Santhera Presents Data from 2-Year Open-Label Intervention Study with Catena in Duchenne Muscular Dystrophy

10-May-2011 - Switzerland

Santhera Pharmaceuticals announced 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 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.

https://www.bionity.com/en/news/132331/santhera-presents-data-from-2-year-open-label-intervention-study-with-catena-in-duchenne-muscular-dystrophy.html