Company confident larger-scale European pivotal trial will come up trumps.

Santhera Pharmaceuticals is not giving up on its lead Friedreich’s Ataxia therapy, Catena® (idebenone), even though the drug 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.

The Swiss-based company said it believed the ongoing European Phase III trial, MICONOS, which has a larger, predominantly adult patient population and is of longer duration, will show the efficacy data needed for regulatory approval to be achieved in the U.S. and Europe. While Santhera admitted it was “tremendously disappointed” by the IONIA data, CEO Klaus Schollmeier, Ph.D., says, “The results have not dampened our confidence in the drug’s potential against Friedreich’s Ataxia.”

Thomas Meier, Ph.D., Santhera CSO, adds, “We remain confident in our development programs with Catena in Friedreich’s Ataxia and other neuromuscular and mitochondrial disorders, and look forward to the upcoming data from several ongoing clinical trials”.

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, Santhera points out. 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.

“I still strongly support the disease-modifying effect of Catena in Friedreich’s Ataxia,” commented lead IONIA study investigator Professor Sue Perlman, clinical professor of neurology at the University of California, Los Angeles. “I strongly recommend that patients continue in the open-label extension study arm of the Phase III IONIA trial to enable us to gather as much longer-term data as possible.”

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