Santhera Presents Data from 2-Year Open-Label Intervention Study with Catena in Duchenne Muscular Dystrophy at the MYOLOGY 2011 Conference
Liestal, Switzerland, May 9, 2011 - Santhera Pharmaceuticals (SIX: SANN) announced today that it will present first analyses of the 2-year open-label study (DELPHI-E) evaluating Catena® for the treatment of Duchenne Muscular Dystrophy. The findings indicate that Catena® can slow the decline in respiratory function associated with patients as they age. Data will be presented on May 9, 2011 in Lille, France at the 4th International Congress of Myology .
Following a 12-month, randomized, placebo controlled Phase II trial (the DELPHI study), participants were offered enrollment in a 24-month open-label study (DELPHI-E). The 19 subjects in the DELPHI-E study received weight-adjusted doses of Catena® at 450/900 mg/day for body weight of below/above 45 kg. Efficacy was assessed as change from baseline for respiratory function tests, e.g. peak expiratory flow (PEF), forced vital capacity (FVC) and maximum inspiratory mouth pressures (MIP). On average, patients did not receive Catena® for 21 months between the end of the DELPHI study and their enrollment into the DELPHI-E study. During this period, a noticeable decline in PEF, FVC and MIP was observed. Despite the patients' higher age, the rate of decline was numerically lower for PEF and MIP when patients received Catena® treatment during the DELPHI-E study compared to the period where they did not receive Catena®. There was no change in the rate of decline in FVC between the off-medication period and the DELPHI-E study period. Comparing data from DELPHI-E with natural history data for Duchenne Muscular Dystrophy obtained at the Children's Hospital of Philadelphia showed that patients in the DELPHI-E study performed better in PEF for ages 16 and older and in FVC for ages 18 and older. This suggests that Catena® possibly slows the progression of respiratory dysfunction in these age periods.
"Despite the limitations from the open-label nature, our preliminary analysis of the DELPHI-E study indicates that treatment with Catena® can slow the decline in respiratory function associated with increased age in patients suffering from Duchenne Muscular Dystrophy. Results from this 2-year intervention study are in agreement with and confirm data from the 12-month placebo-controlled DELPHI study," said Thomas Meier, Chief Scientific Officer of Santhera. "Importantly, these findings add to the validation of the clinical relevance of the respiratory endpoint we are using in the ongoing DELOS study. This Phase III study is set up to further evaluate the efficacy of Catena® on respiratory function in Duchenne Muscular Dystrophy".
In addition to the preliminary analysis of the open label extension of the DELPHI study, Santhera will present an update on the ongoing DELOS Phase III study with Catena® in Duchenne Muscular Dystrophy .
 Buyse G.M., Goemans N., van den Hauwe M., Thijs D., Wei Hui, Rummey C., Meier T., Mertens L.: Results from a two-year open label intervention study with idebenone (Catena®) in patients with Duchene Muscular Dystrophy. Poster presentation at the 4th International Congress of Myology in Lille, France, from May 9 to 13, 2011.
 Buyse G.M., Voit T., Schara U., Verschuuren J., Bernert G., Jeannet P.Y., Sejersen T., Rummey C., Rubino R., Meier T. on behalf of the DELOS study consortium: Update of an Ongoing International Phase III Study with Idebenone (Catena®) in Patients with Duchenne Muscular Dystrophy - The DELOS Study. Poster presentation at the 4th International Congress of Myology in Lille, France, from May 9 to 13, 2011.
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.
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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 severe neuromuscular diseases, an area of high unmet medical need which includes many orphan 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.
For further information, contact
Thomas Meier, Chief Scientific Officer
Phone: +41 (0)61 906 89 52
Thomas Staffelbach, Head Public & Investor Relations
Phone: +41 (0)61 906 89 47
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.
Posted: May 2011