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New Compound Gets Orphan Drug Status for Huntington’s Disease

MP-101 Has Potential to Drastically Alter Disease Progression

Mitochon Pharmaceuticals’ MP-101 has received an orphan drug designation from the FDA for the treatment of Huntington’s disease. The company said that MP-101–a mitochondrial targeted compound–has been shown to protect both spiny neurons and general neurons and minimize brain-volume loss in patients with Huntington’s disease. Combined, these effects could significantly alter the disease’s progression in patients.

MP101 and MP201 are mitochondrial targeted, once-a-day, oral therapies that safeguard cells from the damage caused by numerous degenerative processes. In preclinical studies, both compounds showed significant protective and functional benefits in disease models. These include brain-volume sparing in Huntington’s disease; protection from demyelination in multiple sclerosis; and preserving dopamine neurons in Parkinson’s disease.

Mitochon will be initiating phase 1 studies in normal healthy volunteers this year and phase 2 studies are expected to take place in 2020.

Source: Biospace, March 5, 2019

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