SANTHERA STARTS MUSCULAR DYSTROPHY TRIAL

Santhera Pharmaceuticals has started a Phase IIa clinical study with its lead product SNT-MC17 (idebenone) to evaluate its benefits in the treatment of Duchenne muscular dystrophy (DMD). DMD is the most common and devastating type of muscular dystrophy, which causes weakness and muscle wasting in young boys. This is a new potential indication for SNT-MC17, which is expected to enter Phase III clinical trials in Europe later this year for Friedreich's Ataxia, another life-threatening neuromuscular disease.

The study is a double-blind, randomized, placebo-controlled trial that aims to assess the efficacy of SNT-MC17 in 10- to 16-year-old males with cardiac dysfunction associated with DMD. The primary endpoint is to evaluate cardiac function improvement in DMD patients after one year of treatment. The effect of SNT-MC17 on muscle strength in the limbs and respiratory muscles will also be assessed as secondary endpoints. The study will take place at the University of Leuven in Belgium, and will enroll a total of 21 patients.