SOUTH PLAINFIELD, NJ, USA I November 19, 2015 I PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that it has completed enrollment of ACT CF, the company’s second Phase 3 clinical trial of Translarna™ (ataluren) for patients with nonsense mutation cystic fibrosis (nmCF). Nonsense mutations in cystic fibrosis are categorized as Class I mutations, which are the most difficult to treat, as they result in little or no production of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Approximately 10 percent of cystic fibrosis patients have their disease as a result of a nonsense mutation.
“We are very pleased by the enthusiastic response from our clinical investigators and strong interest from patients to participate in our ACT CF study,” said Stuart Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. “Based on the results from our previous Phase 3 study, we believe that Translarna has the potential to meaningfully improve lung function and decrease pulmonary exacerbations in nonsense mutation cystic fibrosis patients by targeting the underlying cause of the disease.”
On September 30, 2015, the European Medicines Agency (EMA) validated the submission of a variation for a new indication for Translarna for the treatment of nmCF. The company’s regulatory application for Translarna in nmCF was based on clinical data and analyses generated from the company’s previously completed Phase 3 double-blind, placebo-controlled study comparing Translarna to placebo in nmCF patients. Translarna received marketing authorization in Europe in August 2014 for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.
About ACT CF
ACT CF is a 48-week placebo controlled Phase 3 clinical trial designed to evaluate the effect of Translarna in patients six years of age or older with nmCF not receiving chronic inhaled aminoglycosides. The primary endpoint is lung function as measured by relative change in percent predicted FEV1. The study has reached full enrollment across 88 sites globally. Patients in the trial are eligible to participate in an open-label extension study, which has already begun enrolling patients who have completed the initial 48 weeks of treatment.
About Translarna™ (ataluren)
Translarna, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Translarna is an investigational new drug in the United States. The development of Translarna has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA’s Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.
The FDA and the European Commission have granted Translarna Orphan Drug status for the following indications: Duchenne muscular dystrophy, cystic fibrosis, Mucopolysaccharidosis I (MPS 1), and aniridia.
Translarna is an oral protein restoration therapy that has the potential to benefit patients with genetic disorders caused by a nonsense mutation. On average, 11% of every monogenic disorder is caused by a nonsense mutation. PTC’s strategy is to expand the clinical development of Translarna across multiple genetic disorders to deliver on the company’s commitment to address rare and neglected disorders.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare disabling and life-threatening genetic disorder resulting from mutations that cause the lack or dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR), a chloride ion channel function responsible for fluid transport in the lung, pancreas and other organs. In patients who have cystic fibrosis due to a nonsense mutation, an interruption in the genetic code prematurely halts the synthesis of CFTR, causing the protein to be short and non-functioning. Nonsense mutations are categorized as Class I mutations that result in little or no production of the CFTR protein, which makes people with these mutations very difficult to treat. CF patients with Class I mutations typically experience more severe disease symptoms than those with other genotypes, including a shorter life span, a higher probability of end-stage lung disease, and a higher prevalence of pancreatic insufficiency. Approximately 10% of patients have CF due to a Class I nonsense mutation in at least one allele of the CFTR gene. Available therapies for treatment of lung manifestations of CF, such as inhaled antibiotics do not address the underlying defect. There are no marketed treatments that target the defect associated with CF caused by nonsense mutations.
About PTC Therapeutics, Inc.
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology we refer to as post-transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA, or mRNA, molecule is copied from DNA through the transcription process. PTC’s internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. For more information on the company, please visit our website www.ptcbio.com
SOURCE: PTC Therapeutics
Post Views: 144
SOUTH PLAINFIELD, NJ, USA I November 19, 2015 I PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that it has completed enrollment of ACT CF, the company’s second Phase 3 clinical trial of Translarna™ (ataluren) for patients with nonsense mutation cystic fibrosis (nmCF). Nonsense mutations in cystic fibrosis are categorized as Class I mutations, which are the most difficult to treat, as they result in little or no production of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Approximately 10 percent of cystic fibrosis patients have their disease as a result of a nonsense mutation.
“We are very pleased by the enthusiastic response from our clinical investigators and strong interest from patients to participate in our ACT CF study,” said Stuart Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. “Based on the results from our previous Phase 3 study, we believe that Translarna has the potential to meaningfully improve lung function and decrease pulmonary exacerbations in nonsense mutation cystic fibrosis patients by targeting the underlying cause of the disease.”
On September 30, 2015, the European Medicines Agency (EMA) validated the submission of a variation for a new indication for Translarna for the treatment of nmCF. The company’s regulatory application for Translarna in nmCF was based on clinical data and analyses generated from the company’s previously completed Phase 3 double-blind, placebo-controlled study comparing Translarna to placebo in nmCF patients. Translarna received marketing authorization in Europe in August 2014 for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.
About ACT CF
ACT CF is a 48-week placebo controlled Phase 3 clinical trial designed to evaluate the effect of Translarna in patients six years of age or older with nmCF not receiving chronic inhaled aminoglycosides. The primary endpoint is lung function as measured by relative change in percent predicted FEV1. The study has reached full enrollment across 88 sites globally. Patients in the trial are eligible to participate in an open-label extension study, which has already begun enrolling patients who have completed the initial 48 weeks of treatment.
About Translarna™ (ataluren)
Translarna, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Translarna is an investigational new drug in the United States. The development of Translarna has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA’s Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.
The FDA and the European Commission have granted Translarna Orphan Drug status for the following indications: Duchenne muscular dystrophy, cystic fibrosis, Mucopolysaccharidosis I (MPS 1), and aniridia.
Translarna is an oral protein restoration therapy that has the potential to benefit patients with genetic disorders caused by a nonsense mutation. On average, 11% of every monogenic disorder is caused by a nonsense mutation. PTC’s strategy is to expand the clinical development of Translarna across multiple genetic disorders to deliver on the company’s commitment to address rare and neglected disorders.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare disabling and life-threatening genetic disorder resulting from mutations that cause the lack or dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR), a chloride ion channel function responsible for fluid transport in the lung, pancreas and other organs. In patients who have cystic fibrosis due to a nonsense mutation, an interruption in the genetic code prematurely halts the synthesis of CFTR, causing the protein to be short and non-functioning. Nonsense mutations are categorized as Class I mutations that result in little or no production of the CFTR protein, which makes people with these mutations very difficult to treat. CF patients with Class I mutations typically experience more severe disease symptoms than those with other genotypes, including a shorter life span, a higher probability of end-stage lung disease, and a higher prevalence of pancreatic insufficiency. Approximately 10% of patients have CF due to a Class I nonsense mutation in at least one allele of the CFTR gene. Available therapies for treatment of lung manifestations of CF, such as inhaled antibiotics do not address the underlying defect. There are no marketed treatments that target the defect associated with CF caused by nonsense mutations.
About PTC Therapeutics, Inc.
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology we refer to as post-transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA, or mRNA, molecule is copied from DNA through the transcription process. PTC’s internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. For more information on the company, please visit our website www.ptcbio.com
SOURCE: PTC Therapeutics
Post Views: 144
