BOSTON, March 9, 2023 /PRNewswire/ — Stealth BioTherapeutics Inc. (the “Company” or “Stealth BioTherapeutics”), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the company will present an overview of the elamipretide clinical development program in Barth syndrome at the 2023 American College of Medical Genetics Meeting, to be held March 14-18, 2023 in Salt Lake City, Utah. New data from week 168 of the Company’s open-label extension will be presented, demonstrating a greater than 45% improvement (mean 14.4 mL) in left ventricular stroke volume from baseline (mean 30.5 mL), indexed to baseline body surface (nominally significant, p=0.007). The oral presentation will showcase the robust combination of clinical studies used to evaluate the therapeutic potential of elamipretide in Barth syndrome, including a placebo-controlled study, an open-label extension study, and an observational natural history study.
Title: Optimization of Positive Investigational Outcomes in Barth Syndrome using a Combination of Placebo-controlled, Extended Open-label, and Natural History Comparison Studies
Presenter: Hilary J. Vernon, MD, PhD, FACMG
Date: Friday, March 17, 2023
Time: 4:45-5:00 pm MST
Location: Salt Palace Convention Center, Meeting Room: 251
“Ultrarare diseases like Barth syndrome present unique clinical and regulatory challenges to generating evidence of effectiveness. Through our innovative clinical development program, we were pleased to see that elamipretide appeared to contribute to improved exercise tolerance, muscle strength, and cardiac function for young men and boys affected by this life-threatening disease,” said Reenie McCarthy, Chief Executive Officer of Stealth. “Each step of these studies was crucial to evaluating clinical and physiological outcomes and highlights the need for creative data generation and commitment to the patient community when studying potential treatments for ultrarare diseases.”
The Company is 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 eye, the neuromuscular system, the heart and the brain. The Company believes that its lead product candidate, elamipretide, has the potential to treat ophthalmic diseases, such as dry AMD, rare neuromuscular disorders, such as primary mitochondrial myopathy, and rare cardiomyopathies, such as Barth syndrome. The Company is evaluating its second-generation clinical-stage candidate, SBT-272, for neurological disease indications, such as amyotrophic lateral sclerosis and Parkinson’s disease, following promising preclinical data. The Company has a deep pipeline of novel mitochondria-targeted compounds under evaluation as therapeutic product candidates.
SOURCE Stealth BioTherapeutics Inc.