Santhera Joins EU-funded EndoStem Consortium in Preparation for Clinical Study with Omigapil in Congenital 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 dis-eases 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 addi-tion, the study will assess the feasibility and procedures for future efficacy trials in this patient popu-lation. 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 ded-icated 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. “We look forward to working closely with Santhera in preparation of this clinical study with omigapil in Congenital Muscular Dystrophies. Santhera’s participation in the EndoStem program is based on their focus and expertise in advancing drug candidates through preclinical and clinical development in muscular dystrophies,” said David Sassoon, the coordinator of the EndoStem Consortium. “EndoStem has ambitious goals to coordinate this pan-European partnership of 15 research and clinical teams from academia, biotech and pharmaceutical companies. We aim at accelerating the translation of research into the clinical development of new therapies.” “Santhera’s inclusion in this program reflects both our scientific and clinical expertise in this special therapeutic area,” said Thomas Meier, Chief Executive Officer of Santhera. “We will support the consortium by developing and supplying a formulation for omigapil that is suitable for use in children with Congenital Muscular Dystrophies. The planned study will determine the pharmacokinetic profile of omigapil, determine adequate drug dosing in this vulnerable patient population and assess pro-cedures for further efficacy trials.”
Countries
Austria, Belgium, Bulgaria, Cyprus, Czechia, Germany, Denmark, Estonia, Greece, Spain, Finland, France, Hungary, Ireland, Italy, Lithuania, Luxembourg, Latvia, Malta, Netherlands, Poland, Portugal, Romania, Sweden, Slovenia, Slovakia, United Kingdom