Study Highlights Potential of SNT-MC17/idebenone in Duchenne Muscular Dystrophy
News Sep 25, 2008
The authors concluded that early-initiated and long-term treatment of mdx mice, a well-established animal model for Duchenne Muscular Dystrophy, is cardioprotective and improves exercise performance. Santhera's recent Phase II proof-of-concept trial with SNT-MC17/idebenone in a population of young Duchenne Muscular Dystrophy patients showed positive efficacy on cardiac and respiratory functions. Meanwhile, the Company is in scientific discussions with clinical experts and health authorities from the European Union and the United States of America in preparation for a Phase III development program, the initiation of which is planned for the first half of 2009. Lead investigator, Prof. Gunnar Buyse (University Leuven, Belgium), will present data from the preclinical and clinical studies with SNT-MC17/idebenone in Duchenne Muscular Dystrophy at the upcoming 13th Congress of the World Muscle Society (September 29 to October 2 in Newcastle, UK).
The publication in the European Heart Journal reports on the results of a very long-term, blinded and placebo-controlled study in the dystrophin deficient mdx mouse, a disease relevant animal model. The main finding of the study is that presymptomatic-initiated and long-term idebenone treatment significantly prevented cardiac diastolic dysfunction, preserved cardiac systolic contractile reserve and, as such blocked the development of lethal acute heart failure during a dobutamine-mediated stress protocol, reduced cardiac inflammation and fibrosis, and improved voluntary running performance in the mdx mouse model.
In the authors' opinion the beneficial effects of idebenone can be explained by its ability to improve mitochondrial respiratory chain function and to reduce oxidative stress, pathways that have been implicated in the pathophysiology of dystrophin deficient muscular dystrophy. "We have identified a novel potential therapeutic strategy for human Duchenne Muscular Dystrophy, as SNT-MC17/ idebenone was cardioprotective and improved exercise performance in the dystrophin-deficient mdx mouse", the authors concluded.
Thomas Meier, Santhera's Chief Scientific Officer, commenting on the European Heart Journal publication said: "Duchenne Muscular Dystrophy is a severe and still incurable disease. Heart failure is responsible for early death of a significant fraction of Duchenne Muscular Dystrophy patients. The reported cardioprotective data are in line with the evidence collected in our Phase II proof-of-concept trial and support SNT-MC17/idebenone as potential treatment option of Duchenne Muscular Dystrophy."
Original article: Buyse et al. Long-term blinded placebo-controlled study of SNT-MC17/ idebenone in the dystrophin deficient mdx mouse: cardiac protection and improved exercise performance. European Heart Journal (doi:10.1093/eurheartj/ehn406); Epub ahead of print 2008 Sep 10.