9 November 2017 - - Radnor, Pennsylvania-based mitochondrial physiology specialist Mitochon Pharmaceuticals has been notified by the US Patent and Trade Mark Office of allowance on its first patent, "Induced expression of brain derived neurotrophic factor for treatment of neuromuscular, neurodegenerative, autoimmune, developmental and/or metabolic disease," the company said.
This patent allowance will help secure the path forward for the continued clinical development of MP101 in Huntington's Disease, optic neuritis and Duchenne's muscular dystrophy.
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).
These compounds elicit mild increases in energy expenditure that result in strengthening cellular survival similar to the positive effects seen with fasting and exercise, and also induce an important neurotrophin, brain derived neurotrophic factor, involved in cognition and neural growth. It also improves calcium handling associated with many diseases.
In preclinical studies, these compounds have exhibited protective and functional benefits in disease models such as brain volume sparing in Huntington's disease; axonal protection from demyelination in optic neuritis, and preserving diaphragm strength and heart morphology in Duchenne muscular dystrophy.
To understand the critical step of translation, Mitochon will initiate Phase I studies in normal healthy volunteers in 2018 and expected to be in Phase II studies in 2019.
Mitochon's mission is to develop treatments for insidious diseases through the modulation of mitochondrial physiology. The company's lead programs, MP101 and MP-201, specifically target the mitochondria to provide broad neural protection.