Mitochon Pipeline
Compounds
MP101
MP201
Pre-Clinical Efficacy
IND Enabling Activities
Phase I
Phase II
Huntington Disease
- Neurodegenerative disorder resulting in chorea, eventual dementia and premature death. Genetic mutation in huntingtin gene, a “tri-nucleotide repeat expansion disorder” of CAG (glutamine).
- Transgenic mouse, N171-82Q, expressing a cDNA encoding the N-terminal fragment of a 171 amino acids of the huntingtin gene with 82 glutamine repeats.
- Similar to humans, these transgenic mice have the “premanifest” stage prior to showing movement disorders, striatum, intranuclear inclusion / neuritic aggregates and premature death.
- HD mice were treated ~ 17-weeks with MP101 by daily oral gavage.
OUR DATA SHOWS: MP101 Preserves Brain Volume, Medium Spiny Neurons and General Neurons.
MP101 Preserves Movement.
MP101 Significantly Protects from Reactive Oxygen Species in the Cortex.
MP101 Preserves Brain Volume, Medium Spiny Neurons and General Neurons.
Wu, B, Experimental Neurology, 2017 March 27
MP101 Improves Motor Function in a N171-82Q HD Mouse of Huntingon's Disease at Age 26W with 17W Treatment.
The values are the mean and SEM. One-way ANOVA tests were used. n=14-16. *p<0.005 versus the WT Placebo group; #p<0.005 versus the HD Placebo group.
Wu, B, Experimental Neurology, 2017 March 27
MP101 attenuates brain atrophy (at 26 weeks of age) in N171-82 HD mice.
Wildtype (WT) with normal cortex (Ctx) and Striatum (Str). HD placebo (HD) shows loss in both Ctx and Str. MP-101 shows minor loss in Ctx and Str.
MP101 Significantly Reduces Oxidative Stress in Cortex of HD Mouse Model.
MP101 significantly lowers Isoprostanes, a biomarker of Oxidative stress/free radicals production, in HD mice to Equivalent levels in the cortex of Wildtype.
Wu, B, Experimental Neurology, 2017 March 27
MP201 Neuroprotection.
- MP201 prevents RGC loss during experimental optic neuritis.
- Known effects on mitochondria and oxidative stress suggest common mechanism of neuroprotection.
- Reduces demyelination without suppressing inflammation.
- Preserves visual function. (Optokinetic Responses)