Santhera muscular dystrophy drug shows success first time in late clinical trial

For the first time, a drug for Duchenne muscular dystrophy (DMD) – from Swiss drugmaker Santhera Pharmaceuticals (SIX: SANN) - has been successful in a late-stage Phase III clinical study conducted in the US. This is promising news for patients with DMD because the studies indicate that Catena/Raxone (idebenone) can mitigate and delay respiratory weakness and dysfunction, the main cause of death in these patients.

As previously announced, the DELOS trial met the primary endpoint, the difference between Catena/Raxone and placebo in the change from baseline to week 52 in Peak Expiratory Flow (PEF as percent predicted, PEF%p). Hospital-based spirometry assessments demonstrated that Catena/Raxone significantly reduced the annual decline in PEF%p by 66% compared to patients taking placebo. The average annual decline in PEF%p was 9.0% for placebo (Baseline: 54.3%; Week 52: 45.3% (n=27), p<0.001) versus 3.1% for Catena/Raxone (Baseline PEF%P: 53.1%; Week 52: 50.1% (n=30); p=0.13) for a treatment group difference in change from Baseline to Week 52 of 5.96% (p=0.04).

Santhera today announced that this finding has been corroborated by the results of secondary endpoints assessing respiratory function in all randomized and treated subjects. When measured weekly by the patient at home using the hand-held ASMA-1 device (secondary endpoint), Catena/Raxone significantly reduced the annual decline in PEF%p by 80% compared to patients taking placebo. The ASMA-1 device showed a significant 9.0% decline in PEF%p occurred between Baseline and Week 52 in the placebo group (n=31; p<0.001), compared to a non-significant decline of 1.8% in the Catena/Raxonegroup (n=31; p=0.44), for a treatment group difference in change from Baseline to Week 52 of 7.2% (p=0.03).