Research & Development
Enrollment Completed in Stealth BioTherapeutics Study of Elamipretide for Treatment of Barth Syndrome
9 March 2018 - - Newton, Massachusetts-based clinical-stage biopharmaceutical company Stealth BioTherapeutics has completed enrollment in the TAZPOWER clinical trial, a phase 2/3, randomized, double-blind, placebo-controlled crossover study to evaluate the effects of daily treatment with elamipretide in 12 patients with genetically confirmed Barth syndrome, the company said.
Barth syndrome is a rare and debilitating genetic mitochondrial disease that is estimated to affect one in 200,000 to 400,000 individuals worldwide. There are no treatments approved by the US Food and Drug Administration.
Stealth initiated the TAZPOWER study in July 2017. The primary endpoint of the study is change in distance walked during the six-minute walk test and secondary endpoints include additional functional assessments, patient-reported outcomes and safety. I
Privately held Stealth is focused on the development of therapeutics for diseases involving mitochondrial dysfunction.
Based on encouraging preclinical and early clinical data, the company is investigating its lead product candidate, elamipretide, in three primary mitochondrial diseases: PMM, Barth syndrome, and Leber's hereditary optic neuropathy, as well as in heart failure and dry age-related macular degeneration.


Related Headlines