The poster entitled "Elamipretide Improves Functional Assessments when Compared to the Natural History Progression of Cardiomyopathy-related Disease Symptomatology in Patients with Barth Syndrome: A TAZPOWER Analysis" presents data from a Phase 3 retrospective natural history control trial comparing the cardiac and functional effects of elamipretide on Barth syndrome patients at week 72 of the open label extension portion to a natural history control (NHC) cohort derived from a 9-year long
About the Phase 3 Natural History Control Study
The Phase 3 retrospective natural history control study compared findings on functional assessments collected through weeks 36 and 48 and, as an FDA-requested post hoc analysis, week 72 of the open-label extension portion of the TAZPOWER trial with findings on the same assessments for up to 19 prognostically matched natural history control subjects, for whom data were collected longitudinally between 2012 and 2019 by investigators at Johns Hopkins Kennedy-Krieger Institute. The primary endpoint was the change in distance walked during the 6MWT. Secondary endpoints included all additional functional assessments for which there were natural history data, including measurements of muscle strength, balance, and the time to complete an assessment requiring patients to sit and stand five times in succession. A multi-domain responder analysis was also conducted to assess the percentage of patients experiencing at least a 10% clinically meaningful gain (or loss) on these endpoints. Additionally, this study compared changes in left ventricular stroke volume observed during the open-label extension portions of the TAZPOWER trial with changes observed in the natural history dataset. All types of heart failure are associated with a deterioration of stroke volume, which is a key component of both cardiac output and ejection fraction.
About the TAZPOWER Study
TAZPOWER is a Phase 2/3 crossover study evaluating the effects of daily subcutaneous (SC) treatment with elamipretide in 12 patients with genetically confirmed Barth syndrome followed by an open-label treatment extension. Part 1 was a 28-week crossover trial of patients randomized to elamipretide 40 mg SC daily for 12 weeks, followed by SC placebo daily for 12 weeks, or vice versa, separated by a 4-week washout. Part 2 is an open-label assessment for up to 192 weeks of functional and cardiac assessments, patient-reported outcomes and safety/tolerability.
The primary endpoints included change in distance walked during the 6MWT and change in patient-reported outcomes. Secondary endpoints included additional functional assessments, patient-reported outcomes, echocardiographic assessments of cardiac structure and function, and safety/tolerability.
About Barth Syndrome
Barth syndrome is an ultra-rare genetic condition characterized by cardiac abnormalities often leading to heart failure, muscle weakness, recurrent infections, delayed growth and reduced life expectancy, typically due to premature cardiac-related death. Barth syndrome occurs almost exclusively in males and is estimated to affect one in 300,000 to 400,000 individuals worldwide at birth. There are currently no FDA-approved therapies for patients with Barth syndrome.
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, 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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