We are a privately held clinical-stage biotechnology company focused on the development of therapeutics for diseases involving mitochondrial dysfunction. We believe there is a strong rationale for our lead product candidate, elamipretide, in indications in these diseases based on encouraging preclinical and early clinical data. We are investigating elamipretide in three primary mitochondrial diseases - primary mitochondrial myopathy (PMM), Barth syndrome and Leber's hereditary optic neuropathy (LHON), for which we have both Fast Track and Orphan Drug designation. We are also investigating elamipretide in dry age-related macular degeneration, the leading cause of blindness in the elderly. We have a broad pipeline of novel mitochondria-targeting compounds we are evaluating for new indications, including neurodegenerative diseases.

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