European Medicines Agency (EMA) Greenlights Mitochon Pharmaceuticals to Initiate Phase I/IIa Biomarker Study in Neurodegenerative Diseases



BLUE BELL, Pa.--(BUSINESS WIRE)-- Mitochon Pharmaceuticals today announced that it was awarded approval from EMA to begin enrollment for a Phase I/IIa biomarker study in Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Huntington’s Disease (HD) and Alzheimer’s Disease (AD) participants. Mitochon’s MP101, a once-daily, oral brain penetrant mitochondrial stimulator, that improves central nervous system survival and function will be used in this 14-day pilot study. The aim of the study is to show safety in the target patient populations and demonstrate meaningful changes in disease specific biomarkers. Successful completion of this study will provide the basis for long term Phase IIb clinical studies in ALS, Secondary & Primary Progressive MS, HD, and AD patients, leading to the first mitochondrial specific therapy for these truly insidious diseases.

“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,” said Dr. John G. Geisler, Ph.D., co-founder and CSO of Mitochon.

MP101 and MP201 are mitochondrial targeted, once-a-day, oral therapies that have been shown to shield cells from damage caused by a host of degenerative processes (genetic and non-genetic, auto-immune and injury). In preclinical studies, these compounds have exhibited striking protective and functional benefits in disease models. Benefits include: brain volume sparing in Huntington’s disease; axonal protection from demyelination in Multiple Sclerosis, protection of neuromuscular junction in ALS, blocking the loss of short-term memory in Alzheimer’s, and protecting brain cells from traumatic brain injury (TBI) such as severe concussions.

About Mitochon Pharmaceuticals
Mitochon was founded in 2015 with the mission to develop treatments for insidious diseases through the modulation of mitochondrial physiology, with applications to neurodegeneration, neuromuscular, and neuro-trauma. Mitochon’s lead programs, MP101 and MP201, specifically harnesses the power of the mitochondria to provide broad neural protection (MOA: These compounds elicit mild increases in energy expenditure that result in strengthening cellular survival – similar to the positive effects seen with fasting and exercise. These compounds also induce an important neurotrophin, Brain Derived Neurotrophic Factor (BDNF), involved in cognition, growth and repair. Additional Information:


Robert Alonso, President & CEO
Mitochon Pharmaceuticals, Inc.


Source: Mitochon Pharmaceuticals

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