Positive Phase III results for Italfarmaco's givinostat in DMD

Italy’s privately-held Italfarmaco Group has released positive top-line data from its completed Phase III EPIDYS trial with givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor, in boys with Duchenne muscular dystrophy (DMD).

The primary objective of the study was to evaluate the effects of givinostat on slowing disease progression in ambulant DMD boys aged six years and above on chronic steroids.

The study compared givinostat to placebo and met the primary endpoint (change from baseline in the time to climb four stairs) following 18 months of treatment in the target population with key secondary endpoints consistent with the functional primary endpoint. Givinostat continued to demonstrate a tolerability profile in line with previous studies. The top-line data were presented by Italfarmaco’s chief medical officer, Dr Paolo Bettica, on June 25, 2022, at the hybrid  Parent Project Muscular Dystrophy (PPMD) Annual Conference.