Santhera publishes results of DMD trial

Santhera Pharmaceuticals, a Swiss specialty drug company focused on neuromuscular diseases, has published study results that support the potential efficacy of its lead compound SNT-MC17 (idebenone) in Duchenne Muscular Dystrophy in the European Heart Journal.

The article 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 findings of the study are that presymptomatic-initiated and long-term idebenone treatment: significantly prevented cardiac diastolic dysfunction; preserved cardiac systolic contractile reserve and, thus, blocking 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.

The company is in scientific discussions with clinical experts and health authorities from the European Union and the USA in preparation for a Phase III development program, the initiation of which is planned for the first half of 2009. Santhera's recent Phase II proof-of-concept trial with the drug in a population of young DMD patients showed positive efficacy on cardiac and respiratory functions. The firm will present the data at the upcoming congress of the World Muscle Society in Newcastle, UK.