PTC Therapeutics, Inc. has initiated a global confirmatory Phase III clinical trial of Translarna (ataluren), an investigational new drug, in patients with nonsense mutation cystic fibrosis (nmCF). Nonsense mutations within cystic fibrosis are categorized as Class I mutations, a severe form of CF that results in little or no production of the CFTR protein. The Phase III confirmatory trial is referred to as ACT CF (ataluren confirmatory trial in cystic fibrosis) and the primary endpoint is lung function as measured by relative change in percent predicted forced expiratory volume in one second, or FEV1.
"We believe the data from our previous 238-patient Phase III clinical trial in nonsense mutation cystic fibrosis patients demonstrated that Translarna had a positive benefit on lung function versus placebo, particularly in patients not receiving chronic inhaled tobramycin. The ACT CF trial is designed to confirm Translarna's efficacy based on the evidence seen in the previous Phase III study and other earlier work. By focusing ACT CF on the patient population that can most readily demonstrate the effect of Translarna, we believe we have optimized our opportunity for a successful trial," stated Robert Spiegel, MD, FACP, Chief Medical Officer of PTC Therapeutics. "We look forward to completing ACT CF and ultimately bringing this potential first-in-class treatment to those CF patients who may benefit."
"The CF community is excited by the start of this pivotal study in cystic fibrosis," stated Michael Konstan, MD, Pediatric Department Chair at University Hospitals Rainbow Babies & Children's Hospital and Professor of Pediatrics at Case Western Reserve University School of Medicine. "Current treatments for nonsense mutation cystic fibrosis focus on alleviating symptoms and reducing infections, whereas Translarna targets the underlying cause of disease. There is a significant need for better treatment options for patients with this severe form of the disease, and we believe Translarna has the potential to provide this benefit."