A clinical trial application for a Phase 1 study of the investigational drug KL1333 in healthy volunteers and patients with genetic mitochondrial diseases has been approved by a regulatory agency in the UK. For more detailed information about this news, you can view the source press release at PR Newswire by clicking here.
Mitochondria are present in every cell in the human body and produce most of the energy we need to survive. Mitochondrial diseases are a group of disorders caused by mutations that affect the functioning of the mitochondria. As a result, cells in some areas of the body don’t have enough energy to carry out their normal functions. Mitochondrial disease can cause a wide range of different symptoms, depending on which cells are affected. Any organs can be affected, and the disorder may cause seizures, cognitive disabilities, poor growth, fatigue, vision and hearing loss, and many other symptoms. Since the effects of the disorder can vary so much between patients, it is often difficult for doctors to identify, and many people have to wait a long time to achieve a correct diagnosis.
KL1333, an investigational drug, is being developed by NeuroVive as a potential oral chronic treatment for mitochondrial disease. It is designed to affect cellular levels of NAD+, which is a coenzyme that plays an important role in energy metabolism in the cell. KL1333 has received Orphan Drug designation in both the US and Europe.
Research into KL1333
So far, researchers have carried out promising pre-clinical studies that found that KL1333 was associated with a higher mitochondrial energy output, reduced lactate accumulation, and a decrease in the formation of free radicals. The drug was also found to have long-term benefits for energy metabolism, including through mitochondria formation.
Now, KL1333 is being studied in Phase 1 clinical trials. The UK’s regulatory authority, the Medicines and Healthcare Products Regulatory Agency, has recently approved NeuroVive’s clinical trial application for a Phase I study of the drug in healthy volunteers and patients. The study, which is expected to take place in the UK and begin in the last quarter of 2018, will assess how safe and tolerable the drug is in humans, as well as how it is processed in the body (the pharmacokinetics).