Santhera Pharmaceuticals has announced that its Phase III DELOS study of orally administered Catena®/Raxone® (INN: idebenone) in patients with Duchenne Muscular Dystrophy (DMD) met the primary endpoint and achieved its primary objective of delaying the loss of respiratory function compared to placebo.
The Phase III, double-blind, placebo-controlled DELOS study randomized 65 DMD patients who were 10-18 years of age and who were not using concomitant corticosteroids.
The study met the primary endpoint, the difference between Catena®/Raxone® and placebo in the change from baseline to week 52 in Peak Expiratory Flow (p=0.04). Peak Expiratory Flow is a measure of respiratory muscle strength, the decline of which is a major contributing factor to morbidity and mortality in DMD.
Catena®/Raxone® (900 mg/day) was safe and well tolerated with adverse event rates comparable to placebo. Other endpoint analyses are ongoing and results of these will be disclosed shortly.
"We are thrilled by these results which are consistent with the findings of our Phase II DELPHI and DELPHI Extension studies," commented Thomas Meier, CEO of Santhera. "As acknowledged by clinicians and regulatory authorities, preservation of respiratory function is a major benefit for patients with DMD. On the basis of these results we will approach the US and European authorities for discussions on the most expeditious regulatory pathway to approval."
"I am very enthusiastic about the positive outcome for the DELOS trial. This is tremendously good news for patients with DMD since it indicates that Catena®/Raxone® can mitigate respiratory weakness and dysfunction," commented Gunnar Buyse, M.D., Ph.D., Professor of Child Neurology at the University Hospitals Leuven (Belgium) and Principal Investigator for the DELOS study.
Santhera holds global commercialization rights to the DMD program, which has been granted orphan drug designation and patent protection in the US and EU.