Santhera Joins EU-funded EndoStem Consortium in Preparation for Clinical Study with Omigapil in Congenital Muscular Dystrophies

12-Apr-2012 - Switzerland

Santhera Pharmaceuticals announced its participation in the EndoStem Consortium, a pan-European partnership of 15 academic and industry teams. EndoStem is co-funded by the European Union and seeks to accelerate the development of effective therapies for muscular dystrophies. In collaboration with the consortium, Santhera will conduct a Phase I study with its compound omigapil in Congenital Muscular Dystrophies. These severe, genetically determined neuromuscular diseases frequently affect children with life-threatening progressive muscle weakness.

EndoStem is an academic and industry partnership of research and clinical teams and includes partners in six European countries. The consortium's main goals are to conduct clinical trials in muscular dystrophies using innovative biopharmaceuticals with highly specific and well-defined modes of action. EndoStem is co-funded by the European Commission under the 7th Framework Programme and is coordinated by Professor David Sassoon, Institut de Myologie at the Pierre and Marie Curie University in Paris, France.

As part of the program, Santhera will develop a novel formulation of omigapil and support the planning and conduct of an ascending multiple-dose Phase I study. The open-label, 12-week clinical trial will determine the pharmacokinetic, safety and tolerability profile of omigapil in children. In addition, the study will assess the feasibility and procedures for future efficacy trials in this patient population. Study centers will be the Dubowitz Neuromuscular Centre, Institute of Child Health of the University College London, UK, and the National Institute of Neurological Disorders and Stroke of the National Institutes of Health in Bethesda, MD, US. The study is supported by Cure CMD, a dedicated patient advocacy group whose mission is to bring research, treatments and, in the future, a cure for Congenital Muscular Dystrophies. The start of patient enrollment is expected during the second half of 2012.

https://www.bionity.com/en/news/137398/santhera-joins-eu-funded-endostem-consortium-in-preparation-for-clinical-study-with-omigapil-in-congenital-muscular-dystrophies.html