Liestal, Switzerland, November 26, 2013 - Santhera Pharmaceuticals (SIX: SANN) announced today that the Phase III DELOS study of orally administered Catena®/Raxone® (INN:idebenone) in patients with Duchenne Muscular Dystrophy (DMD) remains on track to deliver pivotal data early in 2Q 2014 and is unaffected by recent clinical and regulatory developments related to exon skipping-based treatment strategies.
Santhera`s randomized, placebo-controlled DELOS study completed the enrolment of 65 steroid non-using DMD patients at the end of 2012 and in April 2013 successfully passed a planned futility analysis for safety and efficacy for the primary respiratory function endpoint.
"Following the successful futility analysis earlier this year, we are pleased to confirm that the DELOS study remains on track to deliver pivotal efficacy data early in the second quarter of 2014. In view of the recent setbacks for exon-skipping strategies, where the apparent disconnect between de novo dystrophin synthesis and outcomes for the 6-minute walk test (6MWT) have called this approach into question, it is important to emphasize that Catena®/Raxone® has a completely different mode of action and is unaffected by these developments. Furthermore, for the DELOS trial we selected respiratory function outcomes for the primary and secondary endpoints, the use of which was prospectively agreed with both the FDA and EMA prior to the initiation of the study. As has been emphasized by the FDA, these endpoints have the advantage that they are applicable across a much wider range of disease severities and can be used to assess the efficacy of Catena®/Raxone® in both the non-ambulatory and ambulatory patients being studied in the DELOS program", commented Nick Coppard, Santhera`s Senior VP Development.
"Despite the recent set-backs for exon skipping-based treatment strategies for DMD, the development of Catena®/Raxone® is progressing as planned. Once the data from the independently powered sub-group of patients not using steroids is available, we plan to consult with the regulatory authorities on the best pathway to approval for this sub-group and on the initiation of the enrolment of steroid-using patients, as foreseen in the protocol", commented Thomas Meier, CEO of Santhera.
About Idebenone in DMD
Idebenone is an orally bioavailable, synthetic, short-chain benzoquinone targeting the enzyme NAD(P)H:quinone oxidoreductase (NQO1). The molecule is capable of transferring electrons directly onto complex III of the mitochondrial electron transport chain, thereby restoring mitochondrial function and cellular energy levels. In addition, idebenone is a powerful anti-oxidant. Efficacy of idebenone has been demonstrated in the mdx mouse model of dystrophin deficiency. Promising efficacy data with Catena®/Raxone® in DMD patients were obtained previously in the DELPHI trial, a Phase II randomized, placebo-controlled study in 21 DMD patients  .
DELOS (DuchEnne Muscular Dystrophy Long-term IdebenOne Study) is a Phase III randomized, double-blind study in patients aged 10-18 years with DMD of any mutational etiology, ambulatory or non-ambulatory, recruited in Europe and in North America. DELOS uses the change from baseline to 12 months in per cent predicted Peak Expiratory Flow (PEF) as the primary endpoint and a number of respiratory function tests as secondary endpoints. The study is powered to detect a 10.3% difference in the change from baseline to 12 months in per cent predicted PEF between the Catena®/Raxone® and placebo groups.
In April this year the independent Data Safety Monitoring Board (DSMB) for DELOS informed Santhera that the study has a reasonable chance of achieving its primary endpoint for improving or delaying the loss of respiratory function in Duchenne patients not using steroids and since no safety issues were detected, recommended that the study should continue as planned. The futility analysis included primary efficacy variable data after at least 6 months of treatment from 80%, and final outcome data after 12 months of treatment from over 60% of the planned total number of patients in the steroid non-using patient cohort.
 Buyse G., et al. (2011). Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled trial. Neuromuscular Disorders 21:396-405.
 Buyse G., et al. (2013). Effects of glucocorticoids and idebenone on respiratory function in patients with Duchenne Muscular Dystrophy. Pediatric Pulmonology 48(9):912-920.
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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 need with no current therapies. For further information, please visit www.santhera.com.
Catena® (for US and Canada) and Raxone® (for Europe) are trademarks of Santhera Pharmaceuticals.
For further information, contact
Thomas Meier, Chief Executive Officer
Phone: +41 61 906 89 64
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Source: Santhera Pharmaceuticals Holding AG via GlobeNewswire