The randomized, single ascending dose trial (NCT03056209) was designed to investigate the pharmacokinetics (drug movement through the body), safety, and tolerability of KL1333.
In the trial, 60 healthy volunteers were given a single oral dose of either 25, 50, 100, 400, 600, 800 mg of KL1333 and monitored for two weeks.
A review of the trial’s data shows that KL1333 has a highly favorable and very clear dose-dependent pharmacokinetic profile.
Additionally, none of the participants experienced any serious adverse events. Some participants who were given high doses experienced mild gastrointestinal side effects. NeuroVive is continuing to do a deeper analysis of the complete data set.
Based on these positive pharmacokinetic and safety results, NeuroVive, in conjunction with a leading global contract research organization, is moving quickly to launch the next trial in Europe. This will be a multiple ascending dose trial testing KL1333 in healthy volunteers and genetic mitochondrial disease patients. It is expected to begin in the second half of 2018.
“With the convincing safety profile of KL1333 and favorable PK data, we will confidently bring this promising program forward in development with the highest priority. The next important step is the clinical phase I 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,” Erik Kinnman, NeuroVive’s CEO, said in a press release.
The multiple ascending dose trial, which will further investigate the safety and pharmacokinetics of KL1333, will include two different parts. The first part will be a dose escalation in healthy volunteers. The second part will include multiple dosing of patients with genetic mitochondrial diseases. The study will be conducted at sites in the U.K., with results expected in the first half of 2019.
There is currently a huge unmet need for therapies to treat genetic mitochondrial diseases. Patients with these diseases can have severe symptoms in any organ and can have a significantly reduced life expectancy.
“We are very excited about the results from the first in human clinical trial of KL1333 and see them as clearly promising for the continued clinical development of this important program together with NeuroVive,” said Chae J. Lee, Yungjin Pharm’s CEO and president.