Santhera Pharmaceuticals has announced that the full results of the double-blind placebo-controlled Phase III trial (DELOS) demonstrating efficacy and safety of Raxone®/Catena® (INN: idebenone) in patients with DMD have been published in The Lancet (Lancet 2015; 385: 1748-57. Online publication: http://dx.doi.org/10.1016/S0140-6736(15)60025-3).
The results of the DELOS trial demonstrated that Raxone/Catena significantly reduced the annual decline in Peak Expiratory Flow (PEF as percent predicted, PEF%p) by 66% compared to patients taking placebo.
Other respiratory function endpoints such as Forced Vital Capacity (FVC) and Forced Expiratory Volume (FEV1) corroborated these results and showed a consistent pattern with treatment differences supporting efficacy of Raxone/Catena over placebo in the preservation of respiratory function. Researchers concluded that Raxone/Catena represents a new treatment option for DMD patients.
"Publication of the DELOS trial outcome in The Lancet, one of the most prestigious medical journals worldwide, is an extraordinary tribute to this first ever successful phase III trial in DMD", said Gunnar M. Buyse, MD, PhD, Professor of Child Neurology at the University Hospitals Leuven (Belgium) and Principal Investigator for the DELOS trial and lead author of the publication. "It's also a tribute to the hard work of so many scientists, patients and families involved in the 10 years of innovative research in which we have brought idebenone from the lab bench to the patient. Statistically significant and clinically relevant outcomes of primary and secondary endpoints coherently demonstrated that Raxone/Catena reduced the loss of respiratory function and that it was safe and well tolerated. I am very enthusiastic about the positive data from the trial which demonstrate that this drug represents a suitable treatment option to ameliorate a life-threatening complication of the disease."
"With morbidity and mortality in DMD being associated with progressive restrictive lung disease and irreversible loss of lung function, these findings represent an important treatment effect and are of major clinical relevance for patients with DMD", added Craig McDonald, MD, Professor and Chair of the Department of Physical Medicine & Rehabilitation at UC Davis (USA), investigator of the DELOS trial and co-author of the Lancet publication.
"The degree of slowing of respiratory function loss demonstrated in DELOS is of major clinical relevance for patients with DMD", commented Nicholas Coppard, PhD, SVP Development at Santhera. "Based on this benefit and its well-established safety profile, we are very excited about the prospects of Raxone/Catena as a treatment option for DMD patients and we are currently preparing the regulatory filing dossier for application of marketing authorization both in the US and Europe."