The Phase I trial of IONIS-ENAC-2.5Rx for cystic fibrosis represents the first time an antisense medicine delivered directly to the lung has shown a significant reduction in ENaC messenger RNA levels.

Switzerland’s Santhera is to restructure its business and seek extra funding after the failure of Puldysa (idebenone) in a Phase III Duchenne muscular dystrophy study at interim analysis.

The company has no development programs left in its R&D pipeline and will focus entirely on the commercialization of IBS drug Linzess while seeking late-stage assets from external sources.

Officials from the US Food and Drug Administration cited their reasons for refusing to review new applications for companies with unresolved data integrity failures.