Khondrion

Khondrion is a clinical-stage biopharmaceutical company focused on discovering and developing therapies for primary mitochondrial diseases. Based on proprietary science and a deep understanding of mitochondria in health and disease, the company is advancing its lead candidate, sonlicromanol. This compound was initially studied in adult patients with primary mitochondrial diseases, specifically those with genetically confirmed m.3243A>G PMD mutations in mitochondrial DNA associated with classical MELAS syndrome, MIDD syndrome, CPEO, and mixed phenotypes. This mutation represents the most common mutation associated with primary mitochondrial diseases. Based on the favorable benefit-risk profile observed for sonlicromanol in clinical studies conducted to date, Khondrion is proceeding with preparations for a pivotal Phase 3 registration study.