Publication in European Heart Journal Highlights Potential of Santhera's SNT-MC17

The publication in the European Heart Journal reports on the results of a very long- term, blinded and placebo-controlled study in the dystrophin deficient mdx mouse, a disease relevant animal model. The main finding of the study is that presymptomatic- initiated and long-term idebenone treatment significantly prevented cardiac diastolic dysfunction, preserved cardiac systolic contractile reserve and, as such blocked the development of lethal acute heart failure during a dobutamine-mediated stress protocol, reduced cardiac inflammation and fibrosis, and improved voluntary running performance in the mdx mouse model.

In the authors' opinion the beneficial effects of idebenone can be explained by its ability to improve mitochondrial respiratory chain function and to reduce oxidative stress, pathways that have been implicated in the pathophysiology of dystrophin deficient muscular dystrophy. "We have identified a novel potential therapeutic strategy for human Duchenne Muscular Dystrophy, as SNT-MC17/ idebenone was cardioprotective and improved exercise performance in the dystrophin-deficient mdx mouse", the authors concluded.

Thomas Meier, Santhera's Chief Scientific Officer, commenting on the European Heart Journal publication said: "Duchenne Muscular Dystrophy is a severe and still incurable disease. Heart failure is responsible for early death of a significant fraction of Duchenne Muscular Dystrophy patients. The reported cardioprotective data are in line with the evidence collected in our Phase II proof-of-concept trial and support SNT- MC17/idebenone as potential treatment option of Duchenne Muscular Dystrophy."

Reference [1] Gunnar M. Buyse, Gerry Van der Mieren, Michael Erb, Jan D'hooge, Paul Herijgers, Erik Verbeken, Alejandro Jara, An Van Den Bergh, Luc Mertens, Isabelle Courdier-Fruh, Patrizia Barzaghi, and Thomas Meier (2008). Long-term blinded placebo-controlled study of SNT-MC17/ idebenone in the dystrophin deficient mdx mouse: cardiac protection and improved exercise performance. European Heart Journal (doi:10.1093/eurheartj/ehn406); Epub ahead of print 2008 Sep 10.

About Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy is the most common and a devastating type of muscular degeneration and results in rapidly progressive muscle weakness. It is a genetic, degenerative disease that is inherited in an X-linked recessive mode. Duchenne Muscular Dystrophy affects approximately 30,000 patients in the USA, EU, and Japan and its incidence is approximately 1 in 3,500 live born males worldwide. Duchenne Muscular Dystrophy is characterized by a loss of the protein dystrophin, leading to progressive muscle weakness and wasting through a complex cascade that involves impaired calcium homeostasis, mitochondrial dysfunction and oxidative stress. The average age of onset is between 3 and 5 years of age with a loss of ambulation in teenage patients. Dilated cardiomyopathy and respiratory failure are commonly associated with this chronic disease leading to early morbidity and mortality in Duchenne Muscular Dystrophy patients.

ENDE Pressemeldung / Pressemitteilung Publication in European Heart Journal Highlights Potential of Santhera's SNT-MC17

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Santhera Pharmaceuticals (SWX: SANN) is a Swiss specialty pharmaceutical company focusing on the discovery, development and marketing of small molecule pharmaceutical products for the treatment of severe neuromuscular diseases. Santhera’s vision is to become a leading specialty pharmaceutical company offering therapies for a number of indications in this area of high unmet medical need which includes many orphan indications with no current therapy.

Santhera currently has five clinical-stage development programs, three of which are investigating its lead compound, SNT-MC17 (INN: idebenone), in the treatment of Friedreich’s Ataxia (FRDA), Duchenne Muscular Dystrophy (DMD) and Leber’s Hereditary Optic Neuropathy (LHON). Another clinical program is investigating JP- 1730 (INN: fipamezole) for the treatment of Dyskinesia in Parkinson’s Disease (DPD) in cooperation with Juvantia, the compound’s owner. The fifth program comprises SNT-317 (INN: omigapil) in Congenital Muscular Dystrophies (CMD), a compound in-licensed from Novartis.

Santhera’s first product, SNT-MC17 (INN: idebenone) has received a marketing approval with conditions from Health Canada to treat Friedreich’s Ataxia and will be marketed under its brand name Catena. The product is also under review by health authorities in the EU and in Switzerland for the same indication, while in the United States a pivotal phase III trial is recruiting patients. SNT- MC17 has recently shown efficacy as a potential treatment in DMD, the compound's second indication.