Santhera initiates Pivotal Phase III Study with Catena/Sovrima (Idebenone) in Duchenne Muscular Dystrophy
Santhera Pharmaceuticals and Takeda Pharmaceutical Company Limited announced that Santhera has initiated the pivotal Phase III study with Catena®/Sovrima® (INN: idebenone) for the treatment of Duchenne muscular dystrophy. The 12-month DELOS study will enroll up to 240 patients in 25 centers in Europe and North America. The start of patient enrollment into this Phase III study has triggered a milestone payment of EUR 5 million from Takeda, marketing partner in the European Union and Switzerland, already received by Santhera.
The Phase III study named DELOS (DuchEnne Muscular Dystrophy Long-term IdebenOne Study) is a double-blind, randomized, placebo-controlled clinical trial evaluating Catena®/Sovrima® in up to 240 ambulatory and non-ambulatory patients aged 10 to 18 years. The safety, tolerability and efficacy of one dose of Catena®/Sovrima® (900 mg/day) will be compared to placebo over a treatment period of 12 months. The primary endpoint of the DELOS study is the change from baseline to week 52 in respiratory function measured by peak expiratory flow. Secondary endpoints include other respiratory parameters, muscle strength and motor function over the treatment period and improvement in quality of life amongst others. The study is expected to involve up to 25 study centers in Europe, the United States and Canada.
The principal investigator of the DELOS study is Professor Gunnar Buyse, Professor of Pediatrics & Child Neurology at the University Hospitals Leuven, Belgium. Dr Richard Finkel, MD, Director of Neuromuscular Programs at the Children's Hospital of Philadelphia (PA) will act as Lead Investigator in the United States and Canada. The study design, dose selection and duration of the treatment period of the DELOS study are based on the positive results Santhera obtained in a Phase II study and were pre-discussed with the US Food and Drug Administration as well as with the European Medicines Agency. Furthermore, both agencies agreed that, subject to positive outcome, a single pivotal study could suffice for approval.
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