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Mitochon Pipeline
Mitochon Pipeline
Compounds
Preclinical Efficacy
IND Enabling Activities
Phase I
Phase II

Amyotrophic Lateral Sclerosis (ALS)

ALS is disease of selective motor neuronal loss in the brain and spinal cord for voluntary muscles with median age of symptoms starting at 55.
High levels of Oxidative Stress/Calcium, ER stress and Mitochondrial Dysfunction found in All variants including SOD1 patients.
Loss of motor results in muscle wasting, eventually respiratory muscle loss and subsequently death within ~3-years upon diagnosis.
SOD1 model treatment 8 weeks, once-per-day orally with a ultra-low dose of MP101 at 0.5mg/kg, a human equivalent dose of 2mg/day.
Our Data Shows:
Improved motor function and strength versus placebo.
Durable improvement over time versus placebo.
Preservation of neuromuscular junction similar to Wild-type mice.

MP-101 Improves Motor Scores in Accelerating Rod Coordination Test (0.5mpk MP-101, n=5)

MP101 Significantly Reduces Oxidative Stress » Mitochon Pharmaceuticals
Time to fall using accelerating speeds.

MP201 Treatment 4-18 Weeks Reserved NMJ Structure And Muscle Innervation.

MP101 Significantly Reduces Oxidative Stress » Mitochon PharmaceuticalsMP101 Significantly Reduces Oxidative Stress » Mitochon PharmaceuticalsMP101 Significantly Reduces Oxidative Stress » Mitochon Pharmaceuticals
Tibialis Anterior
Pre-synaptic: Neurofilaments
Post-synaptic: Acetylcholine Receptor

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