Khondrion — a spin-out from Nijmegen Radboudumc — was recently awarded the prestigious H2020 SME Instrument European grant for the testing of a potential new medicine for mitochondrial disorders [ed. metabolic diseases]. CEO Jan Smeitink tells us about the latest developments.
There are approximately 250 known genetic deviations that lead to problems with energy metabolism. This can lead to a number of serious health issues. Khondrion is currently testing a group of patients who, because of a certain DNA deviation, suffer from issues with their cognition, and hearing from diabetes and strokes.
Tests in three countries
The medicine has been given the generic name Sonlicromanol by the World Health Organisation (WHO). In an earlier phase of testing, it was examined whether people could tolerate the drug, whether it was safe to use and if it has an effect. To all three questions the answer was: yes. Especially cognitively patients really noticed an improvement.
“We are currently working on the final preparations for the launch of a multicentre trial, a trial in which we will test patients in three different medical centres in Nijmegen, Netherlands, Munich, Germany and Newcastle, UK. We will test a group of 27 patients to see what the best dosage for Sonlicromanol is, and we are going to check whether we can confirm earlier positive findings”, says Smeitink.
We want to do things as efficiently as possible, but we obviously also need to guarantee the highest quality.
If everything goes well, the medicine could be on the market by 2023. It’s an important step for people who suffer from these serious conditions.
Partly because of the support from the EFRO programme, Khondrion will soon also be able to test the medicine for children: “When it comes to mitochondrial disorders, you want to be able to intervene as quickly as possible to prevent or even undo consequences.” Among children with mitochondrial diseases, the Leigh syndrome is prevalent — this is a progressive disease that takes the lives of 70% of children before the age of 10.
Apart from Sonlicromanol, Khondrion is working on the development of new and possibly even better treatments to tackle this group of diseases. The company is also expanding its organisation. “You need professionalisation in this global market, in order to be future-proof”, Smeitink feels. By now, Khondrion has started collaborating with over thirty partners all around the world. These are companies as well as universities.