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Mitochondrial diseases are a group of rare and often debilitating conditions, currently lacking effective treatment options, except for one specific form, Leber hereditary optic neuropathy. These diseases urgently require therapies to alleviate symptoms and improve patients' quality of life.
A new investigational drug, sonlicromanol, has shown promise in addressing these needs. As a modulator of oxidative and reductive distress, sonlicromanol works by selectively inhibiting microsomal prostaglandin E1 synthase activity, targeting key metabolic and inflammatory pathways involved in mitochondrial disease.
Phase 2b Clinical study overview
Researchers recently completed a Phase 2b clinical trial program to evaluate the safety, tolerability, and efficacy of sonlicromanol in adults with the m.3243A>G mutation, a common cause of primary mitochondrial disease. In total, 27 patients participated in the RCT, and 15 transitioned to the EXT study, with 12 completing the analysis.
Sonlicromanol shows promise as a safe and well-tolerated treatment for mitochondrial disease, with significant improvements observed in mood, cognition, fatigue, and physical functioning, especially in patients with greater baseline impairments. Long-term treatment further enhanced quality-of-life outcomes across various measures, highlighting its potential as a transformative therapy. Importantly, the FDA has approved the initiation of a Phase 3 trial, marking a critical step toward making this treatment available to patients.
About the publication
Jan Smeitink, Just van Es, Brigitte Bosman, Mirian C H Janssen, Thomas Klopstock, Grainne Gorman, John Vissing, Gerrit Ruiterkamp, Chris J Edgar, Evertine J Abbink, Rob van Maanen, Oksana Pogoryelova, Claudia Stendel, Almut Bischoff, Ivan Karin, Mahtab Munshi, Anne Kümmel, Lydia Burgert, Christianne Verhaak, Herma Renkema (2024). Phase 2b program with sonlicromanol in patients with mitochondrial disease due to m.3243A>G mutation. Brain. 2024 Nov 6:awae277. doi: 10.1093/brain/awae277.
