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Compounds

MP101

Huntington’s Disease

Duchenne Muscular Dystrophy

Optic Neuritis

MP201

Multiple Sclerosis

Parkinson’s Disease

Pre-Clinical Efficacy

IND Enabling Activities

Phase I

Phase II

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  • Mitochon Pharmaceuticals Mitochon Pharmaceuticals
    • MP 101:
    • Duchenne Muscular Dystrophy

    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
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