The U.S. Patent and Trademark Office (USPTO) recently notified Mitochon Pharmaceuticals of its allowance on the first patent that the company says will help it proceed with the continued clinical development of MP101 as a treatment for Huntington’s disease (HD) and others.
MP101 is one of two leading programs Mitochon is developing. MP101 and MP201 specifically target the mitochondria to provide broad neural protection. The compounds elicit mild increases in energy expenditure, resulting in strengthening cellular survival, and is similar to the positive effects observed with fasting and exercise.
MP101 and MP201 also induce an important neurotrophin called Brain Derived Neurotrophic Factor (BDNF), which is involved in cognition and neural growth and also improves calcium handling.
In addition, the patent allowance will help advance MP101 as a treatment for refractory epilepsy and Duchenne’s muscular dystrophy (DMD). MP201 is being studied as a treatment for optic neuritis, multiple sclerosis and Parkinson’s disease.
In preclinical studies, MP101 and MP201 have shown protective and functional benefits in disease models. In HD, these benefits include brain volume sparing, preservation of medium spiny neurons and general neurons, preservation of movement and protection from reactive oxygen species in the cortex. Mitochon intends to initiate Phase 1 clinical trials in healthy volunteers in 2018 and expects to enter Phase 2 studies in 2019.
“We are delighted to receive our first patent allowance. It clearly demonstrates the novelty of our work in developing mitochondrial targeted therapies for neurodegeneration. By harnessing the power of the mitochondria, our compounds are able to protect cells from the destructive effects of degeneration. Importantly, this patent allowance will help pave the way for human clinical development, with the hope of helping the millions of people with devastating, neurological diseases,” Roberto Alonso, co-founder and CEO of Mitochon, said in a press release.
The patent (US 15/002,531) is called “Induced Expression of Brain Derived Neurotrophic Factor (Bdnf) For Treatment of Neuromuscular, Neurodegenerative, Autoimmune, Developmental And/Or Metabolic Disease.”
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