Mito research around the world


What is KH-176?

KH-176 is a novel drug that has been developed by a Dutch pharmaceutical company called Khondrion.

It is derived from vitamin E, which is an antioxidant found in many different foods.

How does it work?

KH-176 removes damaging chemicals that are produced during normal energy production. These oxygen species, known as free radicals, may be found at higher levels within cells when the mitochondria don’t work properly. KH-176 may also work by increasing the activity of different parts of the energy chain.

What is it supposed to do?

KH-176 is thought to protect cells from oxidative stress, which occurs when there are high levels of free radicals. Oxidative stress can cause considerable damage to cells and may contribute to some of the symptoms of mitochondrial disease. KH-176 may also 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. KH-176 cannot correct the underlying genetic defect that causes mitochondrial disease.

What types of Mitochondrial Disease could it treat?

KH-176 is currently under development for treatment of inherited mitochondrial diseases. If successful, it could be used to treat any inherited mitochondrial disease.

Have there been any trials of KH-176?

The results of a phase 1 clinical trial of KH-176 were published in 2015. They showed that the drug was well tolerated in healthy male volunteers and had a good pharmacokinetic profile (the affect the body has on the drug).

A ‘first-in-patient’ phase 2 clinical trial, known as the KHENERGY Study, was completed in 2017. This trial recruited 20 adult patients with a particular genetic error causing Mitochondrial Disease (the m.3243A>G mutation). The patients were randomly divided into two groups, with one group receiving KH-176 for 4 weeks and the other receiving a placebo. The groups were then swapped so that all patients received the drug at some point during the trial. This was a double-blind study, meaning that neither the researchers nor the participants knew who was receiving the drug or the placebo.

What is the latest update on this trial?

Preliminary results from the phase 2 trial were announced in December 2017.  They showed that the drug was well tolerated at a dose deemed safe from the phase I study and had no adverse side effects.  Clinical outcomes included a significant improvement in mood and alertness, whilst some patients also self-reported fewer migraines. In contrast, there were no significant improvements in functional outcome measures, such as gait analysis (the clinical assessment of walking), but the findings were still encouraging given the relatively short duration of the study.

What next?

Based on the outcomes of the phase 2 study, Khondrion have decided to continue with all necessary steps to enable the next stage of KH-176 development. This will include a large-scale phase 3 trial involving more patients. The aim of this is to further demonstrate the clinical benefits and safety of the drug and determine the optimal dosage of KH-176.

In addition to the KHENERGY Study, Khondrion announced in September 2016 that they had received a European Union grant to support the development of KH-176 for children with Mitochondrial Disease. The grant will be used to prepare and carry out a phase II clinical trial known as the KHENERGYC Study. No further information is currently available on this trial.

Is there more information on these trials?

More information can be found using the following link:

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