Dutch pharmaceutical company Khondrion have just secured up to €5 million in government funding from the Netherlands Enterprise Agency – a huge boost that will power the final clinical trial of its potential new treatment, sonlicromanol. For families affected by a certain form of mitochondrial disease, there could be real hope on the horizon.
This funding, together with extra backing from Khondrion’s existing investors, paves the way for a Phase 3 clinical trial – the crucial final step before a treatment can be approved for widespread use. It’s an exciting leap forward in the search for the first approved treatment targeting m.3243A>G-related mitochondrial disease, the most common inherited cause of primary mitochondrial diseases (PMDs).
Right now, there are no approved therapies that tackle the root of this mitochondrial syndrome. That’s why this trial matters so much. If successful, sonlicromanol could become a life-changing treatment for people living with this progressive condition.
“This funding is not just a vote of confidence in our science – it’s a lifeline of hope for the PMD community,” said Jan Smeitink, CEO of Khondrion. “We are determined to bring forward a treatment that could change lives.”
Sonlicromanol has already been through several earlier trials, with many adult patients reporting improvements in key symptoms. Some have continued on the treatment for more than two years through a special access programme, providing valuable reassurance about its long-term safety.
During the Phase 3 trial, the treatment will be tested in a larger group of patients to confirm that it’s both safe and effective. If Phase 3 results are successful, it could lead to regulatory approval, meaning the treatment could become available to the wider patient community.
Study sites have not yet been confirmed for the Phase 3 trial, but please stay tuned for further updates by subscribing to our monthly newsletter or following us on our social channels. You can also check out our UK mitochondrial disease trials and research page for all current UK research studies.
Khondrion’s progress is being closely followed by families, clinicians and advocacy groups around the world. Reaching Phase 3 reflects encouraging early data, though much remains to be proven in larger patient populations before we see this as a viable treatment. However, with this new investment, the company is taking a major step closer to what could be the first ever approved treatment for m.3243A>G-related mitochondrial disease – a breakthrough the mito community has long been hoping for.