Skip to main content

Stealth BioTherapeutics Presents Data for Elamipretide on Cardiac Function in Barth Syndrome

BOSTON, Oct. 17, 2019 / PRNewswire

Stealth BioTherapeutics (NASDAQ: MITO), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced the presentation of new data from the open-label extension portion of the Phase 2/3 TAZPOWER study evaluating elamipretide in patients with Barth syndrome. The findings, presented at the American Society of Human Genetics (ASHG) 2019 Annual Meeting in Houston, Texas, showed that treatment with elamipretide resulted in a 27% increase in average cardiac stroke volume, or the amount of blood pumped by the heart's left ventricle per contraction, from the trial baseline (40.8 mL) to week 36 (51.8 mL) of the open-label extension. Left-ventricular stroke volume is one of the primary determinants of cardiac output, or the volume of blood pumped by the heart, which is an important indicator of how efficiently the heart can meet the body's demands for perfusion to various organs. In unaffected adolescent boys, an average stroke volume of 85 mL would be expected.

Barth Syndrome Foundation Appoints New Director of Research, Erik Lontok PhD


This week, Barth Syndrome Foundation (BSF) announced the appointment of Erik Lontok, PhD, as the Foundation’s new Director of Research. Lontok will succeed BSF’s prior Director of Science, Matthew Toth, PhD, in a new role that has been established in order to leverage the past two decades of research partnerships and achievements toward the development of viable therapies for people with Barth syndrome. Trained as a biochemist at the University of California, San Francisco, Lontok joins BSF after serving as the Chief Science Officer of the Lipedema Foundation.

University Hospitals Bristol NHS Foundation Trust, University of Bristol and Barth Syndrome UK Initiate Phase 2 Study of Bezafibrate in Barth Syndrome

BRISTOL, England (PRWEB) July 24, 2019

University Hospitals Bristol NHS Foundation Trust (UHB), University of Bristol (UoB), Barth Syndrome UK and Barth Syndrome Foundation (BSF) recently announced the opening of CARDIOMAN, a Phase II trial evaluating bezafibrate in individuals affected by Barth syndrome, a rare, life-threatening mitochondrial disease caused by a mutation in the tafazzin (TAZ) gene. The trial, funded by an MRC and NIHR Partnership, is a randomized, placebo-controlled pilot trial to assess the efficacy and safety of bezafibrate to increase mitochondrial biogenesis and potentially modify the cellular ratio of monolysocardiolipin to L4- cardiolipin, a biomarker of Barth syndrome. The trial has been designed in collaboration with the UKCRC-registered Bristol Clinical Trials and Evaluation Unit (part of the Bristol Trials Centre), which has managed the conduct of the study throughout.

Healx Pushing New Drug Development Model in Rare Diseases with Barth Syndrome Foundation


Healx, a Cambridge (UK) technology company, today announced a collaboration with Boston Children’s Hospital and Barth Syndrome Foundation (BSF) to advance promising therapeutic compounds using a novel approach that will accelerate drug discovery for applications in Barth syndrome, a life-threatening, genetic mitochondrial disease.

Companies such as Healx are capitalizing on advances in artificial intelligence (AI) and merging technology with new findings in science to overcome the financial and time challenges to develop therapies in rare diseases. “We have to consider alternatives to the traditional drug discovery model,” said Dr. Tim Guilliams, CEO and co-founder, Healx. “Rare disease patients simply cannot rely on the traditional drug development process which doesn’t work in a timely fashion for such complex, commonly fatal diseases in niche patient populations.”

Powered by Firespring