Idebenone failed to show a significant difference when compared to a placebo in Friedreich's ataxia
On May 19, 2009, Santhera Pharmaceuticals reported that a phase 3 trial of its idebenone compound Catena showed the drug was not associated with a statistically significant benefit in 70 children between 8 and 17 years old with Friedreich's ataxia who took it for six months. (See the company's press release, Santhera's US Phase III IONIA Trial in Friedreich's Ataxia Misses Primary Endpoint.)
The drug appeared safe and well tolerated at doses up to 2,250 milligrams per day.
Santhera's press release says there's an ongoing phase 3 trial of Catena now being conducted in Europe that has a different design from the U.S. study and for which results are expected in 2010.
The European trial is a year long, and it includes 232 predominantly adult patients. Santhera says if the results of this European trial are positive, they will form the basis of filings for regulatory approval in the United States and Europe.
Idebenone is believed to improve energy production in cellular structures called mitochondria. Earlier studies showed the drug was 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.
Santhera's press release quotes Sue Perlman, clinical professor of neurology at the University of California-Los Angeles and one of the two study investigators on the U.S. trial, as saying, "I still strongly support the disease-modifying effect of Catena in Friedreich's ataxia. I believe it slows the progression of the neurological and cardiac aspects of this condition over time."
She strongly recommended that patients continue to take the drug in the open-label extension study so that the investigators can gather as much longer-term data as possible.