San Francisco, California, United States
Daneil de Roulet, Nicholas Hertz
2017
Private company
5
Mitochondrial dysfunction
Parkinson's disease, Alzheimer's disease, Huntington's disease
small molecules drugs
MTK-458
Kinase-targeted drugs have been in development for more than thirty years and have made a large impact on the way we treat many human diseases. The great majority of these drugs work by inhibiting kinases – ie, reducing or eliminating their signaling activity. Mitokinin is taking a different approach to targeting kinases: we aim to increase the activity of active-form PINK1.
The levels of active-form PINK1 are very carefully regulated by the cell. PINK1 only becomes catalytically active on the surface of damaged mitochondria. Mitokinin’s therapeutics only boost the activity of active-form PINK1, and have no effect at all on the regulatory processes involved in activating or degrading PINK1. This therapeutic approach is inherently ‘light touch’: unlike constitutively inhibiting an important kinase, Mitokinin’s drugs merely increase the activity of an already-activated kinase.
Mitokinin’s lead candidate, MTK-458, has been extensively validated in in vitro and in vivo models of Parkinson’s disease. IND enabling studies are currently underway, and an IND filing is expected in 2020. In parallel, Mitokinin’s scientists are also investigating the activity of MTK-458 in models of Huntington’s disease, Alzheimer’s disease, and non-CNS diseases of aging where mitochondrial dysfunction is implicated.