Santhera Pharmaceuticals announced the Phase 3 SIDEROS study will be discontinued following an interim analysis conducted by the independent Data and Safety Monitoring Board (DSMB) which concluded that the study was unlikely to meet its primary endpoint. The SIDEROS study was evaluating Puldysa (idebenone) in patients with Duchenne muscular dystrophy (DMD) who are in respiratory decline and receive concomitant glucocorticoid treatment. The interim analysis was based on the primary endpoint of the study, the change of forced vital capacity % predicted (FVC%p) from baseline to 18 months of treatment. The outcome revealed that the probability of reaching the primary endpoint at the end of the study is too small to merit the continuation of the study. There were no safety concerns noted by the DSMB.
In conjunction with closing the trial, Santhera will withdraw the European marketing authorization application and end the global development program for Puldysa. The Company intends to initiate a restructuring plan for the business with a focus on retaining key functions for bringing DMD drug candidate vamorolone to patients and execute on its other pipeline programs.
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