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NIH Completes Collaborative Phase II Study with Santhera's compound SNT-MC17/idebenone in Friedreich's Ataxia (FRDA)

09 Oct 2006 - The US National Institute of Neurological Disorders and Stroke (NINDS) at the National Institutes of Health (NIH) and Santhera Pharmaceuticals completed their six month double-blind placebo-controlled Phase II clinical trial with SNT-MC17/idebenone in Friedreich's Ataxia (FRDA). This trial was primarily designed to establish additional safety data in young patients with FRDA, particularly at higher doses, and to study the potential efficacy of SNT-MC17/idebenone on neurological symptoms and findings in this disease.
 
The trial enrolled a total of 48 patients, with 12 patients in each of three dose groups and a placebo group. Data from this trial support the favourable safety profile of the compound. Preliminary analysis of the trial results suggests that SNT-MC17/idebenone has a trend toward dose proportional improvement in neurological parameters in FRDA patients as determined by the International Cooperative Ataxia Rating Scale (ICARS), a semi-quantitative scale based upon 19 test parameters that assess sensory and motor skills.
 
These new data reinforce Santhera's belief that SNT-MC17/idebenone has the potential to provide a possible treatment for this disease.