Khondrion announces sonlicromanol Phase IIb progress supporting Phase III development in MELAS spectrum disorders
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Tuesday, November 22, 2022
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Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.
Key Points:
- Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.
- In line with previous studies, sonlicromanol was found to be safe and well tolerated in the Phase IIb programme, with no serious adverse effects.
- These longer-term patient data are, therefore, instrumental in providing valuable insights for the design of the upcoming Phase III trial.
- One of the most advanced disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has recently completed a Phase IIb study in adult patients with m.3243A>G MELAS spectrum disorders.