Italfarmaco Presents New Givinostat Data on Preserving Upper Limb Function in Duchenne Muscular Dystrophy

Italfarmaco S.p.A. announced new long-term data from the ongoing Phase 3 EPIDYS open-label extension (OLE) study, which suggests that givinostat treatment may help preserve upper limb function in boys with Duchenne muscular dystrophy (DMD). The findings were presented at the 31st Annual International Congress of the World Muscle Society (WMS).

The study compared the upper limb function of givinostat-treated patients with counterfactual trajectories estimated from an external natural history-based model representing predicted disease progression in the absence of treatment, using the Performance of Upper Limb 2.0 (PUL 2.0) assessment. Upper limb function is becoming increasingly important as DMD advances and loss of ambulation occurs.

The analysis involved 115 patients who were evaluable for a 12-month analysis. In this cohort, the observed mean change in PUL 2.0 score was, 0.78, compared with an average predicted decline of, 4.57 (a difference of 3.79; nominal p<0.001) based on the model developed using natural history data. These findings suggest that patients receiving givinostat experienced a slower average decline in upper limb function than predicted based on counterfactual trajectories.

Additionally, in the cohort evaluable at 24 months (n=96), the observed mean change in PUL 2.0 score was, 2.26, compared with an average predicted decline of, 8.55 (a difference of 6.29; nominal p<0.001). These results further expand the growing body of evidence supporting the potential long-term clinical benefits of givinostat across multiple measures of disease progression.

Italfarmaco, founded in 1938 in Milan, Italy, is a private global pharmaceutical company with operations in more than 90 countries through directly controlled or affiliated companies. The company is a leader in pharmaceutical research, product development, production, and commercialization with a focus on rare diseases, including Duchenne muscular dystrophy.

The EPIDYS Phase 3 study is a randomized, placebo-controlled clinical trial designed to evaluate the efficacy and safety of givinostat in patients with DMD. MRI-based assessments of muscle composition were included as exploratory endpoints to better characterize disease progression. The ongoing open-label extension (OLE) study is intended to provide additional information on longer-term outcomes in patients previously enrolled in clinical trials.

Givinostat, an orally administered histone deacetylase (HDAC) inhibitor, was discovered through Italfarmaco’s research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy). By regulating the excessive HDAC activity characteristic of DMD muscles, it helps restore the expression of key genes and biological processes essential for muscle maintenance and repair.