Investigational Drug Details

Drug ID: D229
Drug Name: MitoQ
Synonyms: Mitoquinone cation; Mitoquinone ion
Type: Chemical drug
DrugBank ID: DB05063
DrugBank Description: Mitoquinone is based on a novel technology, targeted lipophilic cations, that transport and concentrate antioxidants into the mitochondria -- organelles inside cells that provide energy for life processes -- where they accumulate up to a thousand fold. In 2004, a genomic study of hereditary early-onset Parkinson's disease demonstrated a direct molecular link between mitochondrial dysfunction and the pathogenesis of Parkinson's disease. Mitochondrial dysfunction also has been shown to represent an early critical event in the pathogenesis of the sporadic form of Parkinson's disease. Clinical studies by the Parkinson's Study Group show that very high doses of an antioxidant called Coenzyme Q (which Mitoquinone effectively targets into mitochondria) appear to slow the progression of Parkinson's disease symptoms.
PubChem ID: 11388331
CasNo: 444890-41-9
Repositioning for NAFLD: Yes
SMILES: [P+](CCCCCCCCCCC1=C(C)C(=O)C(=C(C1=O)OC)OC)(c1ccccc1)(c1ccccc1)c1ccccc1
Structure:
InChiKey: OIIMUKXVVLRCAF-UHFFFAOYSA-N
Molecular Weight: 583.729
DrugBank Targets: --
DrugBank MoA: Mitoquinone is targeted to mitochondria by covalent attachment to a lipophilic triphenylphosphonium cation. Because of the large mitochondria membrane potential, the cations accumulate within cellular mitochondria up to 1,000 fold, compared to non-targeted antioxidants such as Coenzyme Q or its analogues, enabling the antioxidant moiety to block lipid peroxidation and protect mitochondria from oxidative damage. By selectively blocking mitochondrial oxidative damage, it prevents cell death.
DrugBank Pharmacology: Mitoquinone may help to prevent the nerve cell damage that leads to Parkinson's disease. It aims to slow or halt Parkinson's disease at its cause by tackling cell damage caused when mitochondria cease to function normally. It is anticipated that MitoQ will slow or arrest the progression of Parkinson's disease symptoms. Hepatits C virus can directly alter mitochondrial function, leading to increased reactive oxygen species (free-radical) production that can lead to scarring of the liver and cirrhosis. Mitoquinone could be used to halt or decrease liver inflammation and fibrosis progression, even in the absence of sustained virologic response. Mitoquinone directly affects the mitochondria in two steps: a targeting component directs the drug to the mitochondria; and an antioxidant component helps to prevent cell damage.
DrugBank Indication: Investigated for use/treatment in hepatitis (viral, C) and parkinson's disease.
Targets: --
Therapeutic Category: Antiparkinsonian drug
Clinical Trial Progress: Phase 2 terminated (NCT01167088: This study has been terminated due to poor participant recruitment)
Latest Progress: Under clinical trials