A European clinical trial shows benefits of sonlicromanol on several symptoms of diseases associated with the m.3243A>G mutation of the mitochondrial MT-TL1 gene.
First identified in 1990, the m.3243A>G mutation in the MT-TL1 gene quickly emerged as one of the most common causes of mitochondrial DNA-related diseases. It is now known to be involved, for example, in MELAS, CPEO, and MIDD syndromes.
A phase II clinical trial in Europe evaluated sonlicromanol, developed Par the Dutch laboratory Khondrion, in 27 adults with mitochondrial disease due to the m.3243A>G mutation. They took either sonlicromanol or a placebo orally for one month, and then 15 of these participants received sonlicromanol for one year.
More marked benefits in the long term
The primary endpoint chosen for this trial was attention. Attention did not improve significantly across all participants. However, the drug candidate did provide significant benefits for patients with the most severe attention deficits at the start of the study. Sonlicromanol also improved other symptoms such as depression and certain cognitive disorders, particularly memory. The second phase of the study, involving long-term treatment in 15 participants, confirmed these benefits and revealed others, including quality of life, fatigue, balance, and pain.
Sonlicromanol will now be evaluated against placebo in the framework of the Khenerfin clinical trial. Still in preparation, this time it will bring together 150 participants carrying an m.3243A>G mutation and suffering from mitochondrial myopathy (muscle damage). It is planned to start in 2025.
See the summary of the source publication:
Phase 2b program with sonlicromanol in patients with mitochondrial disease due to m.3243A>G mutation.