Santhera has initiated the pivotal phase III study with Catena/Sovrima for the treatment of Duchenne Muscular Dystrophy.
Reportedly, the 12-month study will enroll up to 240 patients in 25 centers in Europe and North America. The start of patient enrollment into the phase III study has triggered a milestone payment of E5m from Takeda (marketing partner in EU and Switzerland) already received by Santhera.
The phase III study, 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 company said that the safety, tolerability and efficacy of one dose of Catena/Sovrima (900mg/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.
However, the 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 US 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 FDA as well as with the European Medicines Agency. Furthermore, both the agencies agreed that, subject to positive outcome, a single pivotal study could suffice for approval.
Klaus Schollmeier, CEO of Santhera, said: “The start of the DELOS study is another significant landmark in the development of Catena/Sovrima as potential first therapies for rare neuromuscular diseases. Currently, sufferers and physicians have no treatment options for such devastating diseases. The high unmet medical need is reinforced in the frequent, positive feedback we continue to receive from Canada, where Catena is already available to treat Friedreich’s Ataxia.”