Santhera Pharmaceuticals reported that its lead Friedreich’s ataxia therapy, Catena® (idebenone) failed to meet its primary endpoint in a U.S. Phase III trial (the IONIA study), which involved patients aged between eight and 17 years. Nevertheless, the drug appeared safe and well tolerated at doses up to 2.250 mg per day. Idebenone is believed to improve energy production in mitochondria. Earlier studies showed the drug to be safe and well tolerated in Friedreich's ataxia and that there was a statistical trend toward dose-related improvement in neurological function associated with its use. The six-month IONIA trial missed both its primary endpoint, as measured by the International Co-operative Ataxia Rating Scale, and its secondary neurological endpoint, the Friedreich’s Ataxia Rating scale. However, the active treatment arms did show consistent improvements over baseline and placebo. The company put the lack of statistical significance down to both lower than expected improvements in Catena-treated patients, when compared with the previous U.S. Phase II trial (NICOSIA), and the fact that patients on placebo also deteriorated less than expected. The NICOSIA trial data were used to design IONIA. An ongoing Phase III trial of Catena is now being conducted in Europe. This trial has a different design from the U.S. study and results are expected in 2010. The European trial is a year long, and includes 232 predominantly adult patients.

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