Khondrion | Khondrion Secures up to €5 Million Innovation Credit from Dutch Government plus Additional Funding from Current Investors to Advance Phase 3 Clinical Trial in m.3243A>G Primary Mitochondrial Disease
Summary
Government support, alongside private investments, funds first wave of pivotal Phase 3 trial of sonlicromanol for m.3243A>G Primary Mitochondrial Disease (PMD)NIJMEGEN, the Netherlands 6 May 2025 Khondrion, a clinical-stage biopharmaceutical company pioneering therapies for primary mitochondrial disease (PMD), today announced it has been awarded an Innovation Credit of up to 5 million from the Netherlands Enterprise Agency, a government agency that supports entrepreneurs.This vital government funding, matched by a significant investment from Khondrions committed private backers, will fund Wave 1 of the companys pivotal Phase 3 clinical trial of sonlicromanol. This leads to a wide range of severe symptoms across multiple organ systems, including the brain, muscles, and digestive tract. With sonlicromanol, we are advancing a potentially transformative therapy designed not only to alleviate symptoms but also to modify the course of this devastating disease, offering new hope to patients and families who have long been waiting for effective solutions.With the Phase 3 trial on the horizon, Khondrion is taking a bold step towards changing the lives of patients suffering from mitochondrial disease and creating meaningful opportunities for partners ready to join this journey.Sonlicromanol: one of the most advanced drug candidates in development for PMDSonlicromanol has been investigated in four clinical trials. Adult patients participating in our extensive Phase 2 programme have reported continued and progressive improvements on key symptoms (see Smeitink et al., Brain 2025; https://doi.org/10.1093/brain/awae277). Signs and symptoms of these can include cognitive problems, learning disabilities, blindness, deafness, heart failure, diabetes, fatigue, intolerance to exercise, muscle weakness and gait problems, and stunted growth.Originally referred to as MELAS syndrome (mitochondrial encephalopathy, lactic acidosis, and strokelike episodes), primary mitochondrial disease associated with the m.3243A>G variant in the mitochondrial genome is now considered to include a spectrum of phenotypes including classic MELAS, MIDD syndrome (maternally inherited diabetes mellitus and deafness), MP (mixed phenotypes) and CPEO (chronic progressive external ophthalmoplegia).