Khondrion Information

We are a Phase 3 ready clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease. Founded by Professor Jan Smeitink, a world-leader in mitochondrial medicine, we are advancing our proprietary science through a wholly-owned clinical and preclinical small molecule pipeline of potential medicines. Our priority is to rapidly develop our pipeline to deliver transformative medicines for patients living with mitochondrial disease. Our in-house scientists are driving innovative research projects and building a portfolio of promising compounds. We have active discovery programmes underway developing new therapies, biomarkers, diagnostic applications and new read-out technologies in the field of mitochondrial diseases. Our lead pipeline asset, sonlicromanol , is a potential Phase 3 ready first-in-class oral small molecule in clinical development to treat a range of primary mitochondrial diseases. It has been granted Orphan Drug Designation for for all inherited mitochondrial respiratory chain/oxidative phosphorylation disorders in the US en EU. We work in collaboration with patient organisations internationally as well as a global clinical and academic network to accelerate the discovery and development of our potential medicines for patients with mitochondrial diseases