Gili Hart, PhD
Mitoconix Bio Ltd is pioneering a novel disease modifying strategy for neurodegeneraitve diseases by improving mitochondrial health.
Disruptions of mitochondria ﬁssion (fragmentation) and fusion are implicated in many pathological conditions, including neurodegenerative diseases. Therefore, maintaining a proper balance between ﬁssion and fusion is a promising therapeutic approach to prevent cell damage and neurodegeneration. Mitoconix is developing therapeutic peptides designed to selectively inhibit excess mitochondria ﬁssion or enhance fusion for maintaining cellular integrity, thereby conferring neuroprotection and functional beneﬁt.
The target indications are Huntington’s disease (HD), Parkinson’s (PD) and ALS.
MTC-1203 is a disease-modifying therapeutic for neurodegenerative diseases. It is a selective peptide inhibitor of pathological mitochondrial fragmentation (ﬁssion) and dysfunction; that maintains neuronal health through improved mitochondrial function, ATP production and reduced oxidative stress in culture models of HD, PD and ALS. In vivo efﬁcacy demonstrated in animal models of HD, PD or ALS and beneﬁcial activity in patient-derived cells of HD, familial PD or ALS and sporadic or genetic Alzheimer’s disease (AD).
MTC-1203 A DISEASE-MODIFYING THERAPEUTIC FOR HUNTINGTON’S AND PARKINSON’S DISEASE
Demonstrates in vivo efﬁcacy (improves motor and cognitive function, delays progression) in mouse HD and PD models Reduces level of Htt protein aggregates and neuronal loss (causes of the pathology) in a mouse HD model Reduces neuroinﬂammation (microglia activation and inﬂammatory cytokine elevation) Beneﬁts of treatment associated with a decline in peripheral biomarkers Exerts no discernable adverse effects in normal mice even after 5 months of treatment.
MTC-1203 is in preclinical development and is expected to become a disease-modifying therapeutic for treating HD, entering clinical studies late 2018.