In the study, mice were injected with viral particles that over-express human A53T alpha-synuclein, a mutated protein found in PD, in the substantia nigra, which is an area of the brain that plays a critical role in modulating motor movement. Following the injection, the mice displayed PD pathology, including a significant loss of dopaminergic neurons, which are responsible for relaying messages that control voluntary body movement. In addition, the animals displayed aggregation of alpha-synuclein and markers of neuroinflammation. Systemic daily administration of SBT-272 at two different doses significantly protected against the loss of dopaminergic neurons and the aggregation of pathological alpha-synuclein. In addition, the higher dose of SBT-272 significantly reduced two different markers of neuroinflammation. Overall, the data demonstrate that SBT-272 has a neuroprotective effect in this model of PD.
The neuroprotection activity observed in the PD model was supported by an additional collaborative study between
"We are excited about the promise of SBT-272 as a potential therapeutic for neurodegenerative diseases," said Chief Executive Officer
We are 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 syndrome, 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.
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential of SBT-272 to target mitochondrial dysfunction, the potential of SBT-272 as a treatment for neurodegenerative diseases such as PD and ALS, and Stealth's plans, strategies and expectations for its preclinical and clinical advancement of SBT-272 and its other pipeline candidates. Statements that are not historical facts, including statements about Stealth BioTherapeutics' beliefs, plans and expectations, are forward-looking statements. The words "anticipate," "expect," "hope," "plan," "potential," "possible," "will," "believe," "estimate," "intend," "may," "predict," "project," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Stealth BioTherapeutics may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of known and unknown risks, uncertainties and other important factors, including: Stealth BioTherapeutics' ability to obtain additional funding and to continue as a going concern; the impact of the COVID-19 pandemic; the ability to successfully demonstrate the efficacy and safety of Stealth BioTherapeutics' product candidates and future product candidates; the preclinical and clinical results for Stealth BioTherapeutics' product candidates, which may not support further development and marketing approval; investigational review boards at clinical trial sites and publication review bodies, which may affect the initiation, timing and progress of preclinical studies and clinical trials of Stealth BioTherapeutics product candidates;
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