Khondrion Raises €5 Million to Advance Mitochondrial Disease Therapy
Nijmegen, Tuesday, 6 May 2025.
A recent €5 million funding boost is set to propel Khondrion’s Phase 3 trial for a pioneering mitochondrial disease treatment, highlighting significant patient safety and symptom improvement.
Breakthrough in Mitochondrial Disease Treatment
Khondrion, a clinical-stage biopharmaceutical company based in the Netherlands, has secured a significant innovation credit of up to €5 million from the Netherlands Enterprise Agency to advance their groundbreaking mitochondrial disease therapy [1]. The funding will support Wave 1 of their pivotal Phase 3 clinical trial of sonlicromanol, a first-in-class small-molecule therapy targeting m.3243A>G Primary Mitochondrial Disease (PMD), which represents the most common genetic form of mitochondrial disease [1].
Promising Clinical Results
The therapy has already demonstrated significant promise in earlier trials. Patients participating in Khondrion’s extensive Phase 2 programme have reported continued and progressive improvements in key symptoms [1]. The drug’s safety profile has been particularly encouraging, with patients in the named patient program successfully receiving sonlicromanol for more than two and a half years [1]. This development comes at a crucial time, as the global mitochondrial disorders treatment market is projected to grow at a compound annual growth rate (CAGR) of 10.4% from 2025 to 2032 [4].
Strategic Timeline and Market Impact
The Phase 3 trial is scheduled to commence in the second half of 2025 [1], marking a significant milestone in mitochondrial disease treatment development. CEO Jan Smeitink has emphasized that this governmental support, combined with ongoing investor commitment, validates the urgent need for innovative treatments in the PMD community [1]. The development aligns with broader market trends, where increasing patient numbers and advancements in early diagnostic technologies are driving growth in the mitochondrial disorder treatment sector [4].