You are here

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

Recent Headlines

Disrupting Gut Microbiome Could Be Key
Drug Boosts Levels of Natural Endocannabinoids
Judicious Use of Antibiotics May Not Be Enough To Defeat Bacteria That Carry On By Going Into a Dormant State
KRAS Oncogene Is a Problematic Target So Researchers Are Trying Workdarounds
Understanding Neural Ensembles in Infralimbic Cortex May Lead To Improved Addiction Treatment
Vitamin E Found in Samples Around the Country
Study Links Them to Premature Death
Nag With Texting and a ‘Winners Circle’
How Serotonin and Fluoxetine Affect Microbiota Residing in the Gut