The trial is being led in collaboration with Yungjin Pharm in Korea, and explores KL1333 as a treatment for a series of mitochondrial diseases, such as MELAS (mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-led episodes).
Mitochondria are cell components that produce energy. Their dysfunction can affect several of our bodies’ functions, including our brain, nervous system, and muscles.
The report states that the first part of the study has been successful, as drug behavior data aligned with expectations. No adverse safety concerns were found and the study’s remaining higher-dose cohorts have now been approved by the Korean medicinal authority, the Ministry of Food and Drug Safety (MFDS)
“We are very excited about the progress of the first KL1333 clinical study. Along with the recent positive opinion on European orphan drug designation, it brings us one step closer to initiating our own clinical phase Ib study, and to our ultimate goal of providing a treatment opportunity to patients with different genetic mitochondrial disorders, where there is a high medical need and in most cases no specific treatments available,” Magnus Hansson, MD, PhD, chief medical officer and vice president of preclinical and clinical development at NeuroVive, said in a press release.
KL1333 is a modulator of the cellular levels of NAD+, a central co-enzyme in the cell’s energy metabolism. In preclinical models, KL1333 was shown to increase mitochondrial energy output, reduce lactate accumulation, lower the formation of free radicals, and have long-term benefits on energy metabolism, such as the formation of new mitochondria.
In the current study, KL1333 is being investigated for its pharmacokinetics (drug behavior in the body), safety, and tolerability in healthy volunteers. The first part of the study included dose levels of 25 and 50 mg. With both doses having been deemed safe, the MFDS has approved dose escalation and an update to the study’s design.
“The results from the first part of the study are positive and the approval from the MFDS is of crucial importance for KL1333 and the continuation of its global development program. In close collaboration with our partner NeuroVive, we have a great possibility to develop KL1333 into a novel treatment opportunity for patients with genetic mitochondrial disorders, such as MELAS,” said Soo-Hyun Lew, MD, executive director of development at Yungjin Pharm.
In November, NeuroVive announced that KL1333 is likely to receive orphan drug designation after the European Medicines Agency’s Committee for Orphan Medicinal Products decided to recommend the regulatory designation. A final approval is expected by the end of the year.
A Phase 1b study is planned to be initiated by NeuroVive in Europe and/or in the United States in 2018.