Additional data from DELOS study for Raxone (idebenone)shows efficacy on inspiratory function in patients with Duchenne Muscular Dystrophy- Santhera

Santhera Pharmaceuticals announces that additional data from the pivotal phase III trial (DELOS) demonstrating efficacy of Raxone (idebenone) on inspiratory function in patients with Duchenne Muscular Dystrophy (DMD) were published online in the medical journal Pediatric Pulmonology (http://dx.doi.org/10.1002/ppul.23547).

The assessment of dynamic inspiratory function provides valuable information about the degree and progression of pulmonary involvement in patients with DMD. The data now published in Pediatric Pulmonology evaluated the effect of Raxone on the highest inspiratory flow generated during a forced vital capacity (FVC) maneuver (maximum inspiratory flow; V'I,max(FVC)). DMD patients in both treatment groups of the DELOS trial (Raxone, n=31; placebo: n=33) had comparable and abnormally low V'I,max(FVC) at baseline. During the study period, V'I,max(FVC) further declined by -0.29 L/s (liters/second) in patients on placebo (95% CI: -0.51, -0.08; p=0.008 at Week 52), whereas it remained stable in patients on Raxone (change from baseline to Week 52: 0.01 L/s; 95% CI: -0.22, 0.24; p=0.950). The between-group difference demonstrated a positive treatment effect for Raxone by 0.27 L/s (p=0.043) at Week 26 and 0.30 L/s (p=0.061) at Week 52. In addition, during the study period, the fraction of the maximum flow that is not used during tidal breathing, called Inspiratory Flow Reserve (IFR), improved by 2.8% in patients receiving Raxone and worsened by -3.0% among patients on placebo (between-group difference 5.8% at Week 52; p=0.040).

Comment:Since its completion, the DELOS Study Group has published data demonstrating a statistically significant and clinically relevant benefit of Raxone treatment on expiratory and inspiratory function, a reduced risk of patients treated with Raxone to experience bronchopulmonary complications and a reduced need for systemic antibiotics. These results demonstrate an overall benefit for patients receiving Raxone on multiple pulmonary function outcomes, which is of relevance for patients with DMD.