Santhera's MICONOS Trial with Catena/Sovrima in Friedreich's Ataxia Misses Primary Endpoint
Santhera Pharmaceuticals announced that its MICONOS Phase III study evaluating Catena®/Sovrima® for the treatment of Friedreich's ataxia missed its primary endpoint. Trends towards improvement in the key neurological endpoint were however identified by a meta-analysis of all Santhera's Phase II and III studies in the same indication. MICONOS also confirmed that Catena®/Sovrima® is safe and well tolerated at doses of up to 2250 mg/day.
The 12-month MICONOS (Mitochondrial Protection with Idebenone In Cardiac Or Neurological Outcome Study) study enrolled 232 primarily adult patients and evaluated the safety and efficacy of three doses of Catena®/Sovrima® compared to that of placebo. Analysis of the primary endpoint of the study, mean change in the International Cooperative Ataxia Rating Scale (ICARS) score from baseline, did not detect any significant difference between the active dose arms and placebo. Secondary endpoints, including the proportion of patients improving on ICARS score (responder analysis) and change in the Friedreich's Ataxia Rating Scale also did not show statistically significant differences between the placebo and active dose groups. Although a detailed analysis of cardiac endpoints is still ongoing, there was no difference between the active and placebo groups in the key cardio logical secondary endpoint assessing a combination of anatomical and functional cardiac parameters.
A meta-analysis of Santhera's three Phase II and III studies including 344 patients of all age groups and disease stages showed trends for improvement on Catena®/Sovrima® in the mean change in ICARS score in the combined mid- and high dose groups compared to placebo (p=0.083) as well as in the high dose group compared to placebo (p=0.088). Similarly, a larger proportion of patients improved by at least 2.5 ICARS points over a six months treatment period in the Catena®/Sovrima dose groups (placebo: 30.4%; mid-dose group 39.1%; high dose group 41.9%) and comparison with placebo showed a trend in favor of the combined mid and high dose groups (p=0.10) and the high dose group (p=0.098).
The pharmacokinetic analyses of study participants revealed detectable levels of idebenone or its metabolites in the blood of 12% of the patients in the placebo arm. With the exception of one, none of these individuals declared prior use of idebenone before joining the MICONOS study.
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