Treatment candidate elamipretide did not meet expectations stemming from promising early trial results in patients with primary mitochondrial myopathy (PMM), data from a Phase 3 trial show. Elamipretide is an experimental medicine designed to treat both inherited primary mitochondrial diseases and mitochondrial dysfunction in age-related diseases. It…

Elamipretide (MT-131), developed by Stealth BioTherapeutics, is able to improve cardiac function in Barth syndrome patients, new data from a Phase 2/3 clinical trial show. Hilary Vernon, MD, PhD, a professor of pediatrics at Johns Hopkins Hospital, presented the findings in a presentation, titled “An interventional…

Alexion has entered into an agreement with Stealth BioTherapeutics to help — depending on the results of a Phase 3 clinical trial — co-develop elamipretide, an investigational therapeutic for several mitochondrial diseases. Elamipretide (MT-131) is a potential mitochondrial therapy that binds to parts of the inner membrane of mitochondria,…

Stealth BioTherapeutics has completed patient enrollment for its Phase 3 trial ongoing in the United States to evaluate efficacy and safety of daily under-the-skin injections of elamipretide (MT-131), an investigational treatment for primary mitochondrial myopathy (PMM)…

Stealth BioTherapeutics announced that it has raised $100 million from investors to support the development of elamipretide and other compounds that might treat diseases involving mitochondria dysfunction. “We are gratified by the support from these leading healthcare and venture capital investors,” Reenie McCarthy, CEO of Stealth, said in a…

Enrollment has been completed for a Phase 2/3 trial to evaluate the safety, effectiveness, and tolerability of investigational therapy elamipretide in patients with Barth Syndrome, a rare mitochondrial disorder, Stealth BioTherapeutics announced.  In July 2017, the company launched the randomized trial, called TAZPOWER (NCT03098797…

The U.S. Food and Drug Administration (FDA) has granted Stealth BioTherapeutics fast track designation for elamipretide as a treatment for people with Barth syndrome, a rare mitochondrial disease. The designation may shorten the time it takes for the treatment to reach patients. Elamipretide is currently being studied in the Phase…

Stealth BioTherapeutics‘ investigational medicine elamipretide, formerly known as Bendavia, has been granted orphan drug status by the U.S. Food and Drug Administration’s (FDA) for the treatment of primary mitochondrial myopathy (PMM). Stealth specializes in the development of drug candidates for mitochondrial disease treatment. The FDA’s Office of Orphan…

A Phase 2/3 clinical trial of elamipretide, a potential treatment for a rare mitochondrial disease known as Barth syndrome, is now enrolling patients, the therapy’s developer, Stealth BioTherapeutics, announced. The TAZPOWER study (NCT03098797) will be conducted in McKusick-Nathans Institute of Genetic Medicine, at the Johns Hopkins University School…

The investigational drug elamipretide has therapeutic potential to improve the physical capacity of patients with primary mitochondrial myopathy (PMM), according to the latest results of the Phase 2 MMPOWER-2 study. The positive results support the further clinical development of the drug to tackle this medical condition that lacks therapeutic options. These…