Date: 2014-11-25
Type of information: Clinical research agreement
Compound: Catena®/Raxone® (idebenone)
Company: Santhera Pharmaceuticals (Switzerland) Parent Project Muscular Dystrophy (USA - NJ)
Therapeutic area: Genetic diseases - Neuromuscular diseases - Rare diseases
Type agreement: clinical research
Action mechanism: benzoquinone analog/quinone. Raxone®/Catena® (idebenone), a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1), is capable of transferring electrons directly onto complex III of the mitochondrial electron transport chain, thereby circumventing the complex I defect and restoring cellular energy levels in retinal ganglion cells and promoting recovery of visual acuity. Nerve and muscle cells, including heart muscle cells, are particularly energy-demanding and are, therefore, more prone to rapid cell damage or death due to mitochondrial dysfunction. Through preserving mitochondrial function and protecting cells from oxidative stress, it is believed that Raxone®/Catena® can prevent cell damage and increase the production of energy within impaired nerve and muscle tissue in Friedreich's Ataxia and Duchenne patients.
Disease: Duchenne Muscular Dystrophy
Details: * On November 25, 2014, Santhera Pharmaceuticals and Parent Project Muscular Dystrophy (PPMD), the leading US advocacy organization working to end Duchenne Muscular Dystrophy (DMD), will collaborate on a benefit/risk study in DMD. The study will focus specifically on patient and caregiver preferences regarding pulmonary therapies in the disease, and will be based on data from Santhera\'s successful phase III clinical trial with Catena®/Raxone® (idebenone). Earlier this year, PPMD convened a broad coalition of over 80 stakeholders to draft and submit the first-ever patient advocacy-initiated guidance for a rare disease to the FDA to help accelerate development and review of potential therapies for DMD. One of the recommendations in the guidance was to create partnerships between patient groups and industry to study the benefit/risk preferences of the disease community to better inform the company\'s new drug application. Pursuant to this recommendation, Santhera and PPMD have now initiated such a partnership and collaborate to study patient and caregiver benefit/risk preferences for pulmonary therapy with Santhera\'s Catena/Raxone. The study will be based on data from Santhera\'s successful Phase III (DELOS) trial which provided clear evidence of a clinical benefit for Catena/Raxone in delaying the loss of respiratory function in DMD patients not using concomitant glucocorticoid steroids. The preservation of respiratory function is considered to be of major clinical importance. DELOS was a Phase III, double-blind, placebo-controlled trial which randomized 64 European and US patients, 10-18 years of age, to receive either Catena/Raxone tablets or matching placebo. The trial met its primary endpoint and demonstrated that Catena/Raxone can delay the loss of respiratory function in patients not taking concomitant glucocorticoid steroids. Detailed data from the DELOS trial were presented recently at the 19th International Congress of the World Muscle Society in Berlin (Germany).
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