EMA Approves Mitochon's Phase I/IIa Neurodegeneration Biomarker Trial

Mitochon Pharmaceuticals has received approval from the European Medicines Agency (EMA) to initiate a Phase I/IIa biomarker study involving patients with Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Huntington’s Disease (HD), and Alzheimer’s Disease (AD). The company's MP101, a daily oral medication designed to penetrate the brain and stimulate mitochondria, is expected to enhance the survival and functionality of the central nervous system in a 14-day pilot study. The primary objectives are to ensure the safety of the drug in the targeted patient groups and to observe significant alterations in biomarkers associated with each disease. Positive outcomes from this trial could pave the way for more extensive Phase IIb clinical trials, potentially leading to the development of the first therapy specifically targeting mitochondrial issues in these debilitating conditions.

Dr. John G. Geisler, co-founder and Chief Scientific Officer of Mitochon, expressed enthusiasm for investigating the hypothesis that mitochondrial dysfunction underlies a majority of neurodegenerative diseases. He anticipates that long-term, low-dose treatment with MP101 could rectify mitochondrial problems and positively influence biomarkers across all four diseases.

MP101 and its counterpart MP201 are oral therapies that target mitochondria and are administered once a day. They have demonstrated the ability to protect cells from a variety of degenerative processes, both genetic and non-genetic, including autoimmune and injury-induced damage. In preclinical trials, these drugs have shown remarkable protective and functional benefits. For instance, they have been shown to preserve brain volume in HD, protect axons from demyelination in MS, safeguard neuromuscular junctions in ALS, prevent short-term memory loss in AD, and shield brain cells from damage due to traumatic brain injury.

Established in 2015, Mitochon Pharmaceuticals is dedicated to developing treatments for stealthy diseases by modulating mitochondrial physiology, with a focus on neurodegeneration, neuromuscular, and neuro-trauma conditions. The company's lead programs, MP101 and MP201, leverage the mitochondria's capacity to provide extensive neural protection. These compounds slightly increase energy expenditure, which strengthens cell survival, akin to the benefits of fasting and exercise. Additionally, they stimulate the production of Brain-Derived Neurotrophic Factor (BDNF), a neurotrophin vital for cognition, growth, and repair.