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Mitochon Pharmaceuticals Awarded Orphan Drug Designation

Blue Bell, Pennsylvania (BUSINESS WIRE) — Mitochon Pharmaceuticals today announced that it was awarded Orphan Drug Designation by FDA for its mitochondrial targeted compound, MP-101 for treating Huntington’s Disease. This important allowance, along with the Company’s open IND will help secure the path forward for the continued clinical development of MP101 in Huntington’s Disease and it further strengthen Mitochon’s exclusivity for MP-101, a repurposed molecule, that was granted an issued US patent in November, 2017 (US 15/002,531).

“We are delighted to receive our first orphan drug designation for Huntington’s Disease, a devastating neurodegenerative disorder with no disease modifying therapies on the market today. By harnessing the power of the mitochondria, MP-101 has been shown to protect both spiny neurons and general neurons as well as minimize brain volume loss from the destructive effects of Huntington’s Disease. Together these effects have the potential to significantly change the progression of the disease in patients. This designation is an important milestone for Mitochon and we are excited to move into patients and help the thousands of families with Huntington’s Disease,” said Robert Alonso, co-founder and CEO 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, non-genetic, auto-immune and injury). In preclinical studies, these compounds have exhibited striking protective and functional benefits in disease models. These include: brain volume sparing in Huntington’s Disease; axonal protection from demyelination in Multiple Sclerosis, and preserving dopamine neurons in Parkinson’s Disease. To understand the critical step of translation, Mitochon will initiate Phase I studies in normal healthy volunteers in 2019 and expected to be in Phase II studies in 2020.

About Mitochon Pharmaceuticals
Mitochon was founded in 2014 with the mission to develop treatments for insidious diseases through the modulation of mitochondrial physiology, with applications to neurodegeneration, neuromuscular, and developmental diseases. Mitochon’s lead programs, MP101 and MP201, specifically harnesses the power of the mitochondria to provide broad neural protection. 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 and neural growth. Mitochon is supported by Ben Franklin Technology Partners Southeastern PA, an initiative of the Pennsylvania Department of Community and Economic Development funded by the Ben Franklin Technology Development Authority.