The candidate generated promising results in preclinical studies, with Mitochon reporting that MP101 indicated favourable pharmacological and biochemical responses. Data showed that MP-101 preserves brain volume, medium spiny neurons, general neurons and limb movement, as well as reducing seizure duration.
According to GlobalData’s Pharma Intelligence Center, MP101 has been investigated in several clinical studies, including for optic neuritis, in normal healthy volunteers, and in Huntington’s disease patients.
In the announcement accompanying the approval, Mitochon’s co-founder and chief scientific officer (CSO) John Geisler said: “We are delighted for the opportunity to explore this provocative idea that most, if not all neurodegenerative diseases are rooted in mitochondrial dysfunction. We predict that chronic treatment with this unique platform, at micro-doses, will resolve mitochondrial issues and change important disease-specific biomarkers in all four indications similarly.”
In July 2023, InSilicoTrials and Axoltis teamed up to develop disease-modifying drugs for neurological disorders. Precision Biosciences filed a patent in September 2023 on a mitochondria-targeting engineered meganuclease for modifying human mitochondrial DNA.