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 2250mg/day.
Santhera Pharmaceuticals said that the 12-month MICONOS 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 difference between the active dose arms and placebo.
Santhera Pharmaceuticals added that the 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 statistical 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 cardio logical secondary endpoint assessing a combination of anatomical and functional cardiac parameters.
Reportedly, 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 as well as in the high dose group compared to placebo.
MICONOS study revealed that 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 and comparison with placebo showed a trend in favor of the combined mid and high dose groups and the high dose group.
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.