EMA approves biomarker study of mitochondrial stimulator for neurodegenerative diseases

The European Medicines Agency has approved enrollment for a phase 1/2a biomarker study to examine a once-daily, oral, brain penetrant mitochondrial stimulator to treat a range of neurological and neuromuscular conditions.

According to a release from Mitochon Pharmaceuticals, its MP101 therapeutic aims to improve central nervous system survival and function and will be tested in a 14-day pilot study to assess safety and significant change in disease-specific biomarkers for those with amyotrophic lateral sclerosis, MS, Huntington’s disease and Alzheimer’s disease.

In preclinical studies, MP101 and its companion, MP201, exhibited protective and functional benefits in disease models. According to the company, these include brain volume sparing in Huntington’s disease, axonal protection from demyelination in MS, protection of neuromuscular junction in ALS, short-term memory loss prevention in Alzheimer’s and protecting brain cells from traumatic brain injury.

“We are delighted for the opportunity to explore this provocative idea that most, if not all neurodegenerative diseases are rooted in mitochondrial dysfunction,” John G. Geisler, PhD, co-founder and chief scientific officer of Mitochon, said in the release. “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.”

Successful completion of the current pilot study is expected to provide the basis for long term phase 2b clinical studies, Mitochon said in the release.