Topline trial results with the oral agent idebenone (Catena/Raxone, Santhera Pharmaceuticals) in patients with Duchenne muscular dystrophy (DMD) show that treatment delays the loss of respiratory function, the company has announced.
The results of the phase 3 DuchEnne Muscular Dystrophy Long-term IdebenOne Study (DELOS) show the drug significantly improved respiratory function from baseline vs placebo.
"I am very enthusiastic about the positive outcome for the DELOS trial," commented DELOS principal investigator Gunnar Buyse, MD, PhD, professor, child neurology, University Hospitals Leuven, Belgium, in a company release. "This is tremendously good news for patients with DMD," since it indicates that the drug can mitigate respiratory weakness and dysfunction.
"We are thrilled by these results, which are consistent with the findings of our phase II DELPHI and DELPHI Extension studies," commented Santhera CEO Thomas Meier, PhD, in a press release. "As acknowledged by clinicians and regulatory authorities, preservation of respiratory function is a major benefit for patients with DMD."
On the basis of these new results, the company will approach US and European regulatory authorities for discussions on the most expeditious regulatory pathway to approval, said Dr. Meier.
The double-blind DELOS trial included 65 ambulatory and nonambulatory patients with DMD aged 10 to 18 years from 23 centers in the United States and Europe who were not using corticosteroids. These patients were randomly assigned to receive 900 mg of idebenone per day or placebo, taken orally with meals, for 52 weeks.
The primary endpoint was change from baseline in respiratory function, as measured by peak expiratory flow. The decline in respiratory muscle strength is a major contributing factor to morbidity and mortality in DMD.
The study found a statistically significant (P = .04) difference between the active treatment and placebo on this primary endpoint.
Researchers also report that the drug was safe and well tolerated, with adverse event rates similar to those seen with placebo.
Analysis of additional study endpoints, such as other respiratory parameters, muscle strength, motor function, and quality of life, is ongoing. The company said that it expects to announce further trial results shortly.
Santhera holds global commercialization rights to the DMD program, which has been granted orphan drug designation and patent protection in the United States and Europe.
A genetic disease inherited in an X-linked mode, DMD is characterized by a loss of the protein dystrophin, leading to progressive muscle weakness and wasting. According to information on the Santhera Web site, the average age at DMD onset is 3 to 5 years, with a loss of ambulation in teenage patients. There is currently no approved treatment for DMD.
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Cite this: Drug Delays Loss of Respiratory Function in DMD - Medscape - May 13, 2014.