This Biotech Targets Mitochondria to Treat Inherited Diseases

As we come to the end of the global Mitochondrial Disease Awareness Week, it’s time to visit Khondrion. This Dutch company is developing drugs to treat rare diseases such as MELAS syndrome by targeting defective mitochondria.

Mission: To develop small molecule drugs able to treat Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS) syndrome. This condition is caused by abnormalities in the parts of the cell that produce energy, called mitochondria. 

MELAS syndrome is a mitochondrial disease usually diagnosed in childhood. The condition is caused by mutations in DNA within the mitochondria, which can be inherited or occur spontaneously. Since the vast majority of cells in the body carry mitochondria, the symptoms can be diverse, including seizures, headaches and stroke-like symptoms. The disease is progressive and eventually fatal.

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“[Mitochondrial diseases] are complex rare diseases, often overlooked or misdiagnosed because of the wide range and varying severity of symptoms, Jan Smeitink, Khondrion’s founder, told me. “And these symptoms can be common to other diseases, things like migraine, diabetes, deafness, and fatigue. So, it means reaching a final diagnosis may take years.”

Khondrion’s lead candidate sonlicromanol is an anti-inflammatory drug designed to modulate the action of proteins called peroxiredoxins. These proteins are antioxidants that clear up harmful molecules called free radicals, which can damage cells in mitochondrial diseases. 

Khondrion will test sonlicromanol in a phase IIb trial in adult MELAS patients later this year, and is expected to enter phase I in child patients in a year’s time.

Founded in 2012, the company has raised over €3M in funding, including a €2.3M grant from the EU last year.

melas syndrome khondrion mitochondria

What we think:

Mitochondrial diseases such as MELAS are currently very hard to address for two reasons. First, doctors can find it hard to spot the signs of these diseases, and there are no approved treatments. Expected to reach the market by 2023 if successful, Khondrion’s drug could provide a much-needed treatment to rebalance the chemistry in the mitochondria.

Other companies are also developing small molecule drugs to address this rare disease area. One example is Shift Bioscience, which aims to treat MELAS syndrome by making healthy mitochondria outcompete diseased mitochondria, and even possibly reverse the aging process. Other companies such as the US-based Sarepta Therapeutics are developing gene therapies to tackle mitochondrial diseases such as Duchenne Muscular Dystrophy. 

Another way to tackle difficult mitochondrial diseases is to prevent them from being inherited. To achieve this, researchers have developed a technique where babies have three parents. Approved in the UK, this controversial technique involves using mitochondria from a healthy donor to replace mitochondria in an embryo carrying mutations for mitochondrial diseases.


Images from Shutterstock

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