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.
“We would like to thank the patients and the families, as well as investigators and medical professionals, who participated in the SIDEROS study. Without their contributions we would not be able to advance DMD research,” said Dario Eklund, Chief Executive Officer of Santhera. “While this is obviously not the outcome we expected, all our efforts in DMD will now be focused on progressing the promising drug candidate vamorolone which we recently licensed from ReveraGen to its next inflection point, the readout of 6-month topline data from the pivotal VISION-DMD study planned for the second quarter of 2021.”
Puldysa has been granted Orphan Drug status in the US and in Europe, Fast Track and Rare Pediatric Disease designations by the US FDA and Promising Innovative Medicine (PIM) status by the UK MHRA. In the UK, Puldysa is available to patients through the Early Access to Medicines Scheme (EAMS).
About Puldysa (idebenone)
Puldysa (idebenone) is a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1). It has the potential to stimulate mitochondrial electron transport, thus reducing and scavenging reactive oxygen species. It also supplements cellular energy levels within the body.