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 plans to re-submit a Marketing Authorisation Application in Europe for RaxoneŽ as first treatment option in Leber's Herditary Optic Neuropathy. In addition, the compound is being investigated in Duchenne Muscular Dystrophy (Phase III DELOS trial), Primary Progressive Multiple Sclerosis (Phase II IPPOMS study, conducted by the US National Institutes of Health) and MELAS syndrome (Phase II MELTIMI study, conducted the Columbia University of New York City).
Santhera's product pipeline is protected by use patents and orphan drug designations.
CatenaŽ and RaxoneŽ are trademarks of Santhera Pharmaceuticals.