What is KL-1333?
KL-1333 is a drug that is currently being investigated as a potential treatment for mitochondrial disease. It is being developed in partnership by Yungjin Pharma and NeuroVive.
How does it work?
KL-1333 may be able to increase the amount of energy produced by the mitochondria. It does this by boosting the levels of an enzyme called NAD+ (nicotinamide adenine dinucleotide). NAD+ has several roles in the cell and is important for energy production.
What is it supposed to do?
KL-1333 is believed to improve energy production in several ways. These include increasing the availability of NAD+ for energy production. The increase in NAD+ is also thought to enhance the production of new mitochondria, a process known as mitochondrial biogenesis. Together this could increase the amount of energy the mitochondria can produce, which could reduce some of the symptoms of mitochondrial disease.
Is it a cure?
Unfortunately not. KL-1333 cannot correct the underlying genetic defect that causes mitochondrial disease.
What types of Mitochondrial Disease could it treat?
Although preclinical research has suggested that KL-1333 has the potential to treat mitochondrial disease, this still needs to be investigated in patients. It is currently in clinical development for treatment of different types of mitochondrial disease and if successful, KL-1333 could be used to treat any inherited mitochondrial disease.
Have there been any trials of KL-1333?
A ‘first in human’ study to investigate the safety of KL-1333 in healthy male volunteers after a single oral dose of the drug was completed in 2018. This phase 1 study, which also investigated how well the drug is tolerated and what affect it has on the body, showed positive results. A second phase I trial is ongoing at sites in the UK. This is a dose escalation study in healthy volunteers and in patients with mitochondrial disease. Results are expected in 2020.
In December 2017, KL-1333 was granted ‘orphan drug’ designation in the EU and in April 2018 by the FDA in the US. This aims to advance both the evaluation and development of drugs that have the potential to treat rare diseases and should make it easier for the drug to gain marketing approval.
Are there any medical papers on KL-1333?
A paper on the effects of KL-1333 on cells from mitochondrial disease patients was published in 2018 (https://www.frontiersin.org/articles/10.3389/fneur.2018.00552/). Also, a newsletter was published by NeuroVive in May 2017 and describes the KL-1333 project.