April 11, 2012: Santhera Joins EU-funded EndoStem Consortium in Preparation for Clinical Study with Omigapil in Congenital Muscular Dystrophies
Liestal, Switzerland, April 11, 2012 - Santhera Pharmaceuticals (SIX: SANN) announced today 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.
"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 procedures for further efficacy trials."
About Congenital Muscular Dystrophies
Congenital Muscular Dystrophies refer to a wide variety of inherited neuromuscular conditions characterized by different forms of progressive loss of muscle tissue. Severe forms can affect newborns or young children with life-threatening progressive muscle weakness ("floppy infant syndrome"). Complications associated with the disorder cause immobility at young age and early mortality. Patients suffer from loss of body weight, skeletal deformations and respiratory distress resulting in immobility at young age and early mortality. No pharmacological therapy is currently available or in advanced clinical development. Treatment options are confined to ventilatory support and orthopedic surgery for scoliosis as well as supplementary nutrition to avoid malnutrition.
Omigapil is a deprenyl analog and has anti-apoptotic properties. Originally developed by Novartis, Santhera obtained an exclusive license to develop the molecule in Congenital Muscular Dystrophies. Nonclinical studies in a disease-relevant model showed that omigapil inhibits cell death and reduces body weight loss and skeletal deformation while increasing locomotive activity and protecting from early mortality .
EndoStem (www.endostem.eu) is a partnership of 15 research and clinical teams from globally recognised academic centres, small biotech and large pharmaceutical companies working together to develop new strategies aimed at stimulating stem cells that are resident in damaged tissue to repair it in situ. This approach is recognised as one of the most promising approaches to targeting stem cells for regenerative medicine due to the alignment with existing therapeutic development approaches used by large industry and recent advances in understanding the key barriers for tissue regeneration. Coordinated by Professor David Sassoon, cofinanced by the European Commission via the 7th Framework Programme the aims of the project over are: (i) the implementation of clinical trials, with muscular dystrophies as the primary clinical target using innovative biopharmaceuticals; (ii) the development of novel best in class biopharmaceuticals with highly specific and well defined modes of action; (iii) the fast-track clinical translation based on a constant feedback loop between emerging patient responsiveness to new drugs and the development of the next generation of therapeutics; and (iv) the better understanding of the key issues preventing effective tissue repair matched with approaches to circumvent them.
 Erb M. et.al. (2009). Omigapil ameliorates the pathology of muscle dystrophy caused by laminin-alpha 2 deficiency. Journal of Pharmacology and Experimental Therapeutics 331: 787-795
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Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative pharmaceutical products for the treatment of orphan neuromuscular and mitochondrial diseases, areas of high unmet medical with no current therapies. Santhera's first product Catena® is currently marketed in Canada to treat Friedreich's Ataxia. Catena® is also under review for marketing authorization by the European Medicine Agency as the first therapy for patients suffering from Leber's Hereditary Optic Neuropathy. For further information, please visit www.santhera.com.
Catena® is a trademark of Santhera Pharmaceuticals.
For further information, contact
Thomas Meier, Chief Executive Officer
Phone: +41 61 906 89 64
Thomas Staffelbach, Vice President, Head Public & Investor Relations
Phone: +41 61 906 89 47
Jonathan Dando, Consortium Manager
Phone +33 6 225 418 20
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