Proteostasis Therapeutics (PTI): Breakthrough Therapy Designation for PTI-428 in Cystic Fibrosis

 

Proteostasis Therapeutics announces that the FDA has granted Breakthrough Therapy Designation for PTI-428, the Co's cystic fibrosis transmembrane conductance regulator (CFTR) amplifier
FDA granted the Breakthrough Therapy Designation for PTI-428 for the treatment of CF in homozygous patients for the F508del mutation who are receiving Orkambi as background therapy. The designation was based on the results from a recent Phase 2, randomized, placebo controlled study of PTI-428, in 24 CF subjects on background treatment with Orkambi and who were treated with either 50 mg PTI-428 once daily or placebo for 28 consecutive days. The study results showed that treatment with PTI-428 led to mean absolute improvement in percent predicted forced expiratory volume in 1 second (ppFEV1) of 5.2 percentage points.