Publication in BRAIN Highlights Medical Potential of Santhera's Catena in Leber's Hereditary Optic Neuropathy
Liestal, Switzerland, July 27, 2011 - Santhera Pharmaceuticals (SIX: SANN) announced today the publication of the results from its RHODOS clinical study in Leber's Hereditary Optic Neuropathy (LHON) in BRAIN , a leading international journal of clinical neurology. The authors concluded that treatment with Catena® (idebenone) can prevent or reverse vision loss and blindness. LHON patients with discordant visual acuity and in patients harboring G11778A or G3460A mutations are the most likely to benefit from idebenone treatment. Data from the RHODOS study are included in the filing of Santhera's Marketing Authorization Application which is currently under review by the European Medicines Agency (EMA).
The publication in BRAIN, a leading international high-impact journal publishing clinical neurology, reports on significant differences between idebenone and placebo treatment in the first randomized controlled trial in the mitochondrial disorder LHON. Data from the 6-months RHODOS study provide evidence that idebenone ameliorated the visual outcome in affected patients enrolled in this study. In the authors' opinion all prespecified secondary visual acuity endpoints as well as subgroup and responder analyses pointed towards a beneficial effect for idebenone. Based on this study the largest treatment effect of idebenone was seen in patients harboring the G11778A or G3460A mutations which account for approximately 80% of all European and North American LHON cases. The study also showed that patients with discordant visual acuity between the two eyes, who are at greatest risk of disease progression, were effectively protected from further vision loss. "The clinical significance of this finding is that patients with discordant visual acuity may represent the patients with greatest potential reserve and therefore the patients that have the most clinical benefit in regards to preventing further visual loss," concluded the authors.
Thomas Meier, Santhera's Chief Scientific Officer, commenting on the BRAIN publication said: "We are excited that data from our study are now available to a wider audience of ophthalmologists, neuro-ophthalmologists and neurologists. The reported effects of idebenone on patients with the G11778A or G3460A mutations are of particular interest to us as we specifically applied for marketing approval based on the significant treatment effect seen in these patients. Data from the RHODOS study are part of our MAA file currently under review at the EMA."
References  Thomas Klopstock, Patrick Yu-Wai-Man, Konstantinos Dimitriadis, Jacinthe Rouleau, Suzette Heck, Maura Bailie, Alaa Atawan, Sandip Chattopadhyay, Marion Schubert, Aylin Garip, Marcus Kernt, Diana Petraki, Christian Rummey, Mika Leinonen, Günther Metz, Philip G Griffiths, Thomas Meier and Patrick F Chinnery: A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy. DOI: 10,1093/brain/awr170.
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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 severe neuromuscular and neurodegenerative diseases, an area of high unmet medical need which includes many orphan and niche indications with no current therapy. Santhera's first product, Catena® to treat Friedreich's Ataxia is marketed in Canada. For further information, please visit www.santhera.com.
Catena® is a trademark of Santhera Pharmaceuticals.
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News release BRAIN publication<http://hugin.info/137261/R/1533675/467245.pdf>
Posted: July 2011