NeuroVive Pharmaceutical AB and Yungjin Pharm Corporation Ltd. jointly announced positive topline results after data base lock in the phase I single ascending dose (SAD) clinical study of KL1333, a novel treatment in clinical development for orphan genetic mitochondrial diseases. Review of the topline phase I data shows that KL1333 has a highly favourable and very clear dose-proportional pharmacokinetic (PK) profile. There were no serious adverse events (SAEs), and only mild gastrointestinal adverse events (AEs) were recorded at higher doses.

Based on the positive PK and safety results NeuroVive is moving rapidly to initiate the next study in Europe that will include repeated dosing (multiple ascending dose; MAD) in healthy volunteers and patients. Detailed analysis of the complete data set is ongoing. There is a huge unmet medical need for medicines that treat genetic mitochondrial diseases.

Patients can have severe symptoms in any organ and have significantly reduced life-expectancy. These diseases are rare diseases for which there are almost no registered medicines.