Problems in energy production are well-characterized in pediatric and adult heart failure and postulated to be centrally involved in impaired cardiac function in these patients. In this study, mitochondria and muscle fibers from the explanted failing left ventricle were isolated from adult and pediatric patients undergoing cardiac transplant, post organ transplantation. The effects of elamipretide on mitochondrial respiration and muscle function were determined in the laboratory. Mitochondrial respiration was decreased in heart tissue from patients with pediatric cardiomyopathy compared to explanted tissue from non-failing hearts. Study findings showed that these impairments in respiration were significantly improved with elamipretide treatment in the pediatric hearts. The study also demonstrated that elamipretide treatment improved both contraction and relaxation in muscle fibers isolated from failing hearts of patients with dilated cardiomyopathy.
"This study builds upon our previously published findings that elamipretide improves mitochondrial and muscle fiber function in heart tissue isolated from the failing human heart. The observation of improved mitochondrial function from pediatric cardiomyopathy is very encouraging as there are currently no therapies that target mitochondrial dysfunction in this patient population. These data are consistent with our observed effects of elamipretide across other types of heart failure including idiopathic cardiomyopathy and metabolic (muscular dystrophy) cardiomyopathy" said Dr.
We are a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction. Mitochondria, found in nearly every cell in the body, are the body's main source of energy production and are critical for normal organ function. Dysfunctional mitochondria characterize a number of rare genetic diseases and are involved in many common age-related diseases, typically involving organ systems with high energy demands such as the heart, the eye, and the brain. We believe our lead product candidate, elamipretide, has the potential to treat both rare metabolic cardiomyopathies, such as Barth syndrome, Duchenne muscular dystrophy and Friedreich's ataxia, rare mitochondrial diseases entailing nuclear DNA mutations, as well as ophthalmic diseases entailing mitochondrial dysfunction, such as dry age-related macular degeneration and Leber's hereditary optic neuropathy. We are evaluating our second-generation clinical-stage candidate, SBT-272, and our new series of small molecules, SBT-550, for rare neurological disease indications following promising preclinical data. We have optimized our discovery platform to identify novel mitochondria-targeted compounds which may be nominated as therapeutic product candidates or utilized as mitochondria-targeted vectors to deliver other compounds to mitochondria.
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