Stealth Biotherapeutics says TAZPOWER results support previous findings
Stealth BioTherapeutics announced "positive" results from the open-label extension of the Phase 2/3 TAZPOWER study of elamipretide, an investigational drug, in patients with genetically confirmed Barth syndrome. Barth syndrome is an ultra-rare mitochondrial disease in which reduced levels of the mitochondrial phospholipid cardiolipin are associated with skeletal muscle weakness, debilitating fatigue, cardiac abnormalities, recurrent infections, delayed growth and reduced life expectancy. The TAZPOWER open-label results, presented at the 2019 MDA Clinical & Scientific Conference, demonstrated potential for elamipretide to improve the relative levels of normal to abnormal cardiolipin which are diagnostic for the disease, as well as measures of exercise performance and patient-reported outcomes. "These Phase 2/3 results support previous pre-clinical findings suggesting that elamipretide may help improve cardiolipin levels," said Dr. Hilary Vernon, TAZPOWER trial investigator, Associate Professor of Pediatrics at the McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine and Director, Barth Syndrome Clinic at Kennedy Krieger Institute, Baltimore, MD. "Based on the correlation of the cardiolipin ratio with disease severity, these changes in the cardiolipin ratio data may suggest that elamipretide has the potential to modify the course of disease in Barth syndrome rather than merely addressing the symptoms."