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Santhera announces clinical trial results
Swiss-based Santhera Pharmaceuticals (www.santhera.com) and Prof Gunnar Buyse, principal investigator of the study at the University of Leuven, recently presented first indications of clinical efficacy of SNT-MC17/idebenone on functional cardiac and respiratory parameters in Duchenne Muscular Dystrophy (DMD). The results of this Phase II trial are particularly encouraging since SNT-MC17 may protect from the two potentially life-threatening complications of this disease. Safety and tolerability of SNT-MC17 was not different than placebo underlining the excellent safety profile of the compound.
During the 52-week treatment period, patients on SNT-MC17 improved on the peak systolic radial strain of the left ventricular (LV) inferolateral cardiac wall, the region of the heart that is most severely affected in patients suffering from DMD. Expressed as percent change from baseline, the patient group treated with SNT-MC17 improved by 104% which was significantly different from placebo treated patients who improved only by 29% (p=0.03). In addition, peak systolic longitudinal strain of the LV lateral-mid cardiac region also improved significantly, indicating a beneficial effect of SNT-MC17 on early and systolic myocardial dysfunction in DMD.
Significant improvements were also observed in respiratory function. Direct measures of respiratory weakness (peak expiratory flow, maximal inspiratory pressure) improved in patients on SNT-MC17, indicating efficacy of SNT-MC17 to improve early signs of respiratory weakness and insufficiency. For example, peak flow expressed as percentage of the predicted value improved by 2.8% for patients on SNT-MC17 while patients on placebo deteriorated by 8.5% (p=0.042).
The data of this double-blind placebo-controlled Phase II trial were presented at the recent American Academy of Neurology 2008 Annual Meeting in Chicago. Santhera is in the process to prepare for a Phase III trial in Europe and the US.