Khondrion is a clinical stage pharmaceutical company focusing on the development of treatments for mitochondrial (-related) diseases and conditions. Since its operational start in 2012 Khondrion’s team has successfully completed the preclinical development of its lead compound KH176 followed by a SAD and MAD study in healthy male volunteers and a clinical trial phase 2 study in adult patients with the MELAS-spectrum group of disorders. It is Khondrion’s ambition to become a world-leader in mitochondrial disease drug development. Khondrion has a strong multi-continental portfolio of granted and divisional patents with freedom to operate. KH176 has been granted orphan drug designations from both the EMA and the FDA.
Mitochondrial (-related) diseases and conditions
Mitochondrial diseases caused by either mutation in the nuclear or mitochondrial DNA affecting the mitochondria energy production machinery are progressive devastating multi-system disorders with an incidence of 1:5000 live births. The age of onset can be at age. Symptoms predominantly occur is organs and tissues with a high energy demand like brain and skeletal muscle causing e.g. epilepsy, migraine, stroke-like episodes, muscle weakness and exercise intolerance. There are no approved drugs on the market. Khondrion’s lead compound KH176 has been shown to be effective in both pre-clinical disease models and MELAS-spectrum disorders in man. Importantly, based on the mode of action KH176 is expected to positively influence a broad range of diseases and conditions. Examples include but are not limited to congenital muscular dystrophies, genetic forms of Parkinson disease and adverse drug effects.