New Data from Santhera’s Phase III Trial (DELOS) in Duchenne Published in Neuromuscular Disorders

Santhera Pharmaceuticals announced today that additional data from the pivotal phase III trial (DELOS) were published online as an article in press in Neuromuscular Disorders, the official journal of the World Muscle Society.

These data show that Duchenne patients treated with Raxone® (idebenone) have a reduced risk of bronchopulmonary complications including fewer hospitalizations caused by such complications and a reduced need for systemic antibiotic treatment compared to patients receiving placebo.

PPMD is encouraged by the reported data and we congratulate 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.

Read Santhera's press release to learn more: 

New Data from Santhera’s Phase III Trial (DELOS) in Duchenne Muscular Dystrophy (DMD) in Neuromuscular Disorders 

Raxone® Reduces Bronchopulmonary Complications in Patients with DMD 

Liestal, Switzerland, June 1, 2016 – Santhera Pharmaceuticals (SIX: SANN) announces that additional data from the pivotal phase III trial (DELOS) were published online as an article in press in Neuromuscular Disorders, the official journal of the World Muscle Society (http://dx.doi.org/10.1016/j.nmd.2016.05.008). These data show that DMD patients treated with Raxone® (idebenone) have a reduced risk of bronchopulmonary complications includ-ing fewer hospitalizations caused by such complications and a reduced need for systemic antibiotic treatment compared to patients receiving placebo. 

These results originate from additional analyses of the phase III DELOS trial which was performed in DMD patients not taking concomitant glucocorticoids and showed that Raxone® reduced the loss of respiratory function in a statistically significant and clinically relevant manner (Buyse et al., The Lancet 2015 385(9979):1748-57). The data now published in Neuromuscular Disorders demonstrated that Raxone also reduced the proportions of patients falling below clinically relevant thresholds for peak cough flow, relevant for proper airway clearance, and forced vital capacity, an indicator of respiratory failure. Researchers also found that there were fewer bronchopulmonary adverse events (BAEs), including upper and lower airway infections and related complications, in patients on Raxone therapy (6 of 31 patients with 7 BAEs) compared to patients in the placebo group (17 of 33 patients with 28 BAEs). For the patients receiving Raxone, there was a 67% reduction for the risk of experiencing at least one BAE during the 1-year study period (p = 0.0187), and a 72% reduction for the risk of experiencing one or more BAEs (p = 0.0026). Additionally, the overall BAE duration for the Raxone group (82 days) was markedly shorter than for the placebo group (222 days). This result was also supported by a smaller number of hospitalizations due to respiratory complications in the Raxone group compared to the placebo group. The need for systemic antibiotic use was also lower in the Raxone group (7 or 22.6% of patients with 8 episodes of antibiotic treatment) than in the placebo group (13 or 39.4% of patients with 17 episodes).

“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.”

Santhera also recently announced that it has filed a Marketing Authorization Application in the European Union for Raxone® for the treatment of Duchenne. Click here to learn more.

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Comment by Jason Darienzo on June 1, 2016 at 12:42pm

Interesting results but wouldn't non-invasive ventilation (Bi-PAP, volume ventilator, and cough assist machine) have the same effect? 

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