Santhera Pharmaceuticals announced positive results from the SYROS study demonstrating that long-term treatment with idebenone consistently reduced the rate of respiratory function loss in patients with Duchenne for up to 6 years in a real-world setting.
This long-term data further supports the potential for idebenone to modify the course of respiratory function decline and delay the time to clinically relevant milestones. We are optimistic about these results and look forward to learning more from Santhera in the coming weeks.
Read the update from Santhera
Santhera Pharmaceuticals (SIX: SANN) announces results from the SYROS study demonstrating that long-term treatment with idebenone consistently reduced the rate of respiratory function loss in patients with Duchenne muscular dystrophy (DMD) for up to 6 years in a real-world setting. This long-term data further supports the potential for idebenone to modify the course of respiratory function decline and delay the time to clinically relevant milestones.
The SYROS study was a prospectively planned collection of long-term, retrospective real-world data from patients who completed the positive Phase III DELOS trial (18 out of 64) and were subsequently treated with idebenone (900 mg/day) for on average 4.2 years (range 2.4-6.1 years) under Expanded Access Programs (EAPs). The primary objective of this study was to evaluate the long-term evolution of the respiratory function in patients who maintained treatment with idebenone for up to 6 years compared to their preceding off-idebenone period.
The result of this study, which is consistent with outcomes from the pivotal DELOS study, demonstrated that:
- Switching to and maintaining long-term treatment with idebenone reduced the annual rate of decline in forced vital capacity percent of predicted (FVC%p) by 50%.
- The treatment effect was consistently maintained year-on-year for up to 6 years.
- These findings are further supported by consistent reductions in the rate of both inspiratory and expiratory respiratory function loss over the same period.
- Prolonged treatment with idebenone also reduced the risk of important patient-relevant outcomes, including bronchopulmonary adverse events and hospitalizations due to respiratory causes.
“We are very excited to see that the significant treatment effect with idebenone observed in our 52-week Phase III DELOS study is maintained over the long-term,” said Kristina Sjöblom Nygren, MD, Chief Medical Officer and Head of Development at Santhera. “The new findings are highly relevant for DMD patients in respiratory decline who have an urgent need for a therapy to modify the declining course of respiratory function decline and ultimately delay the need for assisted ventilation.”
The findings from the SYROS study have been accepted for presentation at the 2019 MDA Clinical & Scientific Conference (April 13-17, 2019, in Orlando) and will be submitted for publication in a peer-reviewed journal.