Idebenone is Santhera's lead compound and is being developed in a number of orphan mitochondrial and neuromuscular indications, areas of high medical need with no current therapies.
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. For additional information on the mode of action of idebenone, click here Haefeli et al: NQO1-dependent redox cycling of Idebenone: Effects on cellular redox potential and energy levels. PLoS ONE. 2011; 6(3): e17963 and here Erb et al: Features of Idebenone and related short-chain quinones that rescue ATP levels under conditions of impaired mitochondrial complex I. PLoS ONE. 2012; 7(4): e36153.
Santhera's product pipeline is protected by use patents and orphan drug designations.
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