• Khondrion received FDA clearance for its IND application to initiate a pivotal Phase 3 trial of sonlicromanol in adult patients with primary mitochondrial disease (PMD).
• The Phase 3 trial will assess sonlicromanol's efficacy in treating chronic fatigue and muscle weakness, common symptoms in PMD patients with the m.3243A>G mutation.
• Sonlicromanol, a brain-penetrant redox-modulator with anti-inflammatory properties, has shown promising results in Phase 2b studies, demonstrating durable improvements in fatigue and muscle weakness.
• The trial is expected to enroll 150 adult patients and will use the Neuro-QoL Fatigue questionnaire and the Five Times Sit-to-Stand test as primary endpoints.