Mitochondrial medicine company NeuroVive Pharmaceutical AB (STO:NVP) and Yungjin Pharm Corporation Ltd (KRX:003520) jointly announced on Monday 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.

The company said 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 or MAD) in healthy volunteers and patients. Detailed analysis of the complete data set is ongoing.

NeuroVive together with a leading global contract research organization (CRO), will now rapidly progress the planning and preparation of the phase I MAD study of KL1333 in healthy volunteers and genetic mitochondrial disease patients. This study is expected to commence in the second half of 2018.

The MAD study will consist of two parts, namely a dose escalation in healthy volunteers and a multiple dosing of patients with genetic mitochondrial disease. The purpose of the study is to further investigate safety and pharmacokinetics of KL1333 prior to initiating a phase II efficacy clinical trial. The study will be conducted at sites in the UK and results are expected in the first half of 2019.

Also, the KL1333 SAD study is a dose block-randomised, double-blind, placebo-controlled, single -dose, dose-escalation, phase I clinical study. 25 ,50, 100, 400, 600, 800 mg of KL1333 was administered in 60 healthy volunteers to investigate pharmacokinetics, safety and tolerability.