Khondrion is a clinical-stage biopharmaceutical company discovering and developing therapies targeting mitochondrial disease. Based on proprietary science and a deep understanding of mitochondrial dysfunction, the company is advancing its lead drug candidate sonlicromanol for the treatment of patients suffering from several primary mitochondrial diseases first. Currently being tested in Phase II studies in both children and adults, sonlicromanol is one of the most advanced disease-modifying mitochondrial medicines under development. More on this in Our Focus.
Our clinical development focus in adult patients is on cognitive functioning in MELAS spectrum disorders where sonlicromanol has demonstrated significant improvements in a Phase IIa study, with multi-centre Phase IIb and open label extension studies ongoing. MELAS spectrum disorders are some of the most frequently observed primary mitochondrial diseases, in which all patients are characterised by an underlying point mutation (m.3243A>G) in the maternally inherited MTTL1 gene.
Our clinical development focus in children with primary mitochondrial disease is on movement disorders with a Phase II study currently ongoing.
Sonlicromanol and other compounds from Khondrion’s proprietary library have the potential to be developed for a wide range of other diseases and conditions affecting normal mitochondrial function for which preclinical R&D is progressing.
To accelerate the discovery and development of its potential medicines for mitochondrial diseases, Khondrion collaborates with patient organisations worldwide and a global clinical and academic network.
Khondrion was founded by mitochondrial disease expert Prof. Jan Smeitink. Jan has devoted his entire career to the care of mitochondrial disease patients and the study of mitochondria in health and disease, until recently via his long-term affiliation with the Radboud University Medical Center in Nijmegen, The Netherlands. Khondrion’s mission is to develop disease-modifying treatments for this high impact, often early, fatal group of (ultra) rare disorders.
Khondrion has been operational since 2012 and is located in Nijmegen, The Netherlands.