NeuroVive Pharmaceutical AB has completed recruitment of healthy subjects for the second part of an ongoing Phase 1a/b study testing its oral treatment candidate KL1333 for primary mitochondrial disease.
The company also announced that it will soon begin the third part of this three-part, double blind, randomized, placebo-controlled, single and multiple dose study (NCT03888716).
Parts A and B include healthy volunteers only and, according to study design, must be completed before initiation of part C, which will include patients with mitochondrial disease. The study is taking place in Leeds, United Kingdom. Contact information is available here.
In part A, eight healthy subjects were randomly assigned to receive either 25 mg of oral KL1333 while fasting or after consuming a standard high-fat breakfast.
In part B, which just completed recruitment, 16 healthy volunteers were divided into two groups of eight. In each of these groups, six subjects received multiple ascending doses of KL1333, while the other two received a placebo. Subjects will be assessed 15 days after treatment initiation and five days after their final dose. Researchers have not disclosed details on how many doses participants will receive.
KL1333 is a modulator of the cellular levels of NAD+, a central co-enzyme in the cell’s energy metabolism. It has been shown to increase mitochondrial energy output, reduce lactate accumulation, lower the formation of harmful free radicals, and have long-term benefits on energy metabolism, such as the formation of new mitochondria. So far, no safety issues have been reported.
After evaluating results from the first two parts, investigators can start recruiting patients with primary mitochondrial disease for part C.
In part C, a total of eight mitochondrial disease patients will receive KL1333 on a dose no higher than the highest well-tolerated dose in part B. Patients will take KL1333 daily over 10 days and be evaluated on days 4 and 8, and 5 days after their final dose.
“We look forward to be able to start the exciting final part of the study where KL1333 for the first time will be given to patients with primary mitochondrial disease,” Magnus Hansson, PhD, chief medical officer and vice president of preclinical and clinical development at NeuroVive, said in a press release.
The investigational molecule is being developed to treat primary genetic mitochondrial disorders, including mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes; Kearns-Sayre syndrome; chronic progressive external ophthalmoplegia; Pearson syndrome; and myoclonic epilepsy with ragged red fibers syndrome.
“During the first half of  we also plan to initiate a natural history study in primary mitochondrial disease patients as part of our Phase 2 program, as a bridge to our clinical efficacy study, to optimize the patient selection criteria and use of endpoints,” said Erik Kinnman, MD, PhD, CEO at NeuroVive. “These are important steps in the opportunity of developing KL1333 towards a life changing treatment for patients with primary mitochondrial disease.”