FinancialBuzz.com’s latest The Buzz Show: Featuring Our Corporate News Recap on “Stealth BioTherapeutics Corp. Surges Following Orphan Drug Designation from the European Medicines Agency”
Stealth BioTherapeutics Corp. (NASDAQ: MITO) surged 39% in premarket trading after the company received an orphan drug designation from the European Medicines Agency.
The designation is granted to investigational therapies intended for the safe and effective treatment of rare diseases with the Company having received the designation for elamipretide for the treatment of Barth syndrome, an ultra-rare genetic condition.
Stealth 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 heart, the eye, and the brain. We believe our lead product candidate, elamipretide, has the potential to treat both rare metabolic cardiomyopathies, such as Barth, Duchenne muscular dystrophy and Friedreich’s ataxia, rare mitochondrial diseases entailing nuclear DNA mutations, as well as ophthalmic diseases entailing mitochondrial dysfunction, such as dry age-related macular degeneration and Leber’s hereditary optic neuropathy. We are evaluating our second-generation clinical-stage candidate, SBT-272, and our new series of small molecules, SBT-550, for rare neurological disease indications following promising preclinical data. We have optimized our discovery platform to identify novel mitochondria-targeted compounds which may be nominated as therapeutic product candidates or utilized as mitochondria-targeted vectors to deliver other compounds to mitochondria.
For more information, please visit: Stealth BioTherapeutics Corp.
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