Company hopes European Phase III results will uphold drug benefits despite failure of U.S. pivotal trial.
Santhera Pharmaceuticals initiated a named patient program (NPP) in Europe and Switzerland for its Friedrich’s ataxia drug, Catena®. The NPP in European and Switzerland means that physicians will be able to prescribe the drug for qualifying patients ahead of its approval in the relevant countries.
The therapy, a synthetic analogue of ubiquinone, was approved in Canada for this indication in July 2008. In May 2009, however, Santhera reported that Catena had missed both its primary and secondary endpoints in a U.S. Phase III trial with the drug against Friedreich’s Ataxia despite sailing through its Phase II trial. As a consequence of the IONIA study data, the application for marketing approval filed earlier in Switzerland was withdrawn.
The company put the failure down to larger than expected placebo response rates. It is now awaiting results from the ongoing European Phase III MICONOS study, which has a larger patient population and longer study duration. Results are expected in the first half of 2010. If positive, Santhera says they will form the basis of NDA and MAA filings.
Catena is in development for a number of indications. Phase III Duchenne muscular dystrophy studies are being planned, and a Phase IIa evaluation in Leber’s hereditary optic neuropathy is also being prepared.
During June this year Santhera signed a collaboration with Columbia University in New York, centered on investigating Catena in a Phase II study as a treatment for MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes). A separate partnership was inked in June with the NIH for a Phase I/II trial in primary progressive multiple sclerosis (PPMS).
