Santhera Reports Encouraging, First Data from Phase IIa Clinical Trial with SNT-MC17 in Duchenne Muscular Dystrophy
Santhera Pharmaceuticals announced positive, first results from a 12 month Phase IIa clinical trial with SNT-MC17 (INN: idebenone) in Duchenne muscular dystrophy (DMD) as measured by cardiac and respiratory parameters. Santhera therefore is committed to further clinical development of SNT-MC17 in DMD.
This exploratory Phase IIa trial was a 12-month double-blind, randomized, placebo-controlled study conducted at the University of Leuven, Belgium. In total 21 DMD patients between the age of 8 and 16 years were enrolled to assess the efficacy and tolerability of one dose level of SNT-MC17 (450 mg/day) compared to placebo. Thirteen patients were receiving SNT-MC17 while 8 patients were randomized to the placebo group. There were no drop-outs in the study and the compliance was very good. Importantly, there was no difference in the safety and tolerability of SNT-MC17 compared to placebo underlining again the excellent safety profile of SNT-MC17 also in this pediatric population.
The primary objective was to assess whether SNT-MC17 improves or slows the decline in cardiac function in DMD patients, applying a comprehensive echocardiographic approach that included cardiac tissue Doppler and strain rate imaging technology. The primary endpoint was an assessment of the change in contractility of the region of the heart muscle that is affected early and most severely in DMD patients, measured by the peak systolic radial strain of the left ventricular inferolateral wall. After treatment for twelve months with SNT-MC17, DMD patients showed a trend to improve on this functional cardiac parameter compared to placebo (P=0.098).
In addition to these data, patients on SNT-MC17 improved also on certain respiratory parameters. Most striking and statistically significant was the improvement of DMD patients' lung function measured by peak flow (P=0.042). Patients treated with SNT-MC17 ameliorated on this parameter, while patients on placebo deteriorated over the study period.
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