Phase 2b Trial for Mitochondrial Diseases has Begun

Khondrion has just announced that the very first patient has been dosed in their Phase 2b study for mitochondrial diseases called KHENERGYZE. This patient population has a high unmet need and researchers are hopeful about the promise of this investigative therapy.


The trial will take place at three different sites in Germany, the UK, and the Netherlands. It is double-blind, placebo-controlled, randomized examination of Sonlicromanol in patients who have a mutation in mitochondrial transfer RNALeu(UUR). This mutation can cause Mitochondrial diseases including mitochondrial encephalomyopathy, stroke-episodes, lactic acidosis, maternally inherited diabetes or deafness, and others.

Its primary aim is to investigate the effect the therapy has on cognitive functioning, something that affects everything we do as humans.  This will be measured through a Cogstate visual identification test.

The therapy is a small molecule taken orally. Researchers believe it to be one of the most advanced investigative treatments for this group of diseases. It has already been granted Orphan Drug Designation for many forms of mitochondrial diseases in both the United States and Europe. In previous studies it has been shown to be safe, well-tolerated, and have clinical efficacy.

This trial is still currently recruiting patients. You can find out more about the specific details of this trial here. If things continue to go according to plan, results are expected by the second half of this year.  

You can read more about this investigative therapy here.

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