Khondrion

Khondrion is a clinical stage pharmaceutical company focusing on the development of treatments for mitochondrial (-related) diseases and conditions

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.

Team
Jan Smeitink
Startup activities

Pre-Seed Grant 2010

info@khondrion.com