According to a story from Malaysian Tribune, the pharmaceutical company Khondrion has just announced that the first patients have been dosed in its phase IIb clinical trial. This trial is testing the company’s experimental product candidate Sonlicromanol as a treatment for mitochondrial diseases. Khondrion is focused specifically on developing medications for mitochondrial diseases. The trial is beginning at three different disease centers in Europe.
About Mitochondrial Diseases
Mitochondrial diseases are a group of genetic disorders that causes the mitochondria not to function properly. The mitochondria are an essential organelle that is found in most types of cells in the body, with red blood cells being the only exception. They are responsible for generating energy for the cell. Mitochondrial diseases are usually caused by mutations of the mitochondrial DNA or the nuclear DNA. Symptoms tend to be the worst when the issue affects cells that use a lot of energy, such as the muscles or parts of the brain. These symptoms affect many aspects of bodily function and include poor growth, poor muscle coordination, dementia, neurological issues, muscle weakness, breathing disorders, vision problems, digestive disorders, hearing problems, disease of the kidney, liver, and heart, and learning disabilities. Treatment options are limited in number and in their effectiveness. To learn more about mitochondrial diseases, click here.
About The Study
This study will assess the attention domain score of cognitive functioning on patients, who in this trial a specific mutation affecting the mitochondrial transfer RNALeu(UUR) (MT-TL1m.3243A>G). This mutation is implicated in several different mitochondrial diseases including maternally inherited diabetes and deafness (MIDD) and mitochondrial encephalopathy, lactic-acidosis, and stroke-like episodes (MELAS).
Sonlicromanol has displayed capability in prior clinical studies and has also earned Orphan Drug status in both the EU and US. There is an urgent need for more effective, disease-modifying therapies for mitochondrial diseases and this drug has the potential to greatly improve outcomes for these patients if it continues to succeed in the clinic.
For more information about this clinical trial, click here.