PTC Begins Phase III Study For Translarna In nmCF

Biopharmaceutical company PTC Therapeutics announced that it has launched the global confirmatory Phase III trial investigating Translarna (ataluren) in patients with nonsense mutation cystic fibrosis (nmCF).

Translarna is an investigational protein restoration therapy designed to allow the formation of a functioning protein in patients affected with nonsense mutation-caused genetic disorders. A nonsense mutation occurs when a change in the genetic code prematurely stops the synthesis of an essential protein. The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) granted Translarna a positive recommendation for conditional approval in nonsense mutation Duchenne muscular dystrophy in May 2014.

The randomized, placebo controlled, international, double-blind ACT CF (ataluren confirmatory trial in cystic fibrosis) Phase III trial to measure efficacy and safety of ataluren in patients 6 years old and above with nmCF not receiving chronic inhaled aminoglycosides. Primary endpoint of the trial is lung function as measured by relative change in percent predicted forced expiratory volume (FEV). The company said the trial will enroll 208 patients.

Robert Spiegel, CMO of PTC Therapeutics, said, “We believe the data from our previous 238-patient Phase 3 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 3 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.”

Nonsense mutation cystic fibrosis is a severe form of cystic fibrosis that leads to insufficient or zero production of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Patients with Class I mutations in CF are afflicted with severe symptoms including shorter life spans, a higher prevalence of pancreatic insufficiency, and a greater possibility of end-stage lung disease.

Spiegel stated that the company anticipates the completion of the ACT CF trial and the drug’s progress to further development and eventual commercialization for CF patients.