Proteostasis Therapeutics, Inc., announced that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for PTI-428, an investigational oral treatment for cystic fibrosis (CF).
PTI-428 is a novel and orally bioavailable Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator belonging to the amplifier class. Amplifiers are CFTR modulators that selectively increase the amount of an immature form of CFTR protein, thereby providing additional substrate for other CFTR modulators, such as correctors or potentiators, to act upon.
CF is a progressive disease caused by mutations in the gene that encodes CFTR resulting in disrupted ion flow which leads to the buildup of thick mucus in several organs including the lungs, and the vast majority of patients die of respiratory failure.
The Company recently received authorization from the FDA to start first-in-human studies in CF patients which will be initiated in the first quarter of 2016. The goal of the Phase 1 clinical trial is to study safety, pharmacokinetics and preliminary pharmacodynamics and the final report is expected by the end of 2016.
