Liestal, Switzerland, July 11, 2013 - Santhera Pharmaceuticals (SIX: SANN) announced today that it has initiated a collaboration with the European Vision Institute Clinical Research Network (EVICR.net) to assess the natural course of Leber's Hereditary Optic Neuropathy (LHON). Data from this collaboration is intended to be included in a marketing authorization application (MAA) for Raxone® (idebenone) in Europe.
Under the collaboration, Santhera will receive data on the change in vision over time in untreated LHON patients from selected EVICR.net centers. This data will be compared to the vision outcomes data currently being collected from physicians around the world who are treating LHON patients with Raxone® in clinical practice.
"Lack of natural history data is a general shortcoming in clinical research of rare diseases and a limitation in the development of orphan drugs", commented Thomas Meier, CEO of Santhera. "We have initiated this collaboration with EVICR.net to increase our understanding of changes in vision in LHON patients, including spontaneous recovery after severe vision loss. We expect to integrate these data as a comparator group in a revised MAA for Raxone® in the treatment of LHON that we plan to submit as early as the first quarter next year."
"We are excited about this collaboration and the opportunity to support Santhera in the collection of natural history data", added José-Alain Sahel, Professor at the Université Pierre et Marie Curie, Director of the Institut de la Vision, Chairman at the Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts (Paris, France) and Chairman of EVICR.net. "EVICR.net was established as European consortium to promote academic translational research and support industry in conducting clinical research, including assessment of natural history data, which is of particular relevance in rare diseases such as LHON."
About EVICR.net
European Vision Institute Clinical Research Network (EVICR.net) was established in 2010 as an independent European Economic Interest Grouping to serve as a platform for clinical trial research in ophthalmology in Europe. It is a network of 76 European ophthalmological clinical research sites in 16 European countries dedicated to perform clinical research in ophthalmology with the highest standards of quality, following the European and international directives for clinical research according to harmonized standard operating procedures.
About LHON
Leber's Hereditary Optic Neuropathy (LHON) is a heritable genetic disease causing blindness. The disease typically presents in young adults, mostly men, as rapid, painless loss of central vision in one eye, followed by visual loss in the fellow eye within a few months of the onset of symptoms, leading to blindness. Over 95% of patients harbor one of three pathogenic mutations of the mitochondrial DNA which cause a defect in the complex I subunit of the mitochondrial respiratory chain. This defect leads to decreased cellular energy (ATP) production, increased oxidative stress and retinal ganglion dysfunction which cause progressive loss of visual acuity and blindness.
Santhera develops Raxone® (idebenone) as treatment for patients with LHON. The synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) is capable of transferring electrons directly onto complex III of the mitochondrial electron transport chain, thereby circumventing the complex I defect and restoring cellular energy levels in retinal ganglion cells and promoting recovery of visual acuity.
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About Santhera
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 mitochondrial and neuromuscular diseases, areas of high unmet medical need with no current therapies. For further information, please visit www.santhera.com.
Raxone® is a trademark of Santhera Pharmaceuticals.
For further information, contact
Thomas Meier, Chief Executive Officer
Phone: +41 61 906 89 64
thomas.meier@santhera.com
Disclaimer / Forward-looking statements
This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.
