NeuroVive Pharmaceutical has reported positive topline results from a Phase I trial evaluating KL1333 for the treatment of orphan genetic mitochondrial diseases.
South Korean pharmaceutical company Yungjin Pharm was also involved in the dose block-randomised, double-blind, placebo-controlled, single-dose, dose-escalation study.
During the trial, 25mg, 50mg, 100mg, 400mg, 600mg and 800mg doses of KL1333 were administered to 60 healthy volunteers to evaluate the drug’s pharmacokinetics, safety and tolerability.
Findings from the trial have showed that KL1333 has a highly favourable and very clear dose-proportional pharmacokinetic (PK) profile.
The trial did not report any serious adverse events (SAEs), and reported that only mild gastrointestinal adverse events (AEs) were at higher doses.
NeuroVive Pharmaceutical CEO Erik Kinnman said: “With the convincing safety profile of KL1333 and favourable PK data, we will confidently bring this promising programme forward in development with the highest priority.
“The next important step is the clinical Phase I multiple ascending dose (MAD) study, which will take us further towards the goal of offering a novel treatment to patients with severe genetic mitochondrial disease with few or no treatment options.”
The MAD trial will include two parts, including a dose escalation phase in healthy volunteers, and a multiple dosing of patients with genetic mitochondrial disease.
It will try to further investigate safety and pharmacokinetics of KL1333 before beginning a Phase II efficacy clinical trial.
The trial is expected to be carried out at different sites in the UK in the second half of this year and plans to publish results by the first half of next year.
For the MAD trial, NeuroVive will partner with a major global contract research organisation (CRO) to rapidly progress the planning and preparation of the MAD study.