New Data from Santhera's Phase III Trial (DELOS) in Duchenne Muscular Dystrophy (DMD) in Neuromuscular Disorders - Seite 2

"The results published in Neuromuscular Disorders are clearly of clinical relevance," commented Thomas Meier, PhD, CEO of Santhera. "The data show that the efficacy of Raxone on the preservation of respiratory function, a major goal of DMD therapy, translates to a reduced risk of bronchopulmonary complications, which was associated with fewer hospitalizations caused by respiratory complications, and a reduced need for systemic antibiotics. The clinical relevance of these findings is supported by the outcome of the recent survey conducted by Parent Project Muscular Dystrophy (PPMD) and co-sponsored by Santhera, in which DMD patients and their caregivers placed a high value on treatments that could reduce pulmonary complications."

PPMD's Founding President & CEO, Pat Furlong was encouraged by the reported data: "PPMD congratulates Santhera on the latest update and publication. Pulmonary issues, and optimizing pulmonary care for people with Duchenne, has been a longtime priority for PPMD and is a significant part of our extensive care initiatives. Recently, with support from Santhera, we convened an incredible meeting of leading pulmonary experts to discuss the findings from our benefit-risk project and to map out next steps we can take to standardize pulmonary outcome measures collected in clinical trials and improve pulmonary care. We believe in the Santhera team and we are hopeful that Raxone will become one of the tools in the arsenal that will end Duchenne."

About Duchenne Muscular Dystrophy and DELOS
Duchenne muscular dystrophy (DMD) is one of the most common and devastating types of muscle degeneration and results in rapidly progressive muscle weakness. DMD is characterized by a loss of the protein dystrophin, leading to cell damage, impaired calcium homeostasis, elevated oxidative stress and reduced energy production in muscle cells. This results in progressive muscle weakness and wasting and early morbidity and mortality due to respiratory failure.
Idebenone is a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating mitochondrial electron transport, reducing and scavenging reactive oxygen species (ROS) and supplementing cellular energy levels.
DELOS was a phase III, double-blind, placebo-controlled trial which randomized 64 patients, 10-18 years of age, to receive either Raxone tablets or matching placebo. Efficacy measurements included peak expiratory flow (PEF), forced vital capacity (FVC), forced expiratory volume (FEV1) and peak cough flow (PCF). The trial met its primary endpoint and demonstrated that Raxone can slow the loss of respiratory function in patients not taking concomitant glucocorticoids. The positive outcome of the phase III DELOS study was first published in The Lancet (Buyse et al., The Lancet 2015 385(9979):1748-57).