Mitochon Pipeline
Compounds
MP101
MP201
Pre-Clinical Efficacy
IND Enabling Activities
Phase I
Phase II
Duchenne Muscular Dystrophy
- Loss of Dystrophin X-Chromosome Results in muscle atrophy in young boys.
- Aggressive phenotype, with leg braces at the age of ~8, wheel chair at ~10 and death ~20 due to either loss of heart or diaphragm muscle.
- Mdx mouse model on a DBA background provides the early inflammatory “storm” and muscle atrophy seen similar in humans.
- Starting at 4-weeks of age, mice were treated 3mths with MP101.
OUR DATA SHOWS: MP101 Significantly Preserves Diaphragm Strength (Primary endpoint).
MP101 significantly improves muscle bundles and reduced calcium overload.
MP101 Significantly Improves Diaphragm Strength in mdx/DBA Mice after 12-weeks Treatment relative to both Placebo and Prednisolone.
MP101 Significantly preserves diaphragm strength in mdx-DBA mice treated with MP101 from 4-weeks of age to 16-weeks compared to placebo and prednisone.
MP101 Significantly Improves Muscle Bundle Organization with Less Pockets of Inflammation and Calcifications vs. Steroid.
mdx Mice after 12-weeks of Treatment