Proteostasis Therapeutics have identified multiple compounds that improve the folding and trafficking of F508del. The in vitro data demonstrates “improved single-agent activity versus agents currently in development as well as significant synergies with these agents, such as VX-809.” Slide 14 below shows that VX809 and one of the compounds from Proteostasis (PTI-PR1) have a similar level of activity with F508del cells. It also shows that when the compounds from Proteostasis are added to VX809, the CFTR function increases further, reaching about 40% of normal function.
Proteostasis Therapeutics Presents Preclinical Data at EMBO Conference (May 2013):
The following information is from the May press release: “Our CF candidates can correct the folding/trafficking deficits of the major mutant protein in cystic fibrosis, ΔF508 CFTR,” stated Peter Reinhart, Ph.D., President and Chief Scientific Officer of Proteostasis Therapeutics. “We continue to be very encouraged by these preclinical results and will be transitioning both programs to lead optimization this year and advancing them into clinical studies by 2015.” “