targeting the post-transcriptional control processes that regulate VEGF formation. Because PTC299 inhibits VEGF production, its action occurs at a different point in the VEGF pathway than other therapies that target the binding of VEGF to its receptors on the surface of blood vessel cells.

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PTC Therapeutics Announces Promotion of Cláudia Hirawat

SOUTH PLAINFIELD, N.J., April 21, 2006 — PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes, today announced the promotion of Cláudia Hirawat to Senior Vice President of Corporate Development.

"I am extremely pleased to announce this promotion. Cláudia has made significant contributions to our company, providing leadership across multiple areas including leading our efforts in establishing our business collaborations," commented Stuart Peltz, Ph.D., President and CEO of PTC Therapeutics, Inc.

Ms. Hirawat was most recently Vice President of Corporate Development at PTC. Her responsibilities focused on business development, public relations and advocacy group relations. In her new role as Senior Vice President of Corporate Development, Ms. Hirawat will focus on business development, advocacy group relations and commercial development.

Ms. Hirawat joined PTC in 2000 as the company's seventh employee. Prior to PTC, Ms. Hirawat was a Vice President at LedbetterStevens, a management consulting and senior-level retained search firm in New York focused exclusively in the biopharmaceutical area.

 

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PTC Therapeutics Announces Interim Phase 2 Results of PTC124 in Cystic Fibrosis

SOUTH PLAINFIELD, N.J., April 4, 2006 — PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes, today announced interim results from Phase 2 clinical trials of PTC124 in patients with cystic fibrosis (CF) due to a nonsense mutation. These interim data suggest that PTC124 may have pharmacological activity that addresses the underlying cause of CF in these patients.

PTC124 is an orally delivered investigational product candidate that PTC is developing for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are alterations in the genetic code that prematurely halt the translation process, producing a shortened, non-functional protein. Phase 2 clinical trials are ongoing in the two initial indications being pursued, CF and Duchenne muscular dystrophy (DMD) caused by nonsense mutations.

PTC is conducting two comparable Phase 2 clinical trials of PTC124 in CF, one at the Hadassah University Hospital in Jerusalem, Israel, and the other at four sites in the US (University of Alabama at Birmingham Hospital and Clinics, AL; Johns Hopkins Hospital, Baltimore, MD; Rainbow Babies' and Children's Hospital, Cleveland, OH; and Denver Children's Hospital, Denver, CO). In each study, patients receive two sequential two-week courses of treatment, first at a lower and then at a higher PTC124 dose level.

The primary endpoint of these trials is the change in the CFTR chloride channel activity (also known as chloride conductance). Chloride conductance is evaluated using a standardized nasal transepithelial potential difference (TEPD) procedure. Cystic fibrosis patients lack sufficient CFTR protein and therefore have an abnormal TEPD chloride conductance.

Fifteen patients have completed two cycles of treatment and data from these patients were available for inclusion in the interim analysis. Of these patients, three were from the U.S. trial and 12 were from the Israeli trial. All patients had a nonsense mutation and multiple signs and symptoms of CF, with most patients having lung dysfunction, chronic bacterial infection of the lungs, pancreatic insufficiency, and low body weights.

 

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PTC Therapeutics Files for Initial Public Offering

SOUTH PLAINFIELD, N.J., March 31, 2006 — PTC Therapeutics, Inc. today announced that the company has filed with the Securities and Exchange Commission a registration statement relating to the proposed initial public offering of shares of its common stock. All shares of the common stock to be sold in the offering will be sold by PTC Therapeutics, Inc.

Morgan Stanley & Co. Incorporated will act as sole book-runner and lead manager, J.P. Morgan Securities Inc. will act as co-lead manager and Pacific Growth Equities, LLC will act as co-manager in the offering.

A registration statement relating to these securities has been filed with the Securities and Exchange Commission but has not yet become effective. These securities may not be sold nor may offers to buy be accepted prior to the time the registration statement becomes effective. When available, a preliminary prospectus may be obtained from Morgan Stanley & Co. Incorporated, by emailing prospectus@morganstanley.com, or by contacting the prospectus department at Morgan Stanley & Co. Incorporated, 1585 Broadway, New York, New York 10036, by telephone at 212-761-6775.

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 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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PTC Therapeutics Receives Fast Track Designation for PTC124 in the Treatment of Duchenne Muscular Dystrophy

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).

"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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Schering-Plough and PTC Therapeutics Announce Collaboration for Development of PTC'S Preclinical Hepatitis C Compunds

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 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.

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 protein production. PTC discovers and develops small-molecule drugs that alter these processes by selectively modulating how RNA is used to produce proteins. This approach enables PTC to advance its drug discovery programs rapidly from targets to preclinical and clinical drug candidates, building a pipeline across multiple therapeutic areas including genetic disorders, oncology and infectious diseases.

Schering-Plough is a global science-based health care company with leading prescription, consumer and animal health products. Through internal research and collaborations with partners, Schering-Plough discovers, develops, manufactures and markets advanced drug therapies to meet important medical needs. Schering-Plough's vision is to earn the trust of the physicians, patients and customers served by its more than 32,000 people around the world. The company is based in Kenilworth, N.J., and its Web site is www.schering-plough.com.

SCHERING-PLOUGH DISCLOSURE NOTICE: The information in this press release includes certain "forward-looking statements" within the meaning of the Securities Litigation Reform Act of 1995, including statements relating to the company's strategy and the potential 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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PTC Therapeutics Initiates Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy


SOUTH PLAINFIELD, N.J., April 21, 2006 — PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes, today announced the promotion of Cláudia Hirawat to Senior Vice President of Corporate Development.

"The initiation of the Phase 2 study of PTC124 in DMD marks a wonderful milestone for PTC in its search for new treatments for DMD," commented Stuart W. Peltz, Ph.D., President and CEO of PTC, adding, "We share the gratification of this achievement with patients, their families, patient advocacy groups, and investigators who have supported and helped guide our efforts. We hope that information gained from this study will support the further development of PTC124, and complement our efforts to discover and develop additional therapies for boys with DMD."

The Phase 2 clinical study is enrolling patients who have DMD due to the presence of a nonsense mutation in the dystrophin gene. The primary endpoint of this Phase 2 multi-site, open-label, dose-ranging clinical study is assessment of muscle dystrophin expression in response to treatment with PTC124. Other assessments include the presence of dystrophin mRNA and dystrophin-related proteins, muscle function, compliance with treatment, safety, and pharmacokinetics.

"With the initiation of the Phase 2 study, we hope to establish proof-of-concept for PTC124 in nonsense-mutation-mediated DMD," said Langdon Miller, M.D., Chief Medical Officer at PTC. "Building on the positive preclinical data with PTC124 that Doctors Lee Sweeney and Elisabeth Barton have generated in their laboratories at the University of Pennsylvania, we have worked closely with scientists and investigators to design a study that can examine whether the encouraging preclinical findings with PTC124 translate to the clinical setting. Demonstration of pharmacological activity in this study would be an important step toward evaluating the longer-term clinical benefits of PTC124."

Dr. Valerie Cwik, Medical Director of the Muscular Dystrophy Association (MDA), commented: "The start of the PTC124 study opens a new era in clinical trials for DMD, by testing a drug specifically aimed at overcoming the genetic defect that causes the disease. We at MDA are very excited that this potential treatment is now in clinical trials for DMD."

PTC has commenced recruitment for the Phase 2 study in DMD at the Children's Hospital of Philadelphia in Philadelphia, PA, the Cincinnati Children's Hospital Medical Center in Cincinnati, OH and the University of Utah in Salt Lake City, UT. More details regarding the design and conduct of this study are available at www.clinicaltrials.gov.

"Given the lack of effective targeted treatment for DMD, there is tremendous interest in the development of PTC124 within the DMD patient and scientific communities," commented Dr. Richard Finkel, Director, Neuromuscular Program, Children's Hospital of Philadelphia. "We are excited by the results in the animal model and are delighted to collaborate with PTC and the other investigational sites in designing and conducting this important study in boys with DMD."

PTC also has Phase 2 clinical trials for PTC124 underway with CF patients in the United States and in Israel. More information regarding these trials can be found at www.clinicaltrials.gov.

 

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