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.
This US FDA's FTD follows the company's TAZPOWER, a Phase 2/3, randomised, double-blind, placebo-controlled crossover study that will evaluate the effects of daily treatment with elamipretide in patients with genetically confirmed Barth syndrome.
Barth syndrome is a rare genetic mitochondrial disease, caused by mutations in the TAZ gene.
is caused by mutations in the tafazzin (TAZ) gene, which encodes a group of proteins called tafazzins.
Modeling the mitochondrial cardiomyopathy of Barth syndrome
with induced pluripotent stem cell and heart-on-chip technologies," Nature Medicine, vol.
Kate and Sandt Michener lost their first son, Rhys, to Barth Syndrome when he died very unexpectedly of dilated cardiomyopathy on 10 February 2011 at the age of 6 months.
The event at Neill's Taekwondo and Fitness, located at 1706 East Franklin Street Chapel Hill, NC 27514, is open to all with a suggested minimum donation to the Barth Syndrome Foundation of $5.
The engineered diseased tissue contracted very weakly, as would the heart muscle seen in Barth syndrome
Grant/funding Support: The research was supported by a grant of the Barth Syndrome
Foundation to W.
Newton, Massachusetts-based clinical-stage biopharmaceutical company Stealth BioTherapeutics has received Fast Track designation from US Food and Drug Administration for its lead candidate, elamipretide, for the treatment of Barth syndrome
, the company said.
The mean (SD) 3MGH activity in fibroblasts from five Barth syndrome
patients was 2.
Defective remodeling of CL and phosphatidylglycerol in Barth syndrome