PIPELINE

MTC-1203 for Huntington’s Disease

A selective peptide inhibitor of pathological mitochondrial fragmentation (fission) and dysfunction

Maintains neuronal health through improved mitochondrial functions, ATP production and reduced oxidative stress in culture models of HD

Does not affect normal mitochondrial fission or fusion

Improves cell viability and mitochondrial health in HD and PD patient-derived neuronal cells

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Demonstrates in vivo efficacy (improves motor and cognitive function, delays progression) in mouse HD, PD and AD models

Reduces level of Htt protein aggregates and neuronal loss (causes of the pathology) in a mouse HD model brain

Benefits of treatment associated with a decline in peripheral biomarkers.

Reduces neuroinflammation (microglia activation and inflammatory cytokine elevation)

Exerts no discernable adverse effects in normal mice even after months of treatment.

MTC-1203, which is in preclinical development, is expected to be the first disease-modifying therapeutic for treating HD.

MTC-1203 for Parkinson’s Disease

Reduces mitochondrial fragmentation and improved its function in PD cell models carrying the G2019S mutant

Significantly reduces localization of Drp1 to the mitochondrial in PD patient derived dermal fibroblasts carrying the LRRK2 G2019S mutation, the most prevalent familiar PD mutation

Improves neuronal structure and function in dopamine secreting neurons created from patient cells carrying the LRRK2 G2019S mutation

Efficacious in an animal model of PD induced by the PD neurotoxin MPTP. Efficacy is manifested by reduced neuronal loss and improved locomoter behavior