Experimental pill for rare lung and liver disease hits snag: trial stopped early
Disease control
Terminated
This study tested an experimental drug called VX-864 in 14 people with a severe form of alpha-1 antitrypsin deficiency (PiZZ genotype). The goal was to see if the pill could raise protective protein levels in the blood and reduce harmful buildup in the liver. The trial was termin…
Phase: PHASE2 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 26, 2026 15:04 UTC