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Mitochon Pharmaceuticals Awarded Orphan Drug Designation for ALS

Blue Bell, Pennsylvania — is delighted to announced that it was awarded Orphan Drug Designation by FDA for its mitochondrial targeted compound, MP-101 for treating Amyotrophic Lateral Sclerosis (ALS). This important allowance, along with the Company’s open IND will help secure the path forward for the continued clinical development of MP-101 in ALS 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). ALS is a devastating motor neuron disorder with no disease modifying therapies on the market today. In a model of ALS, hSODG93A, MP-101 has been shown to significantly sustain motor neuron skills similar to non-ALS mice. The benefits may stem from the pleiotropic pharmacology of lower damaging ROSs and calcium overload due to ER stress that lead to apoptosis, while increasing BDNF (neurotrophin/myokine) involved in repair.  This our second orphan designation (Huntington’s Disease 2019) and an important milestone for Mitochon and we are excited to move into patients and help the thousands of families with ALS.  MP-101 and MP-201 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 protective and functional benefits in disease models. These include: brain volume sparing in Huntington’s Disease; axonal protection from demyelination in Multiple Sclerosis, preserving dopamine neurons in Parkinson’s Disease and significant reduction of damage after brain trauma (hearing or TBI). Mitochon initiated Phase I studies in normal healthy volunteers in 2020 and with a series of Phase II studies in sporadic ALS, Huntington’s Disease and Alzheimer’s Disease planned in the near future.

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.