Sonlicromanol primary mitochondrial disease program
phase 3drug program · high · Sun Feb 01 2026 00:00:00 GMT+0000 (Coordinated Universal Time)
Develop sonlicromanol for children and adults with primary mitochondrial disease, including m.3243A>G-related disease, by improving clinically relevant symptoms such as cognition, fatigue, mood, balance, pain, and quality of life.
Orally bioavailable small-molecule reductive and oxidative distress modulator; clinical program includes randomized placebo-controlled studies, open-label extension, and the Phase 3 KHENERFIN study.
Phase 3 KHENERFIN study is listed as an ongoing clinical study for sonlicromanol in mitochondrial disease.
Phase 2b data in adults with m.3243A>G primary mitochondrial disease reported good tolerability and favorable safety. The randomized primary endpoint did not reach statistical significance, but treatment-effect signals were observed in more affected patients across cognition, depression, fatigue, pain, balance, and quality-of-life measures; long-term extension treatment showed more pronounced changes from baseline.
KHENERGYC pediatric sonlicromanol study
phase 2drug program · high · Mon Feb 01 2021 00:00:00 GMT+0000 (Coordinated Universal Time)
Evaluate sonlicromanol in children from birth to 17 years with genetically confirmed mitochondrial disease affecting oxidative phosphorylation enzymes and motor symptoms.
Randomized, placebo-controlled, double-blind Phase II clinical study exploring pharmacokinetics, safety, and efficacy of sonlicromanol in pediatric mitochondrial disease.
ClinicalTrials.gov record for the KHENERGYC study published for pediatric Phase II evaluation.
Ferroptosis and intracellular iron research in primary mitochondrial disease
exploratoryresearch program · medium
Investigate whether primary mitochondrial disease fibroblasts are selectively sensitive to ferroptosis and whether intracellular iron contributes to that vulnerability.
In vitro studies of PMD patient fibroblasts and healthy control skin fibroblasts using ferroptosis inducers, iron supplementation, intracellular iron-pool analysis, gene-expression analysis of iron regulators, and complementation of defective OXPHOS in an NDUFS7 mutation cell line.
2026 publication reported that increased labile iron predisposes PMD fibroblasts to ferroptotic cell death.
PMD fibroblasts showed more pronounced sensitivity to class 1 ferroptosis inducers and discriminatory ferroptosis triggered by exogenous iron. Complementation of defective OXPHOS restored ferroptosis sensitivity and labile iron-pool levels, supporting a functional relationship between OXPHOS deficiency, iron homeostasis, and ferroptosis.
Goal Attainment Scale outcome-measure research in primary mitochondrial disease
exploratorybiomarker · medium
Assess whether the Goal Attainment Scale can capture patient-relevant functional change in adults with primary mitochondrial disease and guide future clinical-trial outcome measurement.
Retrospective analysis of data from a double-blind, randomized, placebo-controlled exploratory Phase IIA crossover sonlicromanol trial in 18 adult m.3243A>G patients; GAS goals were mapped to ICF categories and compared with other outcome measures including COPM, fatigue, depression, walking, quality-of-life, and disease-scale measures.
2026 publication reported construct-validity findings and implementation lessons for GAS use in future PMD trials.
GAS showed weak to moderate associations with fatigue and depressive symptoms, moderate correlations with COPM, limited overlap with conventional measures, and suboptimal implementation quality, suggesting potential but requiring standardized methodology and further validation.
Sonlicromanol Post-COVID fatigue study
phase 2drug program · medium
Evaluate sonlicromanol for Post-COVID fatigue, consistent with Khondrion's mitochondrial dysfunction therapeutic focus.
Phase II clinical study targeting Post-COVID fatigue.
Company site analysis states Khondrion is running a Phase II study targeting Post-COVID fatigue.
