Santhera’s Drug for Duchenne Muscular Dystrophy Proven Effective in Phase 3 Clinical Trial

Patrícia Silva, PhD avatar

by Patrícia Silva, PhD |

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Santhera Pharmaceuticals announced in a press release that an international team of researchers recently published in the journal The Lancet the results of the DELOS Phase 3 trial validating the efficacy and safety of Santhera’s Raxone/Catena (idebenone) in patients with Duchenne muscular dystrophy (DMD). The study is entitled “Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomized placebo-controlled phase 3 trial.

DMD corresponds to a muscular dystrophy characterized by a rapid progressive skeletal muscle weakness that leads to the degeneration of muscle cells and tissues, compromising locomotion and respiratory function, leading to breathing complications and cardiac problems. DMD is caused by mutations in the dystrophin gene and affects mainly boys. The majority of DMD patients require a wheelchair by the age of 12 and patients often succumb to the disease in their 20s.

“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 the study’s lead author Dr. Gunnar M. Buyse from the University Hospitals Leuven, Belgium and Principal Investigator for the DELOS trial in the press release. “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.”

Glucocorticoid steroids are currently the only available therapeutic option to slow muscle degeneration, however, their effect is only partial and use is limited due to serious steroid-related side effects. Santhera has developed a synthetic Raxone/Catena (idebenone) drug that ultimately leads to a supplementation on cellular energy levels in muscle cells and tissues. Previous trials have suggested that the drug had a positive impact on cardio-respiratory function, where patients treated with idebenone had a stable peak expiratory flow (PEF, a measure of expiratory muscle strength) while patients given the placebo had a PEF decline in the disease course.

Now, the DELOS Phase 3 double-blind, placebo-controlled trial comprising 64 DMD patients (aged 10 to 18 years old and not under glucocorticoids therapy) revealed that Raxone/Catena tablets given at 900 mg/day dose significantly decreased the PEF decline by 66% after 52 weeks of treatment in comparison to patients receiving a placebo drug. These results were corroborated by other respiratory parameters, namely forced vital capacity (FVC) and forced expiratory volume (FEV1).

The research team concluded that Raxone/Catena is an effective therapeutic option to preserve the respiratory function in DMD patients. “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” concluded a study’s co-author Dr. Craig McDonald from the University of California Davis Medical Center.

“The degree of slowing of respiratory function loss demonstrated in DELOS is of major clinical relevance for patients with DMD” added Dr. Nicholas Coppard from 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.”

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