New Study Strengthens Patient‑Centred Endpoint Selection for KHENERFIN Phase 3 Trial

2 Sep, 2026

Khondrion has announced the publication of an important qualitative study in Orphanet Journal of Rare Diseases examining the lived experience of adults with genetically confirmed m.3243A>G primary mitochondrial disease. The study provides patient‑derived evidence supporting the relevance of two key symptom domains — physical fatigue and mental fatigue — which have now informed endpoint selection for KHENERFIN, Khondrion’s pivotal Phase 3 trial of sonlicromanol.

Through concept‑elicitation and cognitive‑debriefing interviews conducted in the United States and the Netherlands, participants described 21 disease‑related symptoms and their impact on daily life, with physical fatigue, mental fatigue, hearing loss and pain among the most burdensome. The study confirmed that the Neuro‑QoL Fatigue Short Form appropriately captures core physical‑fatigue concepts, while patient feedback guided the development of a new PROMIS Mental Fatigue – Mitochondrial Disease Short Form.

For the mitochondrial community, this publication marks a meaningful step forward: it demonstrates how patient experience is shaping the design of KHENERFIN, ensuring that the trial evaluates outcomes that genuinely matter to adults living with m.3243A>G PMD. As Khondrion’s CEO Jasper Levink notes, “Patients are at the center of Khondrion… we listen to people living with mitochondrial disease and use that evidence to shape our pivotal programme.”

KHENERFIN (NCT06451757) will assess sonlicromanol’s efficacy and safety over 52 weeks, focusing on physical fatigue, lower‑limb function, and mental fatigue as a complementary domain. This study does not evaluate treatment efficacy, but it provides essential content‑validity evidence that strengthens the scientific and patient‑centred foundation of the Phase 3 programme.