Santhera presents long-term efficacy results in patients with Duchenne muscular dystrophy

Santhera Pharmaceuticals (SIX: SANN) and its collaborating clinical experts present results of long-term efficacy on respiratory function outcomes in idebenone-treated patients with Duchenne muscular dystrophy (DMD) at the 2019 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference held in Orlando, USA, April 13-17, 2019.

Posters presented at the MDA conference show consistent long-term efficacy data from two randomized placebo-controlled studies and their extended long-term data collections in patients treated with idebenone (Posters #7 and #9). In addition, new natural history data demonstrates the predictive value of slowing the rate of respiratory function decline on delaying time to initiation of assisted ventilation or death (Poster #11). Collectively, these data support the long-term potential of treatment with idebenone to delay the time to clinically relevant outcomes.

Poster #7: "SYROS study - long-term reduction in rate of respiratory function decline in patients with Duchenne Muscular Dystrophy (DMD) treated with idebenone"
Poster #9: "Consistent long-term effect of idebenone in reducing respiratory function decline in advanced patients with Duchenne Muscular Dystrophy (DMD)"
Poster #11: "Crossing thresholds and changing rates of respiratory function decline are predictive of clinical outcomes in Duchenne Muscular Dystrophy (DMD)"

Additionally, the new data will be presented at a Santhera hosted Industry Forum Lunch by a panel of clinical experts: "Respiratory function decline in Duchenne muscular dystrophy: insights and evolving treatment strategies"

In February, Santhera announced topline results from the SYROS study which demonstrated 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 in DMD and thereby delay the time to clinically relevant milestones.

The posters can be viewed here and the findings from the SYROS study will be submitted for publication in a peer-reviewed journal.