Phase III EPIDYS trial with ITF 2357 meets primary endpoint in Duchenne Muscular Dystrophy.- Italfarmaco

The primary objective of the study was to evaluate the effects of Givinostat on slowing disease progression in ambulant DMD boys aged 6 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 4 stairs) following 18 months of treatment in the target population1 with key secondary endpoints consistent with the functional primary endpoint.

Givinostat continued to demonstrate a tolerability profile in line with previous studies. Primary Endpoint: The mean change from baseline to climb 4 stairs was selected as the primary endpoint to assess the efficacy of Givinostat compared to placebo. The results demonstrated a slower decline to perform this functional task in the Givinostat-treated group (difference vs Placebo of 1.78 seconds, p=0.0345).

Key Secondary Endpoints: A variety of secondary endpoints were analyzed that showed results consistent with the functional primary endpoint. These included functional tests such as the North Star Ambulatory Assessment (NSAA) and the time to rise (TTR) test along with muscle strength analyses. Fat infiltration in the vastus lateralis (VL) muscle of the thigh is a characteristic of disease progression in DMD patients and was measured using a non-invasive objective imaging method called Magnetic Resonance Spectroscopy (MRS) to assess the efficacy of Givinostat. The data indicated that treatment with Givinostat delayed fat infiltration by approximately 30% (difference vs Placebo, -2.9%, nominal p=0.035). Additional exploratory endpoints were also analyzed in the clinical study.

These results further support the potential of Givinostat in providing a clinical benefit for boys with DMD. Safety and Tolerability: The majority of Adverse Events (AE) were mild to moderate in severity (95%). Three (2.5%) boys treated with Givinostat withdrew from the trial because of an AE.The topline data were presented by Italfarmaco Group’s Chief Medical Officer, Dr. Paolo Bettica on June 25, 2022, at the hybrid Parent Project Muscular Dystrophy (PPMD) Annual Conference.