NIJMEGEN, The Netherlands — Khondrion today announced that the first patient has been dosed in the pivotal, randomized, placebo-controlled Phase 3 KHENERFIN study (NCT 06451757) evaluating the efficacy and safety of sonlicromanol, its investigational small molecule for m.3243A>G-related Primary Mitochondrial Disease.
“The initiation of patient dosing in our Phase 3 KHENERFIN study marks an important milestone in the development of a potential treatment for patients with m.3243A>G primary mitochondrial disease,” said Jasper Levink, CEO of Khondrion. “This trial is designed to rigorously evaluate the safety and efficacy of sonlicromanol in a larger, randomized setting, with endpoints reflecting key aspects of disease burden, building on the signals observed in our earlier clinical program.”
Dr. Mirian Jansen, head of the clinical metabolic department of the Radboudumc and PI in the KHENERFIN study noted: “m.3243A>G Primary Mitochondrial Disease is a progressive and debilitating condition with significant unmet medical need, affecting patients across multiple organ systems and often leading to increasing disability over time. As investigators, we are committed to advancing this Phase 3 study and working closely with patients to ensure high-quality data collection on outcomes that matter in daily life. This trial is designed to determine whether this investigational therapy can meaningfully impact patient function and fatigue.”
“The most burdensome symptoms m.3243A>G patients experience are fatigue and muscle weakness. These symptoms are often debilitating and impact every area of life. The initiation of the Phase 3 KHENERFIN study by Khondrion is a significant achievement and provides hope to the international mitochondrial disease community. As the Chair of International Mito Patients (IMP), which represents 25 mitochondrial disease patient advocacy groups across five continents, we are encouraged by the progress being made in research and drug development for those living with Primary Mitochondrial Diseases. We optimistically await the outcome of the trial,” stated Paula Morandi, Chair of IMP.
Jan Smeitink, Khondrion’s founder and CMO continued: “The KHENERFIN study will enroll up to 220 adult patients from 18 years and older across Europe, the United Kingdom and the US with genetically confirmed m.3243A>G Primary Mitochondrial Disease. Participants will be randomized 1:1 to receive sonlicromanol or placebo over a 52-week treatment period. Participants will receive 90 mg sonlicromanol or matching placebo as dispersible tablets twice daily. The study’s two independent primary endpoints are change in NeuroQoL Fatigue Short Form score and performance on the 5-times sit-to-stand test, reflecting key aspects of disease burden.”
About Khondrion
Khondrion is a clinical-stage biopharmaceutical company discovering and developing therapies targeting primary mitochondrial disease. Based on proprietary science and a deep understanding of mitochondria in health and disease, the company is advancing its lead drug candidate sonlicromanol. The compound is initially being investigated in adult Primary Mitochondrial Disease patients as genetically confirmed by the m.3243A>G PMD mutation in the mitochondrial DNA associated with Classic MELAS syndrome and MIDD syndromes, CPEO and mixed phenotypes. This mutation is the most frequently observed mutation associated with primary mitochondrial disease. Based on the favorable benefit-risk profile of sonlicromanol observed in the clinical studies performed so far, Khondrion is advancing the preparations of a registrational Phase 3 study.
Sonlicromanol and other compounds from Khondrion’s proprietary library have the potential to be developed for a wide range of other diseases and conditions affecting normal mitochondrial function and Khondrion is progressing preclinical R&D in several promising areas. To accelerate the discovery and development of its potential medicines for mitochondrial diseases, Khondrion collaborates with patient organisations worldwide as well as its global clinical and academic network.
To learn more visit www.khondrion.com or follow the company on X.
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