Date: 2013-06-06
Type of information: Licensing agreement
Compound: idebenone
Company: Santhera Pharmaceuticals (Switzerland) National Institutes of Health (NIH) (USA)
Therapeutic area: Autoimmune diseases - Neurodegenerative diseases
Type agreement: licensing
Action mechanism: Mitochondrial dysfunction together with aberrant formation of reactive oxygen species are believed to underlie at least partially the development of progressive CNS tissue destruction in ppMS. Idebenone, a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinoneoxidoreductase (NQO1) is capable of transferring electrons directly onto complex III of the mitochondrial electron transport chain, thereby restoring cellular energy levels. In addition, idebenone is a powerful anti-oxidant. In view of the hypothesized involvement of mitochondrial dysfunction, and particularly the reduced activity of the mitochondrial electron transport chain in patients affected by ppMS, idebenone is a rational treatment choice for this disease based on its pharmacological properties including its ability to cross the blood-brain barrier.
Disease: primary progressive Multiple Sclerosis (ppMS)
Details: * On June 6, 2013, Santhera Pharmaceuticals has announced that it has obtained an exclusive license from the National Institutes of Health (NIH) to its rights on a patent granted in the USA for the use of idebenone for the treatment of primary progressive Multiple Sclerosis (ppMS). The NIH is investigating the efficacy of Catena® (idebenone) in ppMS in a placebo-controlled Phase II clinical trial. Lead by the Neuroimmunology Branch of the National Institute of Neurological Disorders and Stroke (NINDS), the NIH is investigating the efficacy of Catena® (idebenone) in patients with ppMS in a double-blind, placebo-controlled Phase II clinical trial (IPPoMS trial). Adult ppMS patients up to 65 years of age with disability ranging from none to moderately severe are eligible to enroll in the trial. This is a Phase I/II safety/efficacy trial with an adaptive trial design: one year of pretreatment baseline period serves the dual purpose of collecting patient-specific biomarkers of disease progression and collecting longitudinal neuroimaging and clinical data for selection of primary outcome measures. This baseline period is then followed by a double-blind, idebenone (2250 mg/day) versus placebo treatment phase for a total of 2 years. (NCT00950248).
Santhera is providing study medication under a clinical trial agreement which gives Santhera the rights to the results. Santhera has now obtained the exclusive rights to the use patent for idebenone in ppMS granted in the USA. Patients who complete the IPPoMS trial can enter into a 12-months open-label extension trial for which Santhera and the NIH recently signed a Materials Cooperative Research and Development Agreement (M-CRADA).
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