Date: 2012-04-11
Type of information: Initiation of preclinical development
phase: 1
Announcement: announcement of Santhera Pharmaceuticals\' participation in the EndoStem Consortium, a pan-European partnership of 15 academic and industry teams that 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
Company: Santhera Pharmaceuticals (Switzerland) and EndoStem Consortium
Product: omigapil
Action
mechanism: neuronal apoptosis inhibitor. Omigapil is a deprenyl analog and has anti-apoptotic properties. The compound interacts with the glycolytic enzyme glyceraldehyde 3-phosphate dehydrogenase (GAPDH) which has been implicated in programmed cell death (apoptosis). In preclinical research, Santhera demonstrated that omigapil prevents apoptosis in muscle tissue, ameliorates muscle histology, prevents loss of muscle tissue and increases body weight and survival of disease-relevant animal models for congenital muscular dystrophy.
Disease: congenital muscular dystrophy
Therapeutic area: Neuromuscular diseases - Rare diseases - Genetic diseases
Country: UK, USA
Trial
details: 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.
Latest
news: Santhera Pharmaceuticals has 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.
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 start of patient enrollment is expected during the second half of 2012.
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 (http://www.endostem.eu/).
